header logo image


Page 4«..3456..1020..»

Archive for the ‘Stem Cell Negative’ Category

Ozone in the air is bad for birds – Massive Science

Tuesday, January 5th, 2021

In 2007, scientists developed a method to determine the sexes of Atlantic walruses using only their jaws' size and shape. Researchers have now put that sexing (identification of an organism's sex) method to the test with Pacific walruses.

There was some doubt about whether this technique would work for one, Pacific walruses are significantly larger than their Atlantic counterparts. This size difference shows even in individual body parts, including the mandibles.

Yet, the team, led by Nathan Taylor at the University of Alaska, Fairbanks, persisted in applying the sex identification strategy since, if successful, it would significantly reduce the time and financial commitment needed for researching preserved, unidentified walrus specimens. To distinguish between male and female Pacific walruses, they measured the length and height of the jawbone, the minimum jawbone depth (from about the middle point of the jaw to the back), and jaw thickness.

A female Pacific walrus and a calf

USFWS via Wikimedia

The scientists had to be mindful of whether the jawbones were "not fully fused" (not fully developed, unique to juvenile walruses) or "fused" (fully developed, the sign of a mature walrus). Walruses with partially fused mandibles were likely to yield misleading results.

For example, jaws from male walruses that had not yet fully fused were similar in dimensions to mature females' jaws. To ensure the results were accurate, they could only include fully matured, fused specimens.

After measuring 67 modern specimens (33 of which were male, 24 belonging to females, and ten unknown) and 11 archaeological samples, the researchers concluded that jaw size is indeed a reliable body part to distinguish between male and female walruses. The most significant differences were jaw length and thickness, with females notably smaller in both categories.

A male Pacific walrus

Joel Garlich-Miller, USFWS, via Wikimedia

With the original sexing method now confirmed to work for Pacific walruses, scientists will be better prepared to perform several types of analyses, including measuring stable isotopes, trace elements, and hormones in study animals, with greater confidence and less risk of misidentification.

This is a crucial finding, given the insufficient data on Pacific walrus populations, and will hopefully push conservation efforts for this species forward.

Visit link:
Ozone in the air is bad for birds - Massive Science

Read More...

How good are the COVID-19 vaccines? – Massive Science

Tuesday, January 5th, 2021

In 2007, scientists developed a method to determine the sexes of Atlantic walruses using only their jaws' size and shape. Researchers have now put that sexing (identification of an organism's sex) method to the test with Pacific walruses.

There was some doubt about whether this technique would work for one, Pacific walruses are significantly larger than their Atlantic counterparts. This size difference shows even in individual body parts, including the mandibles.

Yet, the team, led by Nathan Taylor at the University of Alaska, Fairbanks, persisted in applying the sex identification strategy since, if successful, it would significantly reduce the time and financial commitment needed for researching preserved, unidentified walrus specimens. To distinguish between male and female Pacific walruses, they measured the length and height of the jawbone, the minimum jawbone depth (from about the middle point of the jaw to the back), and jaw thickness.

A female Pacific walrus and a calf

USFWS via Wikimedia

The scientists had to be mindful of whether the jawbones were "not fully fused" (not fully developed, unique to juvenile walruses) or "fused" (fully developed, the sign of a mature walrus). Walruses with partially fused mandibles were likely to yield misleading results.

For example, jaws from male walruses that had not yet fully fused were similar in dimensions to mature females' jaws. To ensure the results were accurate, they could only include fully matured, fused specimens.

After measuring 67 modern specimens (33 of which were male, 24 belonging to females, and ten unknown) and 11 archaeological samples, the researchers concluded that jaw size is indeed a reliable body part to distinguish between male and female walruses. The most significant differences were jaw length and thickness, with females notably smaller in both categories.

A male Pacific walrus

Joel Garlich-Miller, USFWS, via Wikimedia

With the original sexing method now confirmed to work for Pacific walruses, scientists will be better prepared to perform several types of analyses, including measuring stable isotopes, trace elements, and hormones in study animals, with greater confidence and less risk of misidentification.

This is a crucial finding, given the insufficient data on Pacific walrus populations, and will hopefully push conservation efforts for this species forward.

See the original post:
How good are the COVID-19 vaccines? - Massive Science

Read More...

Stem cells from cord blood can now be used across many conditions: Mayur Abhaya, MD & CEO, LifeCell Internat.. – ETHealthworld.com

Monday, December 28th, 2020

Shahid Akhter, editor, ETHealthworld, spoke to Mayur Abhaya, MD & CEO, LifeCell International, to know more about the latest advancements in Stem cell industry and how it has recovered from the Covid challenges.Impact and challenges of Covid-19 on the Stem cell industry ?One of the biggest issues faced by the stem cell industry during the pandemic was the transport of the cells after collection at birth. It needs to reach the lab within 72 hours and in the case of bone marrow stem cell from donation all the way until it reaches the patient that also has to be completed within 72 hours. The bone marrow cells cannot be handed over in courier. It has to be manually hand carried and that created a huge logistics hurdle were transplants significantly reduced in numbers because of the availability of donors and the transport issues around it.

What are the current global trends in the Stem Cell Industry at large?The recent clinical progress that has been made in the medical space of stem cell transplantation is that now cord blood is considered as a better source than bone marrow cells. This was found in a research study based in US, published in 2020. They have also shown that stem cells from the cord blood can now be used across many conditions with the same treatment protocol. Besides that, the preparation of the patient is different in different conditions but now they have simplified that and reduced the risk of death to a very, very low number. So the cord blood is preferred, as the outcomes are improving.How has Life Cell managed the scenario during the pandemic? Do let us know your challenges and the way forward plan?One of the biggest issues during the pandemic was transport, especially the flight operations because we heavily depend on them for moving the samples across the country. We had to revert to an alternate plan where we had to transport these samples through a relay network from one city to another, through a road network. Luckily LifeCell has operations across the country covering more than 250 cities. So still we had the ability to ensure that our commitment of getting the samples to the lab within those 72 hours was very much possible.

Another major milestone during this pandemic that we were able to help was to support a transplant were a child having Aplastic Anaemia needed not one but two cord blood units for the transplant and within the family they couldnt find a match. Luckily because of the LifeCell network and the inventory size of 50,000 units we were able to meet the requirements of the transplant and happy to share the outcome was very successful. So LifeCell ensures that we have appropriate training for its paramedical staff and they are also provided with the appropriate personal protective gears. There are restrictions on the entry of the team inside during the collection we work with the medical staff in the collection rooms, in the operation theatres to ensure a smooth and a well organised collection and even at the lab we have protocols that ensures hygiene and safety within the team and, the operating rooms we have for processing are also well managed.

Your future plans to ensure the smooth collection of Cord Blood?To ensure business continuity we have our teams located very close to our lab itself, you know, so about 100+ member team are placed within a Kilometer of the operating facility. We have adequate stocks, lots of the testing and the processing, consumables that we use are imported. We, at least, maintain 3 month inventory. We also have onsite power back up systems which include a month of diesel supply, month of liquid nitrogen supply and the teams also have a plan that we have a back site also with arrangements done. If for any reason we have cut off of the Chennai centre we have arrangements with an alternate lab to ensure the continuity of the operations

Link:
Stem cells from cord blood can now be used across many conditions: Mayur Abhaya, MD & CEO, LifeCell Internat.. - ETHealthworld.com

Read More...

Allogeneic SCT Benefits Children and Adolescents With Relapsed Anaplastic Large Cell Lymphoma – OncLive

Monday, December 28th, 2020

Findings from the International, Prospective ALCL-Relapse trial [NCT00317408] showed that allogeneic SCT after reinduction chemotherapy can lead to survival improvements in children and adolescents with high-risk relapsed or refractory anaplastic large cell lymphoma (ALCL), provide more insight into how to treat this population.

For patients with early-relapsed ALCL, data showed that autologous SCT wasnt effective.

Current standard chemotherapy reaches an event-free survival (EFS) of 70% at 5 years in children with ALCL, the study authors wrote. Retrospective data on the outcome of pediatric relapsed ALCL show a survival after relapse of more than 50%. In addition, there is no consensus on the optimal treatment approach in relapse, they explained.

Designed by the European Inter-Group for Childhood Non-Hodgkin Lymphoma, the prospective, stratified, multinational clinical trial was opened for patients at sites in 5 countriesthe United Kingdom, Germany, Austria, Switzerland, and the Czech Republic.

Patients were stratified according to the time of relapse, CD3 expression, and prior vinblastine therapy to 3 different consolidation approaches: allogeneic hematopoietic stem cell transplantation (SCT), autologous SCT, or vinblastine monotherapy.

Those whose disease progressed during frontline therapy (very high risk) or with a CD3-positive relapse (high risk) received allogeneic SCT after reinduction chemotherapy. Patients with a CD3-negative relapse within 1 year after initial diagnosis or prior exposure to vinblastine (intermediate risk) received autologous SCT after carmustine, etoposide, cytarabine, and melphalan (BEAM). However, this arm was terminated prematurely, and patients received vinblastine monotherapy instead. Patients with a CD3-negative relapse more than 1 year after initial diagnosis (low risk) received by weekly vinblastine monotherapy (6 mg/m2; maximum, 10 mg) for 24 months.

Investigators analyzed 105 patients; most were male and had ALK-positive tumors. The median age was 12.4 years and median time from initial diagnosis to relapsed/refractory disease was 8.5 months. Patients were recruited from April 2004 to February 2014 and the median follow-up time was 8.1 years.

At 5 years, overall survival (OS) in patients with central nervous systemnegative disease was 78% 4% and EFS, the primary endpoint, was 53% 5%.

Before termination of autologous SCT, EFS rates of patients in the very-high- (n = 17), high- (n = 26), intermediate- (n = 32), and low- (n = 21) risk groups were 41% 12%, 62% 10%, 44% 9%, and 81% 9%. The respective OS rates were 59% 12%, 73% 9%, 78% 7%, and 90% 6%.

Analyzing only the patients in the intermediate-risk group consolidated per protocol by autologous SCT, EFS and OS of 23 patients were 30% 10% and 78% 9%, respectively. The 5 patients with intermediate risk receiving vinblastine monotherapy experienced relapse again.

Compared with data from retrospective analyses, the survival of patients with refractory or relapsed ALCL reached 75% in our prospective trial, demonstrating that relapsed ALCL remains a curable disease, the study authors wrote.

The main limitation to the study was the implementation of the trial as treatment recommendation only in some countries, noted the authors. While recommendations were followed in approximately 90% of patients in the very high risk and high-risk groups, only 70% of patients in the intermediate risk group received autologous SCT, they explained.

Overall, investigators determined that patients with high-risk relapsed disease can benefit from allogeneic SCT and offer a chance for cure.

A long-term remission rate of 81% by outpatient vinblastine monotherapy, with low risk for late effects in patients with a late relapse was also observed by investigators. However, the monotherapy wasnt effective for early relapses.

Patients with early relapsed ALCL dont benefit from consolidation by autologous SCT or vinblastine monotherapy, the authors wrote. However, they may benefit from clinical trials testing a consolidation approach including new targeted therapies. Targeted agents should be tested as reinduction for all but late relapses. Given the efficacy of vinblastine in relapse, this shift-of-paradigm approach should be tested for low-risk patients front line.

More:
Allogeneic SCT Benefits Children and Adolescents With Relapsed Anaplastic Large Cell Lymphoma - OncLive

Read More...

CalvinAyre.com’s most read life stories of 2020 – CalvinAyre.com

Monday, December 28th, 2020

Its time to look back at some of the stories that interested Calvinayre.com readers. Here are the top five stories from the life section that captured the imagination of readers in 2020.

Turn on the Red Light if you want better eyesight

Red lights arent initially associated with healthy eyesight; most people associate red lights with negative situations stop lights, warning lights and even the red-light district. An article that appeared in The Journals of Gerontology puts a positive spin on red lights. The study found that exposure to red lights for a few minutes each day keeps eyesight in better shape as we age.

Next Jeopardy! host odds: Ken Jennings favored over TV hosts

The passing of famed television host Alex Trebek meant that Jeopardy would need a new host. Ken Jennings was chosen to step into the large shoes of Trebek, at least part time, but will he be the permanent host?

Stem Cell Therapy already helping Madonna and Mike Tyson stay young

Mike Tyson and Madonna both swear by stem celling therapy. The Material Girl made the headlines for undergoing stem cell therapy to fix a busted knee. Former heavyweight champ Tyson used the therapy to help him stay youthful. In Tysons case, it seems to have done the trick with a trilogy fight against Evander Holyfield on the cards for 2021.

Ten years in the digital trenches: life as a CalvinAyre.com writer

An industry favourite, Steve Stradbrooke took a quick walk down memory lane to celebrate a decade as a senior writer at Calvinayre.com. Stradbrooke was one of the first on the team in the early days of Calvinayre.com. Buy him a pint to hear some of his stories from the wild west days of Costa Rica to Black Friday.

NASA to give the moon Internet access, SpaceX to give it to Earth

Well end this on a positive note for 2020. Its good to know when we do our first trip to the moon, well have decent internet access. NASA partnered with Nokia to create a cellular network for the moon, while Elon Mush and SpaceX have been creating an internet network from satellites.

Check out the rest of our 2020 year in review.

Read this article:
CalvinAyre.com's most read life stories of 2020 - CalvinAyre.com

Read More...

Coronavirus | Over 6,000 travellers from U.K. traced across States – The Hindu

Monday, December 28th, 2020

Passengers testing positive moved to separate quarantine units.

In line with a Health Ministry directive, several States have stepped up efforts to trace and test persons who have flown in or transited from the United Kingdom between November 25 and December 23, for a more infective version of the COVID-19 virus. Flights between India and the U.K. remain suspended till December 31.

Eleven persons, on board four flights, tested positive for COVID-19 in Delhi and are now admitted to the government-run LNJP Hospital. The Delhi government has also asked the hospital to create a separate isolation unit for passengers from the U.K. who test positive.

Coronavirus | New virus strain increases transmissibility, not severity: V.K. Paul

Around 950 passengers from London were tested at the airport and 11 of them were found positive, according to Genestrings Lab, which conducted the testing. A further 50 people were put under quarantine. Positive samples have been sent to the National Centre for Disease Control for genome sequencing.

In Tamil Nadu, the Health Department tracked 2,724 arrivals from or through the U.K. on different routes to the State from November 25 to December 23.

We found that 179 were double entries. The reconciled figure of those from the U.K. to the State is 2,724. Of this, till Wednesday, we have tracked 996 persons. We have lifted samples from 516 persons. So far, 203 samples have returned negative for COVID-19, Health Secretary J. Radhakrishnan said.

Coronavirus | U.K. strain unlikely to affect efficacy of vaccines, say scientists

The Health Department is also monitoring 111 persons who had moved to a different district after returning. As of Thursday, only one person a 25-year-old man who had returned from the UK by air via Delhi had tested positive and is undergoing treatment in Chennai.

In Kerala, eight persons have tested positive for COVID-19, from amongst the 1,609 travellers from the UK, who reached Kerala between December 9 -23.

As part of the intensified surveillance that has been initiated in the State, health officials have traced a total of 2,116 passengers from UK who arrived in the State till December 23.

Explained | The new coronavirus variant in Britain

All 1,609 passengers were tested using RT-PCR and they will be followed up for the next 14 days. Even those who tested negative will be re-tested using RT-PCR after the quarantine. Apart from the eight passengers who tested positive, their household contacts will also be tested using PCR.

The nasopharyngeal samples from the eight persons who have tested positive for COVID-19 will be sent to the National Institute of Virology, Pune for genome sequencing so that we may identify the virus strain. We have also identified DBT-inStem (Institute for Stem Cell Science and Regenerative Medicine), in Bangalore as another location where we can do genome sequencing, the official said.

A total of 1,016 of the 2,127 U.K. returnees who arrived in Karnataka from December 1 till December 22 have been tested. Out of them six have tested positive and their samples have been sent for genome sequencing.

No home isolation is allowed for passengers from the U.K. who test positive, irrespective of symptoms, till their genome sequencing is completed. Three persons have so far have tested positive and their genome sequencing results are awaited. The sequencing of the positive samples (done to ascertain if they are infected with the new strain of the virus) is likely to take at least four days.

Coronavirus | All international passengers arriving in Karnataka without COVID-19 negative report to be tested

Till then, those who test positive will be isolated in a separate unit in an institutional isolation facility, Jawaid Akhtar, Additional Chief Secretary (Health and Family Welfare) told The Hindu.

Seven persons, of a total 1,200, who had flown in to Hyderabad either directly or via U.K. since December 9, have tested positive for the coronavirus. The samples of these seven positive cases are being sent to the Centre for Cellular and Molecular Biology (CCMB) Hyderabad to check whether the affected persons had the mutated U.K. strain.

Coronavirus | New, more contagious strain in U.K. does not appear to be deadlier: Vivek Murthy

A press release later said officials were trying to trace the primary contacts of positive cases. The health status of those who tested negative was also being monitored. Out of 1,200 UK returnees, as many as 846 persons were traced and tested. Barring the seven, all others tested negative.

A woman who returned from the UK to Rajahmundry has been kept in isolation after testing positive. Overall, information is being gathered on 68 persons who have returned to Andhra Pradesh from the U.K. The Health Department sounded an alert on the likelihood of the spread of the new viral strain on Tuesday and stressed the need for enhanced epidemiological surveillance.

The Chittoor district administration received a list of 38 passengers who came from London via New Delhi and Bengaluru on Thursday. Efforts are on to know more details about them, according to District Medical and Health Officer (DMHO) M. Penchalaiah, who said surveillance teams had been formed to trace them.

(With Bureau inputs)

You have reached your limit for free articles this month.

Find mobile-friendly version of articles from the day's newspaper in one easy-to-read list.

Enjoy reading as many articles as you wish without any limitations.

A select list of articles that match your interests and tastes.

Move smoothly between articles as our pages load instantly.

A one-stop-shop for seeing the latest updates, and managing your preferences.

We brief you on the latest and most important developments, three times a day.

Support Quality Journalism.

*Our Digital Subscription plans do not currently include the e-paper, crossword and print.

Go here to see the original:
Coronavirus | Over 6,000 travellers from U.K. traced across States - The Hindu

Read More...

Exosomes act as messengers and decoys to save healthy cells from viral infection – Massive Science

Monday, December 28th, 2020

In 2007, scientists developed a method to determine the sexes of Atlantic walruses using only their jaws' size and shape. Researchers have now put that sexing (identification of an organism's sex) method to the test with Pacific walruses.

There was some doubt about whether this technique would work for one, Pacific walruses are significantly larger than their Atlantic counterparts. This size difference shows even in individual body parts, including the mandibles.

Yet, the team, led by Nathan Taylor at the University of Alaska, Fairbanks, persisted in applying the sex identification strategy since, if successful, it would significantly reduce the time and financial commitment needed for researching preserved, unidentified walrus specimens. To distinguish between male and female Pacific walruses, they measured the length and height of the jawbone, the minimum jawbone depth (from about the middle point of the jaw to the back), and jaw thickness.

A female Pacific walrus and a calf

USFWS via Wikimedia

The scientists had to be mindful of whether the jawbones were "not fully fused" (not fully developed, unique to juvenile walruses) or "fused" (fully developed, the sign of a mature walrus). Walruses with partially fused mandibles were likely to yield misleading results.

For example, jaws from male walruses that had not yet fully fused were similar in dimensions to mature females' jaws. To ensure the results were accurate, they could only include fully matured, fused specimens.

After measuring 67 modern specimens (33 of which were male, 24 belonging to females, and ten unknown) and 11 archaeological samples, the researchers concluded that jaw size is indeed a reliable body part to distinguish between male and female walruses. The most significant differences were jaw length and thickness, with females notably smaller in both categories.

A male Pacific walrus

Joel Garlich-Miller, USFWS, via Wikimedia

With the original sexing method now confirmed to work for Pacific walruses, scientists will be better prepared to perform several types of analyses, including measuring stable isotopes, trace elements, and hormones in study animals, with greater confidence and less risk of misidentification.

This is a crucial finding, given the insufficient data on Pacific walrus populations, and will hopefully push conservation efforts for this species forward.

Link:
Exosomes act as messengers and decoys to save healthy cells from viral infection - Massive Science

Read More...

Celtics adjust to two-game series designed to reduce team travel – The Boston Globe

Monday, December 28th, 2020

These are pretty quiet times, Stevens said. I havent left the hotel since Ive been here. And obviously no one from our side can travel unless theyre part of our tested group every single day. All of that are parts of the new unique experiences of being on the road.

Teague said he was able to spend the day after Christmas with his family after all of them tested for negative for COVID-19.

This whole season has been weird, he said. Its had its challenges and curveballs. I leave (the hotel) and then I go home, so its a different experience for me. Its different for me because Im from here. I enjoy being here this long.

Stevens said the two-game series should be something the league considers beyond the pandemic. In January, the Celtics host the Orlando Magic for two straight games and travel to Philadelphia for two. In February, they host Atlanta for a pair.

It makes sense from a travel perspective, a health perspective, Stevens said. Its not just about the COVID health part; its also about the sleep and the opportunity to not overdo it. Theyre playing all the time and if we could lessen the miles of all these teams are traveling, I think thats really important. So I like the idea of a series.

No free throws for Tatum

Jayson Tatum has yet to attempt a free throw this season despite playing 69 minutes and attempting 50 shots through the first two games. Meanwhile reserve forward Semi Ojeleye has attempted seven, including six in the Christmas Day loss to the Brooklyn Nets.

The Celtics are hoping to find a way for Tatum to score easier points, and drawing shooting fouls would help boost what has been an inconsistent offense at times.

No. 1 is, youre always looking to take what the defense gives you, Stevens said. They are very dialed into him. Hes done a good job attacking the rim I thought against Brooklyn. He could finish better at the rim. Hes gonna be such a focal point I think weve talked about trying to get a few easier baskets. Any time you can get to the line its a good thing for us because he makes them. But his lack of getting to the line has a lot more to do with our spacing and our execution that it has with his game.

Free throw attempts arent only a Tatum problem. The Celtics entered Sundays game ranked 29th in the league in free throw attempts (15). Atlanta leads the NBA with 33.5 attempts per game.

Thompson update

Tristan Thompson was again in the starting lineup on a minutes restriction because of a strained left hamstring. He has been limited to 20-plus minutes in the first two games and the Celtics have not revealed when the restriction would be lifted.

Of course Id like to be on the floor with my teammates more but Ive got to trust the training staff, he said. As games go on, hopefully theyll take that restriction [off] , so I can go out and be myself.

Thompson is a plus defender but assigning him to Kevin Durant on Friday could be perceived as an impossible task. Thompson said he relished the matchup. Durant finished with 29 points.

Ive guarded LeBron (James), KD (Durant), Steph (Curry), Kawhi (Leonard), he said. Ill take that challenge every night, I trust myself. Its totally up to the training staff. Id love to play 30 minutes right now and go full speed and go full blast.

Walker on the floor

Kemba Walker traveled with the Celtics and went through a pre-game workout that consisted of shooting 3-pointers and free throws. According to Stevens, Walker has not done anything beyond individual work but Walker did look comfortable and nimble on his left knee, which received a stem cell injection in October. The Celtics initially said Walker would return in January but the club is expected to be cautious with his recovery and it could be several weeks before he returns to action.

Gary Washburn can be reached at gary.washburn@globe.com. Follow him on Twitter @GwashburnGlobe.

Go here to read the rest:
Celtics adjust to two-game series designed to reduce team travel - The Boston Globe

Read More...

Experts Reflect on Most Impactful FDA Moves of 2020 in Solid Tumors, Hematologic Malignancies – Targeted Oncology

Monday, December 28th, 2020

Despite the rapid spread of the coronavirus disease 2019 (COVID-19) that plagued not only the oncology field but the healthcare system as a whole, the treatment options for patients with countless different solid tumors and hematologic malignancies were expanded with a number of new FDA approvals indicated throughout 2020. These approvals cover updates in lung, breast, gastrointestinal (GI), and genitourinary (GU) cancers, as well as a variety of hematologic malignancies and other solid tumors.

On Twitter, Targeted Oncology asks in a poll, In what field do you think had the most impactful additions to its armamentarium? The options include lung cancer, breast cancer, hematologic malignancies, and gastrointestinal cancers.

As the year comes to a close, the FDA continues to advance the field with more approvals coming through. On Friday, December 18, 2020, alone, the FDA granted 5 indications approval. These include ponatinib (Iclusig) for treatment of adult patients withs chronic-phase chronic myeloid leukemia who have become resistant or intolerant to therapy following at least 2 prior tyrosine kinase inhibitors (TKIs), osimertinib (Tagrisso) as adjuvant treatment of patients with nonsmall cell lung cancer (NSCLC) whose tumors harbor an EGFR exon 19 deletion or exon 21 L858R mutation for use following tumor resection, the triplet regimen selinexor (Xpovio) plus bortezomib (Velcade) and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least 1 prior therapy, oral relugolix (Relumina) for the treatment of advanced prostate cancer, and a biosimilar to rituximab (Rituxan) was approved for the treatment of adult patients with non-Hodgkin lymphoma (NHL), chronic lymphocytic leukemia (CLL), granulomatosis with polyangiitis, and microscopic polyangiitis.

These approvals follow many others that have provided hope throughout the year to physicians and healthcare providers across the United States, but 1 striking area of advancements worth noting this year is the recently approved Emergency Use Authorizations for 2 separate vaccinations for the prevention of coronavirus disease 2019 (COVID-19), which plagued the world, particularly impacting oncology practices treating immunocompromised, sick patients. These approvals, including the BNT162b2 vaccination and the mRNA-1273 Moderna COVID-19 Vaccine on December 18, 2020, demonstrate a crucial step in overcoming the pandemic.

We've learned this year what we can do quickly without being overly hasty, and I think COVID-19 was the ultimate impetus for unity, Mark Lewis, MD, director, GI Oncology, Intermountain Healthcare, told Targeted Oncology. At my center, which is not primarily a research institution, we've run almost 40 trials around COVID-19, largely in-patient, but also largely driven by the clinical need and queries of our intensivists, among others. It's shown us that given the right prompt, you can actually move pretty fast, and learn things very quickly.

Despite the challenges brought on by the pandemic, the healthcare system remained dedicated to keeping patients with cancer safe. Telehealth played a major role in this, as well as other adjustments physicians and cancer centers have made in light of this pandemic. These changes are not only keeping patients safe during these unprecedented times, but they lay the groundwork for the future of treating these patients better in the community setting.

I think we've learned what has to be done at a major research institution and what can be done either at a [community] center or even in the patient's home, said Lewis. Oncology, for a long time, unfortunately, has not been very patient-centric. The idea has been, you're sick, but you need to come to us. Obviously, there are still patients that absolutely have to make that effort to come see us, but on the flipside, we've learned what we can you do remotely and what we can do to bring treatment and research to the patient rather than the other way around.

One important update in the field of oncology in light of the COVID-19 pandemic was the FDAs approval of a new dosage for pembrolizumab (Keytruda) in April 2020, reducing the frequency of clinic visits for patients with cancer. The prior dosage for this immune checkpoint inhibitor was 200 mg every 3 weeks, which remains an approved dosing option, but the newly approved dose of 400 mg is administered every 6 weeks, which is approved across all indications whether the PD-1 inhibitor is given as a monotherapy or in a combination regimen. Pembrolizumab is approved across many disease types and has also gained tumor agnostic approvals over the last few years as well.

We have these really sweeping histology agnostic approvals, and I think the biggest 1 that comes to mind for me is the FDA setting eligibility for immune therapy at a tumor mutational burden [TMB] of greater than 10 mutations/megabase. I think that was pretty amazing, said Lewis. Just in the last couple years, we've seen this shift towards histology agnosticism, whether it's about NTRK fusion proteins, which have become a unicorn in oncology that every oncologist looks for, or we're talking about microsatellite instability [MSI]-high status and eligibility for pembrolizumab.

The immune checkpoint inhibitor pembrolizumab received approval in June 2020, for use in adult and pediatric patients with unresectable or metastatic solid tumors with tissue TMBhigh who have progressed on prior therapy and have no satisfactory alternative treatment options. This marks the second tumor-agnostic approval for the agent, following a prior approval from 2017, for the treatment of patients with MSIhigh or mismatch repair deficient solid tumors.

While we have these very broad indications, now we're also seeing every cancer become a rare cancer. One thing I've seen [more of] this year is selecting out molecular subsets of disease, and a great example is the approval of pralsetinib [formerly BLU-667; Gavreto] for RET-positive tumors, whether of the lung or the thyroid, Lewis said. I think we're seeing that across the categories, which is just site of origin largely, and you can see these mutations that might occur in both lung and thyroid, for the example of RET, and presumably be targetable with the same agent.

Pralsetinib (Gavreto)gained approval in September 2020 for the treatment ofRETfusion-positive NSCLC based on findings from the phase 1/2 ARROW study (NCT03037385), and a few short months later, the RET inhibitor also received approval for use in patients with advanced or metastaticRET-mutant medullary thyroid cancer, as well as those with RET fusion-positive thyroid cancer, also supported by the ARROW data.

Similarly, selpercatinib (formerly LOXO-292; Retevmo) capsules, was approved the treatment of patients with either lung or thyroid cancer who harborRETalterations. This therapy received approval in May 2020, marking it the first approved treatment to target RET. Specifically, the indications for selpercatinib include adult patients with metastatic RET fusionpositive NSCLC, adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer who require systemic therapy, or patients with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine (RAI)refractory, if RAI was appropriate.

Several new therapies gained approval in lung cancer in 2020 across a variety of subsets. In particular, capmatinib (Tabrecta) was approved in May 2020, for the treatment of patients with metastatic NSCLC whose tumors have aMETexon 14 skipping mutation based on the findings from the phase 2 GEOMETRY mono-I study (NCT02414139). This approval fills a gap in the landscape for patients with NSCLC since no other approved therapies target the MET exon 14 mutation in advanced disease.

In March 2020, the FDA approved durvalumab (Imfinzi) as a frontline treatment for adult patients with extensive-stage small cell lung cancer (ES-SCLC) in combination with standard-of-care chemotherapy, etoposide and carboplatin, or cisplatin, offering a new therapeutic approach to a population of patients who have had limited treatment options up until recently. The approval was based on findings from the phase 3 CASPIAN study (NCT03043872), which showed overall survival (OS) analyses favored the durvalumab arm across patient subgroups compared with chemotherapy alone.

The field of breast cancer saw a number of new advances during the year of 2020, many of which impacting patients with metastatic and advanced disease. The FDA granted approval across a number of subsets of patients, including those with HER2-positive disease, triple-negative breast cancer (TNBC), and hormone receptor (HR)-positive disease.

In each of these areas, in the very recent past, there's been a marked expansion of therapeutic options, William J. Gradishar, MD, chief of hematology and oncology, department of medicine, Betsy Bramsen Professorship of Breast Oncology, professor of medicine (hematology and oncology), Northwestern University's Feinberg School of Medicine, told Targeted Oncology. We still have a lot of work to do, patients are still dying of metastatic breast cancer, but we've made significant progress in not only preventing recurrences from developing in patients with early-stage disease, but we've also extended the survival of patients who have metastatic disease.

The FDA granted approval in February 2020 to neratinib (Nerlynx) in combination with capecitabine for the treatment of adult patients with advanced or metastatic HER2-positive breast cancer who received at least 2 prior anti-HER2-based regimens in the metastatic setting. This approval was based on supportive findings from the phase 3 NALA trial (NCT01808573). This study showed a 24% reduction in the risk of disease progression or death compared with lapatinib (Tykerb) and capecitabine. Prior to this, neratinib was approved for use as extended adjuvant treatment in adult patients with early-stage HER2-positive disease following adjuvant trastuzumab-based therapy.

In combination with trastuzumab (Herceptin) and capecitabine, tucatinib (Tukysa) received FDA approval for the treatment of adult patients with advanced unresectable or metastatic HER2-positive breast cancer in April 2020, including patients with brain metastases who have received at least 1 prior line of HER2-based therapy in the metastatic setting. This approval sparked a lot of excitement in the field, receiving its indication from the FDA 4 months ahead of the Prescription Drug User Fee Act (PDUFA) target action date. The oral, small molecule TKI of HER2 received its approval based on the phase 2 HER2CLIMB study (NCT02614794), which showed a 46% reduction in the risk of disease progression or death among heavily pretreated patients with unresectable, locally advanced, or metastatic HER2-positive disease.

An abundance of new drugs in the HER2 space is a great thing because we can go from 1 therapy to the next. For patients with advanced disease, tucatinib and neratinib are examples of that; these all expand the number of options we have for patients, and tucatinib in particular shows clear evidence of activity in the brain, which is critically important, Gradishar said. In the triple-negative space, sacituzumab govitecan [Trodelvy] is an example of an antibody-drug conjugate [ADC] that has activity, and then, of course, the approval of pembrolizumab has expanded the number of treatment options we have for patients with triple-negative disease.

In April 2020, sacituzumab govitecan received accelerated approval for the treatment of adult patients with metastatic TNBC who have received at least 2 prior lines of therapy for metastatic disease, based on the findings from the phase 3 ASCENT study (NCT02574455). The study demonstrated that sacituzumab govitecan, the first ADC approved specifically in mTNBC, had induced an objective response rate (ORR) of 33.3% (95% CI, 24.6%-43.1%), and the clinical benefit rate was 45.4%, which included stable disease for at least 6 months.

The immune checkpoint inhibitor pembrolizumab plus chemotherapy was approved for the treatment of patients with locally recurrent unresectable or metastatic TNBC whose tumors express PD-L1, and this news was joined by approval of the PD-L1 Immunohistochemistry (IHC) 22C3 pharmDx as a companion diagnostic for identifying patients likely to derive benefit from this therapy. The FDA granted this approval based on the findings from the phase 3 KEYNOTE-355 clinical trial (NCT02819518), which showed statistically significant and clinically meaningful improvement in the median progression-free survival (PFS) of 9.7 months with pembrolizumab and chemotherapy compared with 5.6 months in the chemotherapy-alone arm (HR, 0.65; 95% CI, 0.49-0.86; one-sided P =.0012).

Among other updates in breast cancer, the combination of pertuzumab, trastuzumab, and hyaluronidase-zzxf (Phesgo) by subcutaneous injection was approved by the FDA in June 2020 for the treatment of patients with HER2-positive breast cancer that has spread to other parts of the body, as well as for the treatment of adult patients with early HER2-positive breast cancer.This approval, which came 4 months ahead of the PDUFA date, is the first regimen approved for subcutaneous administration that contains 2 monoclonal antibodies. In the FeDeriCa study (NCT03493854), Phesgo was found to have comparable efficacy and safety to the intravenous regimen of pertuzumab and trastuzumab, meeting the studys primary end point of non-inferiority.

There have been a lot of fantastic FDA approvals in the hematologic malignancy space, and obviously, we have to think about multiple myeloma, said Naveen Pemmaraju, MD, associate professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, in an interview with Targeted Oncology. When I started out as a faculty 10 years ago, there were just not that many drugs approved, and so in the multiple myeloma space now as a consequence of 5 to 10 years of clinical trials, many of which were negative, we are seeing the appearance of some of these drugs.

Multiple myeloma saw approval of a number of new indications throughout 2020, including the May 2020 approval ofdaratumumab in combination with hyaluronidase-fihj (Darzalex Faspro) as treatment of adult patients with newly diagnosed or relapsed/refractory multiple myeloma, allowing a subcutaneous dosing of daratumumab. The regimen is approved under several indications for patients with multiple myeloma based on findings from the phase 3 COLUMBIA (NCT03277105) and the PLEIADES (NCT03412565) clinical trials. This approval offers patients a more convenient therapeutic option as the fixed-dose injection is administered in approximately 3 to 5 minutes, considerably reducing the treatment burden for these patients.

The immunoconjugate targeting B-cell maturation antigen (BCMA) belantamab mafodotin-blmf (GSK2857916; Blenrep) gained approval in August 2020, for the treatment of patients with relapsed/refractory multiple myeloma who previously received at least 4 prior lines of therapy, which should include an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody.This is the first anti-BCMA therapy available for these patients anywhere in the world, which was supported by a unanimous vote from the FDAs Oncologic Drugs Advisory Committee. Based on findings from the phase 2 DREAMM 2 clinical trial (NCT03525678) exploring 2.5-mg/kg and 3.4-mg/kg doses, the lower dose received the FDAs recommendation as an intravenous infusion given over approximately 30 minutes once every 3 weeks.

Selinexor, the only nuclear export inhibitor approved by the FDA for use in 2 hematologic malignancies, generated excitement in the field. This includes the triplet regimen of selinexor with bortezomib/dexamethasone in multiple myeloma that gained approval in December 2020, as well as the accelerated approval for single-agent treatment with selinexor in adult patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, who have had at least 2 prior systemic therapies, including patients with DLBCL arising from follicular lymphoma. This marks the only single-agent oral therapy approved for patients with relapsed/refractory DLBCL.

We saw a lot of movement in the DLBCL space. Up until before CAR T was approved, frankly, we had almost nothing besides chemotherapy. Now in the span of a year, we not only have CAR T-cell therapy, but we have a couple of other agents including tafasitamab which is in combination with lenalidomide and is an active antibody against CD19, Andrew M. Evens, DO, MSc, of the Rutgers Cancer Institute of New Jersey, told Targeted Oncology. Selinexor is the other FDA-approved agent that is an option for patients, so its promising to me in a very difficult disease, especially when relapsed/refractory, just to have now a multitude of agents approved.

The combination of tafasitamab-cxix (Monjuvi) with lenalidomide (Revlimid) was approved for the treatment of adultpatients with relapsed/refractory DLBCL not otherwise specified, including disease arising from low-grade lymphoma and patients who are not eligible for autologous stem cell transplant. The accelerated approval was granted on the basis of findings from the phase 2 L-MIND study (NCT02399085) on the safety and efficacy, as well as the observational retrospective cohort RE-MIND study (NCT04150328) on the real-world use of tafasitamab, the CD19-directed monoclonal antibody.

The approval of tafasitamab and lenalidomide came up this year, and that was certainly a very exciting approval. It is a very innovative chemotherapy-free approach to treatment of patients with relapsed/refractory DLBCL, Alexey V. Danilov, MD, PhD, associate professor of medicine at the Oregon Health & Science University, said to Targeted Oncology. CAR T-cell therapy in mantle cell lymphoma [MCL] has been approved this year as well, and that is also life changing for patients with MCL, where we've had very limited options in those who progressed on ibrutinib or ibrutinib and venetoclax. That's a life-changing option.

The autologous CD19-directed CAR T-cell therapy brexucabtagene autoleucel (formerly KTE-X19; Tecartus) received FDA approval for the treatment of adult patients with relapsed or refractory MCL in July 2020, based on findings from the phase 2 ZUMA-2 clinical trial (NCT02601313). According to Evens, the ORR among the first 60 patients treated and followed for at least 7 months was 93% (95% CI, 84%-98%), and the overall ORR was 85%. The 1-year PFS rate was 61%, and the estimated 1-year OS rate was 83%. Findings demonstrated cytokine release syndrome was observed in 91% of patients with 15% being grade 3 or higher in severity and none being fatal. Neurologic events, another notable side effect associated with CAR T-cell therapy, were observed in 63% with 31% being grade 3 and none being fatal.

Not every patient needs next-generation sequencing, but there are some cancer types where you can make a strong argument that is becoming almost mandatory, said Lewis. A great example from this also would be biliary tract cancers. With cholangiocarcinoma and gallbladder cancer, we know that they are underneath the umbrella groups of highly targetable mutations, whether it's IDH1 or FGFR2. Now, thankfully, it's not just an academic exercise to find the mutation because now we have a drug to pair with it, so another FDA approval I would call attention to was pemigatinib [Pemazyre] for FGFR2-driven cholangiocarcinoma because when I started, there was literally 1 drug for biliary tract cancer, gemcitabine, and now slightly over a decade later, there are a host of possibilities.

Pemigatinib received approval in April 2020, for the treatment of adult patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma who harbor an FGFR2 fusion or rearrangement, representing the first approved treatment for this indication. Findings from cohort A of the phase 3 FIGHT-202 study (NCT02924376) supported this approval, demonstrating an ORR of 36% and a median duration of response of 9.1 months in the multicenter, open-label single-arm study.

The combination of atezolizumab (Tecentriq) plus bevacizumab (Avastin) was approved as treatment of patients with unresectable or metastatic hepatocellular carcinoma (HCC) who have not had a prior systemic therapy, based on findings from the phase 3 IMbrave150 study (NCT03434379). This study demonstrated a reduced risk of death by 42% with the combination compared with sorafenib (Nexavar) alone (HR, 0.58; 95% CI, 0.42-0.79; P =.0006). Compared with standard of care sorafenib, this regimen is the first to markedly improve survival in the frontline setting for patients with HCC in several years.

In addition, the treatment landscape for patients withHCC was enriched with an approval in March 2020 of nivolumab (Opdivo) in combination with ipilimumab (Yervoy) in patients who have been previously treated with sorafenib. This approval allows for a new second-line option for patients with advanced HCC that has demonstrated promising improvements in OS, according to findings from the phase 1/2 CheckMate-040 study (NCT01658878). This study showed the longest duration of OS in the second-line setting for advanced HCC tested in clinical trials.

Patients with colorectal cancer (CRC) also saw a couple of new additions to the armamentarium, with 1 notable approval being the combination of encorafenib (Braftovi) and cetuximab (Erbitux) as treatment for patients with metastatic CRC with aBRAFV600E mutation, as detected by an FDA-approved test, after prior therapy. The combination received its approval from the FDA based on findings from the phase 3 BEACON CRC study (NCT02928224), which showed significant improvements in OS and a higher response rate compared with standard treatment. Shortly after this news, the FDA granted approval to the therascreen BRAF V600E Kit (therascreen BRAF V600E RGQ PCR Kit) as a companion diagnostic for this regimen.

In the field of GU cancers, the FDA granted several approvals in 2020, providing more treatment options and hope for physicians treating patients with various diseases. In particular, several approvals in prostate cancer stood out, as well as the first and only approval of an immunotherapy that has demonstrated significant overall survival (OS) benefit in the frontline setting in a phase 3 study of bladder cancer; the FDA granted approval to frontline maintenance avelumab (Bavencio) as treatment of patients with locally advanced or metastatic urothelial carcinoma who have not progressed with frontline platinum-based chemotherapy. Avelumab maintenance extended the OS by 50% compared with best supportive care in the phase 3 JAVELIN Bladder 100 study (NCT02603432).

Overall, in my opinion, this is practice-changing because right now instead of finishing chemotherapy frontline and waiting until progression happens, we can utilize avalumab based on this study as a switch maintenance frontline approach, Petros Grivas, MD, PhD, 1 of the principal investigators in the JAVELIN Bladder 100 trial, told Targeted Oncology.

The study demonstrated a 7.1-month improvement in the median OS with frontline avelumab maintenance and best supportive care versus best supportive care alone. The median OS was 21.4 months with avelumab (95% CI, 19.9-26.1) versus 14.3 months in the control arm (95% CI, 12.9-17.9), which was a statistically significant improvement with a 31% reduction in the risk of death in the overall population (HR, 0.69; 95% CI, 0.56-0.86; 2-sidedP= .001).

In prostate cancer, the approval of 2 PARP inhibitors excited the field. In May 2020, the FDA approved olaparib (Lynparza) for the treatment of patients with metastatic castration-resistant prostate cancer(mCRPC) who have deleterious or suspected deleterious germline or somatic homologous recombination repair (HRR) gene mutations and have progressed following prior therapy with a new hormonal agent. The approval of this PARP inhibitor was based on findings from the phase 3 PROfound clinical trial, which demonstrated a 66% reduction in the risk of disease progression or death with olaparib versus enzalutamide (Xtandi) or abiraterone acetate (Zytiga) in patients with BRCA1/2 or ATM mutations.

A few days prior to this approval, rucaparib (Rubraca) received FDA approval for adult patients with mCRPC who have a deleterious BRCA mutation (germline and/or somatic)-associated disease and have received prior androgen receptor-directed therapy and a taxane-based chemotherapy. The TRITON trials provided supportive data for this approval, exploring the use of rucaparib in patients with mCRPC and alterations in HRR-related genes.

Olaparib was approved in prostate cancer, and rucaparib also, so around summertime, there were 2 approvals issued by the FDA. The companion diagnostic was also approved for testing for the patients, said Maha Hussain, MD, who is the Genevieve E. Teuton Professor of Medicine in the Division of Hematology and Oncology, Department of Medicine, and deputy director at the Robert H. Lurie Comprehensive Cancer Center of the Northwestern University Feinberg School of Medicine, in an interview with Targeted Oncology. What I'm looking forward to is more approvals from the FDA in prostate cancer. I have to say in my career time, the last 10 years have been really tremendous in terms of the approvals from the FDA in the castration-resistant space. It's exciting times. We've been set back by COVID-19, but I do think that we are on a move forward and an upward ladder, so to speak, in terms of better research, and looking forward to much better times and 2021 and onwards.

The FoundationOneLiquid CDx approval was expanded as a companion diagnostic for olaparib in November 2020. The test was initially granted approval in August 2020 for the indication of patients with any solid tumor. This olaparib indication followed news from October 2020, indicating FoundationOne CDx as a companion diagnostic to identify patients who may receive benefit from rucaparib, alectinib (Alecensa), or alpelisib (Piqray), which are approved in different solid tumors. In the prostate cancer space, the companion diagnostic is used to identify BRCA1/2 and ATM genes in patients with mCRPC who are eligible for treatment with olaparib.

There is a lot of excitement about PARP inhibitors, and there are many PARP inhibitors right now that are undergoing evaluation in prostate cancer, which is exciting, said Hussain. The research in prostate cancer has really blossomed in an incredible way at multiple fronts, and so I do think we're seeing acceleration in terms of the research and its outcomes.

See more here:
Experts Reflect on Most Impactful FDA Moves of 2020 in Solid Tumors, Hematologic Malignancies - Targeted Oncology

Read More...

FDA Resumes eIND Approval for Severe-to-Critical COVID-19 Patients Use of Vyrologix (leronlimab) Following Full Enrollment in CytoDyn’s Phase 3 Trial…

Monday, December 28th, 2020

FDAs decision will enable CytoDyn to respond to ongoing requests for leronlimab until Phase 3 trial data is unblinded

VANCOUVER, Washington, Dec. 22, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn or the Company"), a late-stage biotechnology company developing Vyrologix (leronlimab-PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced today a treating physician has received authorization from the U.S. Food and Drug Administration (FDA) to administer leronlimab for a COVID-19 patient under emergency IND (eIND).

Nader Pourhassan, Ph.D., President and Chief Executive Officer of CytoDyn, commented, We are very thankful the FDA is allowing severe-to-critical COVID-19 patients access to Vyrologix (leronlimab) again under eIND while we await the unblinding of data from our recently completed Phase 3 registrational trial. We are receiving daily requests from families seeking our drug for a loved one with COVID-19. In recent months, leronlimab received more than 60 eIND authorizations from the FDA, and during the pendency of our COVID-19 trials, we deferred seeking authorizations for eINDs in order to accelerate the pace of enrollment. Now that enrollment has been completed, we are pleased to be able to assist once again and remain hopeful the upcoming results of our Phase 3 trial will enable leronlimab to be more readily available for severe-to-critical COVID-19 patients.

CytoDyns Phase 2b/3 trial to evaluate the efficacy and safety ofleronlimabfor patients with severe-to-critical COVID-19 indications is a two-arm, randomized, double blind, placebo controlled, adaptive design multicenter study. Patients are randomized to receive weekly doses of 700 mg leronlimab, or placebo. Leronlimab and placebo are administered via subcutaneous injection. The study has three phases: Screening Period, Treatment Period, and Follow-Up Period. The primary outcome measured in this study is: all-cause mortality at Day 28. Secondary outcomes measured are: (1) all-cause mortality at Day 14, (2) change in clinical status of subject at Day 14, (3) change in clinical status of subject at Day 28, and (4) change from baseline in Sequential Organ Failure Assessment (SOFA) score at Day 14.

About Coronavirus Disease 2019 CytoDyn completed its Phase 2 clinical trial (CD10) for COVID-19, a double-blinded, randomized clinical trial for mild-to-moderate patients in the U.S. which produced statistically significant results for NEWS2. CytoDyn completed enrollment of 390 patients in its Phase 2b/3 randomized clinical trial for the severe-to-critically ill COVID-19 population and expects to release results in mid-January 2021.

About Leronlimab (PRO 140) The FDA has granted a Fast Track designation to CytoDyn for two potential indications of leronlimab for critical illnesses. The first indication is a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer. Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including NASH.Leronlimab has completed nine clinical trials in over 800 people and met its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab could significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

In the setting of cancer, research has shown that CCR5 may play a role in tumor invasion, metastases, and tumor microenvironment control. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is, therefore, conducting a Phase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells.CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to support further the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD, blocking the CCR5 receptor from recognizing specific immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted orphan drug designation to leronlimab for the prevention of GvHD. Due to the lack of patients during the COVID-19 pandemic, the Company is closing down its Phase 2 trial for acute GvHD.

About CytoDyn CytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a critical role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as GvHD and NASH.

CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. The FDA met telephonically with Company key personnel and its clinical research organization and provided written responses to the Companys questions concerning its recent Biologics License Application (BLA) for this HIV combination therapy in order to expedite the resubmission of its BLA filing for this indication.

CytoDyn has completed a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication. If successful, it could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV. No drug-related serious site injection reactions reported in about 800 patients treated with leronlimab and no drug-related SAEs reported in patients treated with 700 mg dose of leronlimab. Moreover, a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients; some patients on leronlimab monotherapy have remained virally suppressed for more than six years.

CytoDyn is also conducting a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is at http://www.cytodyn.com.

Forward-Looking StatementsThis press release contains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as "believes," "hopes," "intends," "estimates," "expects," "projects," "plans," "anticipates" and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. Forward-looking statements specifically include statements about leronlimab, its ability to have positive health outcomes, the possible results of clinical trials, studies or other programs or ability to continue those programs, the ability to obtain regulatory approval for commercial sales, and the market for actual commercial sales. The Company's forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i) the sufficiency of the Company's cash position, (ii) the Company's ability to raise additional capital to fund its operations, (iii) the Company's ability to meet its debt obligations, if any, (iv) the Company's ability to enter into partnership or licensing arrangements with third parties, (v) the Company's ability to identify patients to enroll in its clinical trials in a timely fashion, (vi) the Company's ability to achieve approval of a marketable product, (vii) the design, implementation and conduct of the Company's clinical trials, (viii) the results of the Company's clinical trials, including the possibility of unfavorable clinical trial results, (ix) the market for, and marketability of, any product that is approved, (x) the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Company's products, (xi) regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii) general economic and business conditions, (xiii) changes in foreign, political, and social conditions, and (xiv) various other matters, many of which are beyond the Company's control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form 10-K, and any risk factors or cautionary statements included in any subsequent Form 10-Q or Form 8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CONTACTSInvestors: Michael MulhollandOffice: 360.980.8524, ext. 102mmulholland@cytodyn.com

Read more here:
FDA Resumes eIND Approval for Severe-to-Critical COVID-19 Patients Use of Vyrologix (leronlimab) Following Full Enrollment in CytoDyn's Phase 3 Trial...

Read More...

Magenta Therapeutics Announces Commencement of First Phase 2 Clinical Trial of MGTA-145 for Stem Cell Mobilization, Oral Presentation of MGTA-145…

Saturday, December 12th, 2020

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplant to more patients, today announced final clinical results from its earlier completed Phase 1 clinical trial as well as development updates for its MGTA-145 stem cell mobilization therapy, including commencement of enrollment in a Phase 2 clinical trial in multiple myeloma, and its plans for a Phase 2 clinical trial in allogeneic stem cell transplant for patients with acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL) and myelodysplastic syndrome (MDS). The company also previously announced a clinical collaboration with bluebird bio to evaluate MGTA-145 for mobilizing and collecting stem cells in adults and adolescents with sickle cell disease (SCD). Additional preclinical results were also presented at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, taking place virtually from December 5-8, 2020, on the Magenta conditioning platform, including MGTA-117 program, which is a targeted antibody-drug conjugate (ADC) to prepare patients for stem cell transplant.

MGTA-145 Advancement to Phase 2 Development in Blood Cancers

The company announced that enrollment has started and is ongoing in a Phase 2 clinical trial of MGTA-145, used in combination with plerixafor, to mobilize and collect stem cells for autologous stem cell transplantation of multiple myeloma patients at Stanford University. Magenta expects that this trial will provide patient-level data on stem cell mobilization and collection, characteristics of the mobilized graft and engraftment in patients with multiple myeloma.

Additionally, through a collaboration with the National Marrow Donor Program/Be The Match, a global leader in facilitating allogeneic hematopoietic stem cell transplantation, Magenta plans to initiate a Phase 2 clinical trial in early 2021 using MGTA-145 to mobilize and collect stem cells from allogeneic donors for transplant in patients with AML, ALL and MDS. Allogeneic stem cell transplant provides a potentially curative therapeutic option for patients with these diseases. This clinical trial will evaluate stem cell mobilization, collection, cell quality, engraftment and the potential for reduced Graft-versus-Host Disease (GvHD), which is of particular importance in the allogeneic transplant setting.

MGTA-145 in Sickle Cell Disease

Magenta Therapeutics recently announced an exclusive clinical collaboration with bluebird bio to evaluate the utility of MGTA-145, in combination with plerixafor, for the mobilization and collection of stem cells in adults and adolescents with SCD.

The data from this clinical trial could provide proof-of-concept for MGTA-145, in combination with plerixafor, as the preferred mobilization regimen for patients with SCD. bluebird bios experience with plerixafor as a mobilization agent in SCD aligns with Magentas combination therapy approach, utilizing MGTA-145 plus plerixafor with potential for safe, rapid and reliable mobilization of sufficient quantities of high-quality stem cells to improve outcomes associated with stem cell transplantation.

MGTA-145 Presentations at ASH

Magenta presented final clinical data from its MGTA-145 stem cell mobilization Phase 1 clinical trial in healthy volunteers at the ASH Annual Meeting. All primary and secondary endpoints were met in the study completed earlier this year.

The results demonstrate that a single dose of MGTA-145, in combination with plerixafor, rapidly and reliably mobilized high numbers of stem cells in a single day without the need for G-CSF for potential use in diseases that can benefit from autologous and/or allogeneic stem cell transplantation. The additional data also offer further confirmation that MGTA-145, in combination with plerixafor, was well tolerated and provides a rapid and reliable method to obtain large numbers of hematopoietic stem cells. Transplant of these cells in preclinical models resulted in enhanced, durable engraftment, in addition to highly immunosuppressive properties, leading to reduced GvHD.

Results from this study provide a robust dataset and proof of concept that MGTA-145, in combination with plerixafor, provides rapid and robust mobilization of stem cells and that these cells have better engraftment potential, are able to be gene modified and engraft and reduce GvHD in preclinical models compared to cells mobilized with other available agents. The data reinforce the availability of compelling opportunities for development in both the autologous and allogeneic transplant settings, said John Davis Jr., M.D., M.P.H., M.S., Head of Research & Development and Chief Medical Officer, Magenta Therapeutics.

The data were presented by Steven M. Devine, MD, Chief Medical Officer of the National Marrow Donor Program/Be The Match and Associate Scientific Director of the CIBMTR (Center for International Blood and Marrow Transplant Research).

Conditioning Program (MGTA-117 and CD45-ADC) Presentations at ASH

Magenta also provided updates on its conditioning platform at the ASH Annual Meeting, including MGTA-117 and CD45-ADC programs. Preclinical data from a study of MGTA-117 demonstrate that it is an effective, potent conditioning agent for transplant with anti-leukemic activity, significantly decreasing tumor burdens, leading to delayed tumor growth and increased median survival rates in animal models of AML. Ongoing GLP toxicology and GMP manufacturing progress continue to be supportive of advancing MGTA-117 towards an IND filing in AML and MDS.

Additionally, preclinical data from a study of Magentas CD45-ADC, a CD45-targeted conditioning agent designed to remove the cells that cause autoimmune diseases to enable curative immune reset, demonstrated the ability to achieve successful outcomes as a single agent in the most challenging disease model through fully mismatched allogeneic hematopoietic stem cell transplant, where only radiation or combinations of toxic chemotherapies are available, potentially providing patients the option of a reduced toxicity conditioning regimen. The company continues to evaluate this program preclinically.

About MGTA-145

MGTA-145 is being developed in combination with plerixafor to harness complementary chemokine mechanisms to mobilize hematopoietic stem cells for collection and transplantation. This new combination has the potential to be the preferred mobilization regimen for rapid and reliable mobilization and collection of hematopoietic stem cells to improve outcomes in autologous and allogeneic stem cell transplantation, which can rebuild a healthy immune system for patients with blood cancers, genetic diseases and autoimmune disorders.

MGTA-145 has the potential to replace the current standard of care for patients and allogeneic donors who currently rely on the use of granulocyte-colony stimulating factor (G-CSF) alone or in combination with plerixafor, which can take up to five days or longer to mobilize sufficient numbers of stem cells, often resulting in significant bone pain and other side effects.

About Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of immune system reset through stem cell transplant to more patients with blood cancer, genetic diseases and autoimmune diseases. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant world to revolutionize immune reset for more patients.

Magenta is based in Cambridge, Mass. For more information, please visit http://www.magentatx.com.

Follow Magenta on Twitter: @magentatx.

Forward-Looking Statement

This press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, will, could, should, expects, intends, plans, anticipates, believes, estimates, predicts, projects, seeks, endeavor, potential, continue or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation risks set forth under the caption Risk Factors in Magentas Annual Report on Form 10-K filed on March 3, 2020, as updated by Magentas most recent Quarterly Report on Form 10-Q and its other filings with the Securities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

See the rest here:
Magenta Therapeutics Announces Commencement of First Phase 2 Clinical Trial of MGTA-145 for Stem Cell Mobilization, Oral Presentation of MGTA-145...

Read More...

Daratumumab Regimen Shows Promise in Transplant-Eligible Patients With Newly Diagnosed Myeloma – Targeted Oncology

Saturday, December 12th, 2020

The combination of daratumumab (Darzalex) with lenalidomide, bortezomib, and dexamethasone (D-RVd) followed by maintenance therapy with daratumumab and lenalidomide (D-R) improved response rates and depth of response rates with statistically significance as treatment of patients with transplant-eligible newly diagnosed multiple myeloma compared with RVd followed by lenalidomide maintenance, according to findings presented at the 2020 American Society of Hematology (ASH) Annual Meeting.

The updated analysis of the GRIFFEN study showcased that D-R maintenance therapy improved the depth of response for patients while maintaining remissions.

As noted, these results support D-RVd as a potential new standard of care for transplant eligible newly diagnosed multiple myeloma patients, Jonathan L. Kaufman, MD, Winship Cancer Institute, Emory University, said in his presentation of the data.

The researchers found in earlier data cuts that the responses from patients to these treatments deepened over time, with response rates and depths greater for patients in the D-RVd arm at all points in time. At the end of consolidation, the stringent complete response (sCR) rate was 42.4% in the D-RVd arm (n = 104) compared to 32% in the RVd arm alone (n = 103; P < .0014).

For the data regarding the 12 months of maintenance cutoff, the sCR rate was 63.6% versus 47,4% for the D-RVd arm and RVd alone arm, respectively.

Researchers also examined minimal residual disease (MRD)-negativity after 12 months of maintenance therapy, finding that, in an intent to treat manner, the total MRD-negative rate in the D-RVd arm was 62.5% compared to 27.2% for the RVd alone arm (P < .0001). Solely examining the MRD evaluable group of patients, the MRD-negativity rate was 78.3% for the D-RVd arm (n = 83) versus 39.4% for the RVd alone arm (n = 71; P < .0001).

More, durability of MRD-negativity at both 6 and 12 months was significantly higher in the D-RVd arm versus the RVd alone arm. For 6 months, the MRD-negative rate was 37.5% in the D-RVd arm, compared to only 7.8% in the RVd alone arm (P < .0001). At 12 months, the rates for the D-RVd and RVd alone arms were 28.8% and 2.9%, respectively (P < .0001). The data showed a significant improvement in the rates of sustained MRD-negativity for the D-RVd arm versus RVd alone.

From an MRD-negativity standpoint, there is not a subgroup of patients that does not benefit from adding DARA to RVd treatment.

D-RVd is now considered a treatment that is appropriate for (the National Comprehensive Cancer Network). I know that we have made this our standard of care at our center, explained Kaufman It is a regimen that is well tolerated, its effective, it doesnt negatively impact the ability to collect stem cells or patients to engraft after stem cell transplant. The goal of our induction therapy is to induce MRD-negatively and what we see here is that this regimen is very good at reducing MRD-negativity.

The primary end point of the study was sCR, with secondary end points including MRD-negativity rates and overall response rates (ORR).

The researchers also explained that there is currently no statistical difference between progression-free or overall survival in both arms, with a median follow-up of 27.4 months.

While there was a noticeable increase in rates of neutropenia and upper respiratory infections for the D-RVd group compared to RVd alone, these increased rates did not lead to higher rates of discontinuation for patients. More, similar rates of any grade and grade3/4 infections were observed for D-RVd versus RVd.

Looking ahead, there is an ongoing phase 3 PERSEUS study that is evaluating subcutaneous D-RVd in the transplant-eligible population of patients with newly diagnosed multiple myeloma.

Reference

Kaufman JL, Laubach J, Sborov DW, et al. Daratumumab (DARA) Plus Lenalidomide, Bortezomib, and Dexamethasone (RVd) in Patients with Transplant-Eligible Newly Diagnosed Multiple Myeloma (NDMM): Updated Analysis of Griffin after 12 Months of Maintenance Therapy. Presented at: 2020 ASH Annual Meeting and Exposition; December 5-8, 2020; Virtual. Abstract 549.

Originally posted here:
Daratumumab Regimen Shows Promise in Transplant-Eligible Patients With Newly Diagnosed Myeloma - Targeted Oncology

Read More...

HSCT Found Potentially Curative for Some T-Cell Lymphoma Patients – Cancer Therapy Advisor

Saturday, December 12th, 2020

Allogeneic hematopoietic stem cell transplant (allo-HSCT) may be a curative option for some patients with relapsed/refractory T-cell lymphoma (TCL), according to results of a retrospective study presented at the virtual 62nd American Society of Hematology (ASH) Annual Meeting and Exposition.

Outcomes from previous registry studies suggest that only about one-third of patients with relapsed TCL are disease-free 3 years after allo-HSCT, including a prior study by this same group of researchers, which found the 2-year PFS and OS to be 48.9% and 61.7%, respectively.

The group conducted the current study to better understand the dynamics of allogeneic transplant in T-cell lymphoma and with longer follow-up, Neha Mehta-Shah, MD, of the Washington University School of Medicine in St. Louis, Missouri, and presenter of the study, said.

The retrospective study included 508 consecutive patients with TCL who underwent allo-HSCT between 2000 and 2019 across 12 academic institutions. The median age of patients in the cohort was 51 years, and at the time of transplant, 54.4% were in complete remission (CR), 37.2% were in partial remission (PR), 5% had stable disease, and 3.2% had progressive disease. There were 15.5% of patients who had undergone prior autologous transplant. The conditioning regimens used prior to transplant were myeloablative in 35.4%, reduced intensity/nonmyeloablative in 63.6%, and unknown in less than 1% of patients.

After allo-HSCT, the 2- and 5-year progression-free survival (PFS) rates were 45.8% and 39.4%, respectively.

The time from relapse to death post allo-HSCT was a median 10.2 months. The 2- and 5-year overall survival (OS) was 59.1% and 50.8%, respectively. Among the 261 deaths that were recorded, 31% were transplant-related, 26.4% were due to progressive disease, and 42.5% were nonrelapse-related and/or unknown.

PFS was similar among patients with the angioimmunoblastic, not otherwise specified, and ALK-positive or -negative anaplastic large cell subtypes. Although PFS outcomes were worse for patients with cutaneous TCL (P =.0008), OS outcomes were not significantly different from other TCL subtypes (44% for cutaneous TCL vs 53.1% for others; P =.4573).

CR at the time of transplant was associated with longer median PFS and OS, with progressive disease associated with the shortest PFS and OS.

Dr Mehta-Shah concluded that in the largest series of allogeneic transplant to date, allogeneic transplant for patients with TCL can lead to durable remissions in patients with otherwise poor outcomes.

Disclosures: Some of the presenters disclosed financial relationships with the pharmaceutical industry and/or the medical device industry. For a full list of disclosures, please refer to the presentation abstract.

Read more of Cancer Therapy Advisors coverage of the ASH 2020 meeting by visiting the conference page.

Reference

Mehta-Shah N, Kommalapati A, Teja S, et al. Successful treatment of mature T-cell lymphoma with allogeneic stem cell transplantation: the largest multicenter retrospective analysis. Presented at: 62nd American Society of Hematology (ASH) Annual Meeting and Exposition; virtual; December 5-9, 2020. Abstract 41.

Read more:
HSCT Found Potentially Curative for Some T-Cell Lymphoma Patients - Cancer Therapy Advisor

Read More...

Researchers Trace the Origin of Blood Cancer to Early Childhood, Decades before Diagnosis – Yahoo Finance

Saturday, December 12th, 2020

Researchers Trace the Origin of Blood Cancer to Early Childhood, Decades before Diagnosis

PR Newswire

WASHINGTON, Dec. 8, 2020

Study in ASH Late-Breaking Abstracts session may pinpoint potential cancer origins and present opportunities for earlier detection efforts

WASHINGTON, Dec. 8, 2020 /PRNewswire/ -- Genetic mutations linked with cancer can occur during childhood or even before birth and proliferate in the body for many years before causing cancer symptoms, according to a new study. The study, which traced the genetic origins of a blood cancer in 10 individuals, suggests there may be untapped opportunities to detect cancer warning signs much earlier and potentially intervene to prevent or slow cancer development.

"Our preliminary findings show these cancer driver mutations were often acquired in childhood, many decades before the cancer diagnosis," said senior study author Jyoti Nangalia, MD, of the Wellcome Sanger Institute and University of Cambridge. "Our results finally answer the common question posed by patients, 'How long has this cancer been growing?' as we were able to study how these particular cancers developed over the entire lifetime of individual patients."

The researchers analyzed bone marrow and blood samples from 10 people with Philadelphia-negative myeloproliferative neoplasms, a type of cancer that causes stem cells in the bone marrow to produce too many blood cells. In the majority of patients, this cancer is driven by a genetic mutation called JAK2V617F. By assessing JAK2V617F, other cancer-linked mutations and hundreds of thousands of other mutations that a person naturally acquires throughout life, the researchers were able to trace the ancestry of different blood cells and estimate the time at which each patient acquired JAK2V617F and other important mutations.

They determined that, in these 10 patients, the first cancer-linked mutations emerged as early as a few weeks after the start of life and up to the first decade of childhood despite clinical disease presenting many decades later in life.

Story continues

"We were not expecting this," said Dr. Nangalia. "In fact, in one patient, the JAK2 mutation was acquired more than 50 years before their diagnosis."

While it is often assumed that most cancers are diagnosed within a few years of their emergence, the findings point to a more gradual, lifelong process in which a single cell acquires a cancer-linked mutation early in life and then slowly grows over decades, ultimately leading to cancer.

"Some of these cancer-linked mutations are found in healthy individuals as we get older, suggesting that aging causes them," said Dr. Nangalia. "However, aging per se doesn't drive such growth it simply takes a long time for the clones to grow." Sometimes, the growing clones pick up additional cancer-linked mutations along the way, accelerating their growth, researchers found.

"For these patients, we calculated how many of these cancer clones would have been present in the past, and our results suggest that these clones may have been detectable up to 10 to 40 years before diagnosis," said Dr. Nangalia. "In addition to detecting the mutations, the rate at which the mutated clones grew was also very important in determining whether, and when, cancer develops." The findings suggest that genetic testing could help identify people at risk for cancer much earlier than current methods allow, according to researchers.

The next steps would be to understand the factors that influence the different rates of cancer growth and determine whether there could be ways to intervene and slow the growth of cells with cancer-linked mutations. The researchers say their method for pinpointing the origin of this blood cancer could also be applied to other mutations and other blood cancers. "Understanding the timelines of development of different cancers is critical for efforts aimed at early cancer detection and prevention," said Dr. Nangalia.

Jyoti Nangalia, MBBChir, Wellcome Sanger Institute and University of Cambridge, will present this study during the Late-Breaking Abstracts session on Tuesday, December 8 at 7:00 a.m. Pacific time on the ASH annual meeting virtual platform.

For the complete annual meeting program and abstracts, visit http://www.hematology.org/annual-meeting. Follow ASH and #ASH20 on Twitter, Instagram, LinkedIn, and Facebook for the most up-to-date information about the 2020 ASH Annual Meeting.

The American Society of Hematology (ASH) (www.hematology.org) is the world's largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment, and prevention of disorders affecting the blood. For more than 60 years, the Society has led the development of hematology as a discipline by promoting research, patient care, education, training, and advocacy in hematology. ASH publishes Blood (www.bloodjournal.org), the most cited peer-reviewed publication in the field, and Blood Advances (www.bloodadvances.org), an online, peer-reviewed open-access journal.

View original content to download multimedia:http://www.prnewswire.com/news-releases/researchers-trace-the-origin-of-blood-cancer-to-early-childhood-decades-before-diagnosis-301188621.html

SOURCE American Society of Hematology

Go here to read the rest:
Researchers Trace the Origin of Blood Cancer to Early Childhood, Decades before Diagnosis - Yahoo Finance

Read More...

ALLO-715, Off-the-Shelf CAR T-Cell Therapy, Produces Early Promise in Multiple Myeloma – Cancer Network

Saturday, December 12th, 2020

Treatment with an off-the-shelf CAR T-cell therapy that targets B-cell maturation antigen (BCMA), ALLO-715, elicited responses in heavily pretreated patients with relapsed/refractory multiple myeloma in early findings from a first-in-human study presented at the 2020 ASH Meeting.1

The therapy generated responses in 6 of 10 patients (60%), including a very good partial-plus response (VGPR+) in 4 patients (40%), who were treated with ALLO-715 at a dose of 320 x 106 CAR cells plus a lymphodepleting regimen that included ALLO-647, an anti-CD52 monoclonal antibody, during the ongoing phase 1 UNIVERSAL study (NCT04093596).1

The findings mark the first results for an allogeneic CAR therapy directed at BCMA, said lead study author Sham Mailankody, MBBS, a medical oncologist and investigator in the Cellular Therapeutics Center at Memorial Sloan Kettering Cancer Center in New York, New York. BCMA, which is highly expressed on plasma and multiple myeloma cells, has sparked intensive research interest.2

These results demonstrate the feasibility and safety of an off-the-shelf CAR T cell therapy for multiple myeloma. In this first report of an allogeneic BCMA CAR T-cell therapy, we show that nearly 90% of patients were treated within 5 days of enrollment and without needing any bridging therapy, Mailankody said.

Allogeneic CAR therapy offers the potential for scalable manufacturing for on-demand treatment with shorter waiting times, which would overcome some of the logistical challenges posed by autologous CAR therapy, Mailankody said. The T cells needed for ALLO-715 are harvested from healthy donors and genetically engineered to express CARs aimed at specific cancer targets, according to Allogene Therapeutics, the company developing the therapy.3

ALLO-715 includes a human-derived single-chain variable fragment anti-BCMA cell with a 4-1BB costimulatory domain. Mailankody said the 2 key attributes of the construct are a knockout of CD52, which allows for selective lymphodepletion with ALLO-647 to prevent graft rejection without affecting the CAR T cells, and a knockout of the TRAC gene, which also minimizes the risk of graft-versus-host disease (GVHD).1

The UNIVERSAL study, which is being conducted at 11 cancer centers in the United States, is recruiting patients with multiple myeloma who have received 3 or more prior therapies, including an immunomodulatory drug, a proteasome inhibitor, and an anti-CD38 agent, and are refractory to their last treatment. Participants must have an ECOG performance status score of 0 or 1.

The dose escalation portion of the study is testing ALLO-715 as a single infusion across 4 doses: 40, 160, 320, or 480 x 106 CARs. Lymphodepletion regimens consist of fludarabine (F; 30 mg/m2/day) plus cyclophosphamide (C; 300 mg/m2/day) given on 3 days with ALLO-647 (A; 13-30 mg x 3 days; FCA) or cyclophosphamide plus ALLO-647 (CA).

Among the 35 patients enrolled at the time of the presentation, 4 became ineligible because of organ failure due to rapidly progressing disease. Of 31 patients in the safety population, the median age was 65 years (range, 46-76). Nearly half of the patients (48%) have high-risk cytogenetics and 23% had extramedullary disease. The efficacy population at data cutoff on October 30, 2020, comprised 26 patients across the 4 dosing levels, with a median follow-up of 3.2 months.1

The overall response rate (ORR) varied across dosing cohorts and lymphodepleting regimens. No responses were observed among 3 patients each who received CARs at 40 x 106 with FCA or 160 x 106 with CA, both with low-dose ALLO-647. The ORRs were 50% in 4 patients who received CARs at 160 x 106 with lowALLO-647 FCA; 33% in 3 at 480 x 106 with lowALLO-647 FCA; and 67% in 3 at 320 x 106 with lowALLO-647 CA.

The most robust responses were seen among those who received ALLO-647 at 320 x106. For this cohort, the ORR was 60% among 10 patients, including 3 of 6 who received CARs with lowALLO-647 FCA and 3 of 4 who had the therapy with highALLO-647 FCA. Overall, 6 patients had a VGPR+, defined as stringent complete response, complete response, or VGPR. These included 1 at 160, 4 at 320, and 1 at 480 10 x 106 CARs. Of the VGPR+ patients, 5 were negative for measurable residual disease. Additionally, 6 of 9 patients treated at the 320 or 480 x 106 dose levels remain in response.

Mailankody highlighted the experience of 1 participant, a 71-year-old man whose myeloma had progressed after undergoing 9 prior lines of therapy including autologous stem cell transplant and an experimental BCMA-targeted therapy. The patient received a conditioning regimen of FCA with low-dose ALLO-647 and ALLO-715 at 320 x 106. He reached a VGPR on day 14 that deepened to a stringent complete response by day 28 that remains in effect at 6 months, while experiencing grade 1 cytokine release syndrome (CRS).

The patient is clinically doing very well and is back at work, Mailankody said.

Among 31 patients in the safety population, most adverse effects were of grade 1 or 2 severity. These included CRS in 14 patients (45%) and infusion-related reactions to ALLO-647 in 7 patients (23%). The use of drugs to manage CRS also was low, at 19% for tocilizumab and 10% for steroids.

All-grade infections were reported in 13 patients (42%), including grade 3 events in 4 (13%). One patient (3%) died from a presumed fungal pneumonia related to progressive disease and the CA conditioning regimen but unrelated to ALLO-715. There were no instances of neurotoxicity or GVHD.

Notably, the fact that we did not see any GVHD is encouraging for an off-the-shelf allogeneic product, Mailankody said.

In response to a question from a conference attendee, Mailankody said it is too soon to compare efficacy levels seen with this allogeneic CAR therapy with those observed with investigational autologous CARs, which have been under study for several years.

Moving forward, investigators are continuing to evaluate dosing levels for ALLO-715. UNIVERSAL is enrolling patients to the 480 x 106 cohort, Mailankody said, adding that the appropriate dose likely would land between 320 and 480 x 106.

References

1. Mailankody S, Matous JV, Liedtke M, et al. Universal: an allogeneic first-in-human study of the anti-Bcma ALLO-715 and the Anti-CD52 ALLO-647 in relapsed/refractory multiple myeloma. Presented at: 2020 American Society of Hematology Annual Meeting and Exposition. December 5-8, 2020; Virtual. Abstract 129. Accessed December 5, 2020. https://ash.confex.com/ash/2020/webprogram/Paper140641.html

2. Cho SF, Anderson KC, Tai YT. Targeting B cell maturation antigen (BCMA) in multiple myeloma: potential uses of BCMA-based immunotherapy. Front Immunol. 2019;9:1821. doi:10.3389/fimmu.2018.01821

3. AlloCAR T Therapy. Allogene Therapeutics. Accessed December 5, 2020. https://www.allogene.com/allocar-t-therapy

Read more from the original source:
ALLO-715, Off-the-Shelf CAR T-Cell Therapy, Produces Early Promise in Multiple Myeloma - Cancer Network

Read More...

BeiGene Announces the Approval in China of BLINCYTO (Blinatumomab) for Injection for Adult Patients with Relapsed or Refractory B-Cell Precursor Acute…

Saturday, December 12th, 2020

BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)--BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biotechnology company focused on developing and commercializing innovative medicines worldwide, today announced that the China National Medical Products Administration (NMPA) has approved BLINCYTO (blinatumomab) for injection for the treatment of adult patients with relapsed or refractory (R/R) B-cell precursor acute lymphoblastic leukemia (ALL). The biologics license application (BLA) had been submitted by Amgen and received priority review by the Center for Drug Evaluation (CDE) of the NMPA. Developed by Amgen and licensed to BeiGene in China under a strategic collaboration commenced earlier this year, this is the first approval for BLINCYTO in China and BeiGenes first product licensed from Amgen to be newly approved. With this approval, BLINCYTO has become the first bispecific immunotherapy approved in China.

This approval of BLINCYTO provides us with an opportunity to offer adult patients in China with relapsed or refractory B-cell precursor ALL the first approved immunotherapy treatment for their disease. BLINCYTO is the first immunotherapy to demonstrate superior overall survival versus chemotherapy, more than doubling patients chances for survival, when used in first salvage R/R ALL in studies outside of China, commented Xiaobin Wu, Ph.D., General Manager of China and President of BeiGene. We are working to ensure BLINCYTO is available to patients in China as soon as possible. Our commercial organization of more than 1,500 people in China is excited to add BLINCYTO to our product portfolio, which now includes six approved cancer treatments.

The approval of BLINCYTO was based on results from the Phase 3 trial (NCT03476239) in China evaluating the efficacy and safety of BLINCYTO in adult patients with Philadelphia-negative R/R B-cell precursor ALL. Results of the interim analysis of 67 patients showed that the efficacy results in Chinese subjects were generally consistent with those in the global and Japan studies in subjects with Philadelphia-negative R/R ALL. The complete response/complete response with partial recovery of blood cells (CR/CRh) rate within two cycles of BLINCYTO treatment (the primary endpoint) was 47.8% (32 of 67 subjects; 95% CI: 35.4, 60.3). The median overall survival time was 9.6 months (95% CI: 6.4, not estimable). The safety profile observed for Chinese subjects in this study was consistent with that observed in the global studies evaluating BLINCYTO in R/R ALL. No new safety risks were identified based on these interim analyses of adverse events in Chinese subjects.

Our collaboration with BeiGene is advancing Amgens oncology pipeline for patients with significant unmet medical needs. We are confident the approval of BLINCYTO in China has the potential to make a meaningful difference to adult patients with R/R B-cell precursor acute lymphoblastic leukemia, said My Linh Kha, Vice President & General Manager, Amgen Japan Asia-Pacific (JAPAC). We are deeply committed to continuing to bring therapeutic options to treat debilitating cancers for patients in China, while also actively supporting the Governments focus on healthy aging through innovative products and initiatives designed to prevent chronic diseases, such as cardiovascular disease and fragility fracture.

About Acute Lymphoblastic Leukemia (ALL)

Acute lymphoblastic leukemia (ALL), also known as acute lymphocytic leukemia, is a rapidly progressing cancer of the blood and bone marrow that occurs in both adults and children1. ALL accounts for approximately 20% of all adult leukemia, and in China there were an estimated 82,607 new cases of leukemia in 20182,3. In children, the relapse rate of ALL is nearly 10%, while in adults the relapse rate is closer to 50%4.

About BLINCYTO (blinatumomab)

BLINCYTO is a bispecific CD19-directed CD3 T cell engager (BiTE) immuno-oncology molecule that binds specifically to CD19 expressed on the surface of cells of B-lineage origin and CD3 expressed on the surface of T cells.

BiTE molecules are a type of immuno-oncology therapy being investigated for fighting cancer by helping the body's immune system to detect and target malignant cells. The modified molecules are designed to engage two different targets simultaneously, thereby juxtaposing T cells (a type of white blood cell capable of killing other cells perceived as threats) to cancer cells. BiTE immuno-oncology molecules help place the T cells within reach of the targeted cell, with the intent of allowing T cells to inject toxins and trigger the cancer cell to die (apoptosis). BiTE immuno-oncology therapies are currently being investigated for their potential to treat a wide variety of cancers.

BLINCYTO was granted breakthrough therapy and priority review designations by the U.S. Food and Drug Administration and is approved in the U.S. for the treatment of:

In the European Union (EU), BLINCYTO is indicated as monotherapy for the treatment of:

In China, BLINCYTO is indicated for the treatment of adult patients with relapsed or refractory B-cell precursor ALL.

Important U.S. Safety Information

WARNING: CYTOKINE RELEASE SYNDROME and NEUROLOGICAL TOXICITIES

Contraindications

BLINCYTO is contraindicated in patients with a known hypersensitivity to blinatumomab or to any component of the product formulation.

Warnings and Precautions

Adverse Reactions

Dosage and Administration Guidelines

Please see full Prescribing Information and medication guide for BLINCYTO at http://www.BLINCYTO.com.

About BeiGene

BeiGene is a global, commercial-stage biotechnology company focused on discovering, developing, manufacturing, and commercializing innovative medicines to improve treatment outcomes and access for patients worldwide. Our 4,700+ employees in China, the United States, Australia, Europe, and elsewhere are committed to expediting the development of a diverse pipeline of novel therapeutics. We currently market two internally discovered oncology products: BTK inhibitor BRUKINSA (zanubrutinib) in the United States and China, and anti-PD-1 antibody tislelizumab in China. We also market or plan to market in China additional oncology products licensed from Amgen Inc., Celgene Logistics Srl, a Bristol Myers Squibb (BMS) company, and EUSA Pharma. To learn more about BeiGene, please visit http://www.beigene.com and follow us on Twitter at @BeiGeneUSA.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding the commercialization and potential benefits of BLINCYTO; and BeiGenes plans and expectations for the commercialization of its and Amgens other oncology products and pipeline assets. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeiGene's ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; BeiGene's ability to achieve commercial success for its marketed products and drug candidates, if approved; BeiGene's ability to obtain and maintain protection of intellectual property for its technology and drugs; BeiGene's reliance on third parties to conduct drug development, manufacturing and other services; BeiGenes limited operating history and BeiGene's ability to obtain additional funding for operations and to complete the development and commercialization of its drug candidates; the impact of the COVID-19 pandemic on the Companys clinical development, commercial and other operations, as well as those risks more fully discussed in the section entitled Risk Factors in BeiGenes most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in BeiGene's subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeiGene undertakes no duty to update such information unless required by law.

BLINCYTO and BiTE are registered trademarks of Amgen Inc.

1 Mayo Clinic. Acute lymphocytic leukemia. https://www.mayoclinic.org/diseases-conditions/acute-lymphocytic-leukemia/symptoms-causes/syc-20369077

2 Baljevic M, Jabbour E, O'Brien S, Kantarjian HM (2016). "Acute Lymphoblastic Leukemia".

3 Global Cancer Observatory. https://gco.iarc.fr/today/data/factsheets/populations/160-china-fact-sheets.pdf

4 Leukaemia Care. Relapse in Acute Lymphoblastic Leukaemia (ALL). https://media.leukaemiacare.org.uk/wp-content/uploads/Relapse-in-Acute-Lymphoblastic-Leukaemia-ALL-Web-Version.pdf

Read this article:
BeiGene Announces the Approval in China of BLINCYTO (Blinatumomab) for Injection for Adult Patients with Relapsed or Refractory B-Cell Precursor Acute...

Read More...

Flintshire youngster goes the extra mile to raise funds for Lymphoma Action | The Leader – LeaderLive

Saturday, December 12th, 2020

A FLINTSHIRE superhero is raising hundreds of pounds for a cause close to her heart in a bid to 'turn a negative into something positive'.

In 2019, Lauren Claire Pringle was diagnosed with Non-Hodgkins Lymphoma and began a course of gruelling treatment.

However, since finding out it has not gone completely, Lauren must have more chemotherapy and eventually a stem cell transplant.

Daughter Lily Pringle-Purcell decided to play her part and raise funds for a charity that has helped their family, and others going through similar.

The seven-year-old set out a target to raise 1,000 for Lymphoma Action and after shaving her mother's hair off and completing a five-mile walk, she has since raised over 800 of her target.

But the fundraising will not stop there.

Mum Lauren told the Leader: "She's done really well, I'm so proud of her.

"She's a fabulous little girl, really caring and always thinking about other people."

Anyone wishing to donate can do so online at https://www.justgiving.com/fundraising/laurenandlily.

The fundraising page reads: "My daughter has been raising money for Lymphoma Action. She has raised 720 so far on her Just Giving page. She shaved my head to raise money and also we did sponsored walk today of 5 miles to raise more money. She wanted to do this after my diagnosis last year of lymphoma and ongoing treatment.

"My name is Lily and I am 7 and in September 2019 my mum Lauren was diagnosed with Non Hodgkins Lymphoma. She had to have 6 cycles of chemotherapy and 12 sessions of radiotherapy. Recently we found out it hasnt all gone and she needs to have more chemotherapy and eventually a stem cell transplant. This has been really hard on us as a family and we want to try and turn a negative situation into something positive and raise as much money and awareness as we can to hopefully find better treatments and help other families going through the same thing.

"I want to do a sponsored walk and a sponsored bike ride and I am thinking of other things I can do to try and raise as much money as I can.

"If anyone has any ideas then please let me know! Thankyou all so much! Lots of Love Lily and Lauren."

Read the original:
Flintshire youngster goes the extra mile to raise funds for Lymphoma Action | The Leader - LeaderLive

Read More...

Meat-Tech Agrees to Acquire Cultured Fat Pioneer ‘Peace of Meat’ – PRNewswire

Saturday, December 12th, 2020

NESSZIONA,Israel, Dec. 8, 2020 /PRNewswire/ -- Meat-Tech 3D Ltd. (TASE: MEAT), today announced that it has signed an agreement to acquire 100% of the share capital of Peace of Meat PV, a pioneering Belgian producer of cultured avian products, for EUR 15 million in a combination of cash and Meat-Tech ordinary shares. The Company believes that it will be able to leverage Peace of Meat's technologies, including through novel hybrid food products, to expedite market entry while Meat-Tech develops an industrial process for cultivating and producing real meat using 3D bioprinting technology, without harming animals. The acquisition is expected to close in the coming weeks, subject to customary closing conditions.

Peace of Meat has developed a proprietary, stem-cell-based bioreactor technology for cultivating animal fats from chicken and ducks, without harming animals. It has conducted a number of taste tests, demonstrating the potential that its cultured fat has to enhance the taste of plant-based protein products. The technology's first expected application is in hybrid food products, combining plant-based protein with cultured animal fat, designed to provide meat analogues with qualities of "meatiness" (taste and texture) closer to that of conventional meat products. Meat-Tech estimates that the first hybrid products based on Peace of Meat technology could hit the market as early as 2022.

Pursuant to the acquisition agreement, Meat-Tech will pay half of the consideration immediately, with the payment of the balance subject to Peace of Meat complying with preset technological milestones over a period of two years, that were designed to scale up cultured fat production capabilities in preparation for market entry. To that end, it was agreed that Peace of Meat's management will continue in place to lead the development process.

This acquisition is consistent with Meat-Tech's growth strategy, aiming to streamline development processes and expand the Company's product range to penetrate cultured meat technology markets as quickly as possible. Meat-Tech is working to create synergy and added value for food manufacturers in the advanced production of cultured meat, while sustaining animal welfare and meeting the growing global demand for meat.

Sharon Fima, Meat-Tech's CEO: "Meat-Tech's novel technology for producing meat using 3D printing is gaining increasing international recognition. Boosted by our acquisition strategy, we believe we can turn Meat-Tech into a leading global center and home for innovative and groundbreaking cell-based food solutions that are both healthy and environmentally friendly. The combination of Peace of Meat's human capital and technology make this acquisition a significant step in that direction. I am pleased that both management teams share a common vision and strategy, and can join forces to advance the development of cultured food products with the potential to create real alternatives in the global meat market."

David Brandes and Dirk von Heinrichshorst, Co-Founders of Peace of Meat:"In an industry that is working towards a kinder, more sustainable planet, joining forces makes us stronger together. Peace of Meat has developed a powerful system for upscaled cultured biomass production and together with Meat-Tech we intend to accelerate product development toward commercialization.

"While Peace of Meat's core activity remains focused on the production of tasty, cultured fat as a B2B ingredient for meat alternatives, we see tremendous opportunity in jointly building a leading food-tech enterprise with Meat-Tech, based on a cellular platform.

"As entrepreneurs, we are excited about this acquisition as it poses a novel way of building and growing a company while significantly increasing the prospects of launching our product into the market."

About Peace of Meat:

Peace of Meat was established in Belgium in 2019 and is developing cultured chicken fat directly from animal cells without the need to grow or kill animals. The company believes that its innovative technology has the potential to support an industrial process for the production of cultured chicken fat. Peace Of Meat has entered into a number of scientific and commercial collaborations, in the process of positioning itself as a future B2B provider, with the potential to cover the entire value chain and to accelerate research and production processes in the industry, and has conducted taste tests for hybrid products it has developed.

About Meat-Tech:

Meat-Tech is developing a novel biological printing process designed to create living, edible meat tissue using cellular agriculture. Meat-Tech is developing technologies, processes and machines for cultivating, producing, and printing cultured meat. The company believes that it was the first in the world to use edible biological inks to 3D-print living tissue made up of various cells of bovine origin. The Company has the technology, knowledge and experience in applying tissue engineering practices for producing fat and muscle tissue for food consumption, as well as the ability to print, using a 3D bioprinter, a combination of live animal cells, growth factors and biological materials to produce living tissues that mimic the characteristics of natural tissue.

Forward-Looking Statements:

This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, but not limited to, statements regarding the Company's development of the next generation of cultured meat food products by leveraging 3D digital printing technology, Peace of Meat's development of cultured fat products, the expected closing of the Company's acquisition of Peace of Meat and the expected post-closing synergies of the combined companies. These forward-looking statements include information about possible or assumed future results of the Company's business, financial condition, results of operations, liquidity, plans and objectives. In some cases, you can identify forward-looking statements by terminology such as "believe," "may," "estimate," "continue," "anticipate," "intend," "should," "plan," "expect," "predict," "potential," or the negative of these terms or other similar expressions. Forward-looking statements are based on information the Company has when those statements are made or management's current expectation and are subject to risks and uncertainties that could cause actual performance or results to differ materially from those expressed in or suggested by the forward-looking statements. Actual results could differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. Except as required by law, the Company undertakes no obligation to update publicly any forward-looking statements after the date of this press release to conform these statements.

COMPANY / INVESTOR CONTACT:Eran Gabay, Partner, Director of Strategy Gelbart-Kahana Investor Relations: [emailprotected]

SOURCE Meat-Tech 3D Ltd.

Read more here:
Meat-Tech Agrees to Acquire Cultured Fat Pioneer 'Peace of Meat' - PRNewswire

Read More...

Stem Cell Manufacturing Market Size, Overview with Detailed Analysis, Competitive landscape, Forecast to 2027 – Cheshire Media

Saturday, December 12th, 2020

What are the emerging opportunities in the Stem Cell Manufacturing Market?

Currently, significant business sectors are going through changes. This has prompted huge benefits for a few and misfortunes for other people. To make the most out of the emerging chances, you can exploit the most granted Stem Cell Manufacturing market report. It offers nitty-gritty experiences into the market. This will doubtlessly manage their organizations in creating the following move over the impending industry quarters.

It must be noticed that the Stem Cell Manufacturing market is encountering unexpected ascent in reception by numerous new players (from MNCs to SMEs). It is because of the way that even with the market turbulences the Global market came out with solid numbers. With the new business running into the market, it is basic to stand apart from the group. The customers can undoubtedly accomplish this utilizing the means referenced in the Stem Cell Manufacturing market report.

What type of investigation is done in the Stem Cell Manufacturing market report?

Analysis of various socioeconomics for venturing into the market is important as it will hugely affect the development throughout the following coming years. The Stem Cell Manufacturing market report is planned subsequent to doing long periods of exploration and the information sifted through in the report was gathered from dependable sources, for example, government sites.

As the market is gigantic, it turns out to be imperative to comprehend the market from its underlying foundations. Get a superior perspective on the Stem Cell Manufacturing market through the data referenced in the committed areas of the report. With this, the customers likewise get a perspective on the business structure of the contenders.

What are the reasons that impact the growth of the Stem Cell Manufacturing Market?

As of late, the advanced transformation has pushed the associations in changing into a computerized business for coming to the intended interest group present over the globe. The Stem Cell Manufacturing market report shares the significance of neighborhood language, partners, and political situation that will drive the Global market higher than ever.

With the best possible comprehension of the Stem Cell Manufacturing market, the associations can hop onto the open doors that will bring productive outcomes. The customers can exploit the biggest in-house information base for taking the most effective actions. Likewise, the Stem Cell Manufacturing market report has committed segments covering the insights concerning the current market players. With the information on how they function and accomplish their objectives, your business can likewise be formed as needs be.

What type of analysis short-term or long-term is added in the Stem Cell Manufacturing Market report?

This is important to sidestep any negative circumstances that may affect the development of the organizations. In addition, the associations need to comprehend Porters five powers that shape the market elements. In the event that the customers wish to add or eliminate the names of the organizations, it tends to be done, to suit the desires for the clients, for accomplishing long-haul objectives. SWOT investigation helps in uncovering the qualities and shortcomings of the business. The PESTLE investigation helps in checking the outer elements that shape the market overall. Subsequently, for making a name in the Stem Cell Manufacturing market, it gets important to get thought from both SWOT and PESTLE investigations.

The Stem Cell Manufacturing market report likewise brushes over the significance of language and partners for the smooth working of the associations. Thusly, the customers are stacked with data and steps and are prepared to move into the market for accomplishing the ideal objectives.

About Us:

Verified Market Reports is a leading Global Research and Consulting firm servicing over 5000+ customers. Verified Market Reports provides advanced analytical research solutions while offering information enriched research studies. We offer insight into strategic and growth analyses, Data necessary to achieve corporate goals, and critical revenue decisions.

Our 250 Analysts and SMEs offer a high level of expertise in data collection and governance use industrial techniques to collect and analyze data on more than 15,000 high impact and niche markets. Our analysts are trained to combine modern data collection techniques, superior research methodology, expertise, and years of collective experience to produce informative and accurate research.

Contact us:

Mr. Edwyne Fernandes

US: +1 (650)-781-4080UK: +44 (203)-411-9686APAC: +91 (902)-863-5784US Toll-Free: +1 (800)-7821768

Email: [emailprotected]

See original here:
Stem Cell Manufacturing Market Size, Overview with Detailed Analysis, Competitive landscape, Forecast to 2027 - Cheshire Media

Read More...

Rocket Pharmaceuticals Presents Positive Clinical Data from its Fanconi Anemia and Leukocyte Adhesion Deficiency-I Programs at the 62nd American…

Saturday, December 12th, 2020

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today presents updated interim data from its Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) programs at the 62nd American Society of Hematology (ASH) Annual Meeting. The data are highlighted in two oral presentations.

We are highly pleased with the data presented at ASH demonstrating ongoing evidence of efficacy and durability using Process B in both FA and LAD-I as we move towards potential registration, said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. Follow-up data from the Phase 1 and 2 trials for FA continue to support RP-L102 as a potential hematologic treatment option in the absence of cytotoxic conditioning. In five of the seven patients treated as of October 2020, there was evidence of engraftment. In addition, stabilization of peripheral blood counts in two of the three patients with at least 12-month follow-up, which declined substantially in these patients prior to gene therapy, suggests a halt in bone marrow failure progression. We look forward to reporting longer-term follow-up on these patients in the first half of 2021.

Dr. Shah continued, Additionally, we continue to see encouraging evidence of efficacy for RP-L201 for the treatment of LAD-I. Patients have shown sustained CD18 expression of 23% to 40%, far exceeding the 4-10% threshold associated with survival into adulthood. These data, on top of our exciting results from our lentiviral program for PKD, show our steady progress across three of our five gene therapy programs. We are proud of this progress and are committed to advancing our investigational gene therapies through development for patients and families facing these devastating disorders.

Key findings and details for each presentation are highlighted below. To access the presentations at the conclusion of the oral presentation, please visit: https://www.rocketpharma.com/ash-presentations/

Gene Therapy for Fanconi Anemia, Complementation Group A: Updated Results from Ongoing Global Clinical Studies of RP-L102The data presented in the oral presentation are from seven of the nine patients treated as of the cutoff date of October 2020 in both the U.S. Phase 1 and global Phase 2 studies of RP-L102 for FA. Seven patients had follow-up data of at least 2-months, and three of the seven patients had been followed for 12-months or longer. Key highlights from the presentation include:

Presentation Details:Title: Gene Therapy for Fanconi Anemia, Complementation Group A: Updated Results from Ongoing Global Clinical Studies of RP-L102Session Title: Gene Editing, Therapy and Transfer IPresenter: Agnieszka Czechowicz, M.D., Ph.D., Assistant Professor of Pediatrics, Division of Stem Cell Transplantation, Stanford University School of MedicineSession Date: Monday, December 7, 2020Session Time: 11:30 a.m. - 1:00 p.m. (Pacific Time)Presentation Time: 12:15 p.m. (Pacific Time)

Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1The data presented in the oral presentation are from three pediatric patients with severe LAD-I, as defined by CD18 expression of less than 2%. The patients were treated with RP-L201, Rockets ex-vivo lentiviral gene therapy candidate. Patient L201-003-1001 was 9-years of age at enrollment and had been followed for 12-months as of a cutoff date of November 2020. Patient L201-003-1004 was 3-years of age at enrollment and had been followed for over 6-months. Patient L201-003-2006 was 7-months of age at enrollment and was recently treated with RP-L201. Key highlights from the presentation include:

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

Presentation Details:Title: Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1Session Title: Gene Editing, Therapy and Transfer IPresenter: Donald Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los AngelesSession Date: Monday, December 7, 2020Session Time: 11:30 a.m. - 1:00 p.m. (Pacific Time)Presentation Time: 12:30 p.m. (Pacific Time)

Conference Call DetailsRocket management will host a conference call and webcast today December 7, at 6:00 p.m. EST. To access the call and webcast, please click here. The webcast replay will be available on the Rocket website following the completion of the call.

Investors may listen to the call by dialing (866) 866-1333 from locations in the United States or +1 (404) 260-1421 from outside the United States. Please refer to conference ID number 50038102

About Fanconi AnemiaFanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natural gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells.

About Leukocyte Adhesion Deficiency-ISevere Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

About Rocket Pharmaceuticals, Inc.Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking StatementsVarious statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, filed November 6, 2020 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

The rest is here:
Rocket Pharmaceuticals Presents Positive Clinical Data from its Fanconi Anemia and Leukocyte Adhesion Deficiency-I Programs at the 62nd American...

Read More...

Page 4«..3456..1020..»


2024 © StemCell Therapy is proudly powered by WordPress
Entries (RSS) Comments (RSS) | Violinesth by Patrick