StemCell Therapy

Israeli biotech Pluristem is canning its experimental phase 3 critical limb ischemia therapy after an outside review said it was no good.

Haifa, Israel-based Pluristems R&D operation is built upon placenta-derived adherent stromal cells, which the biotech has designed for use in patients of all human leukocyte antigen types. This approach is made possible by the low immunogenicity of the cells. Once inside the body, Pluristem hopes the cells will drive the healing of injured tissue.

But one of its leading contenders using this approach has been judged a failure in phase 3: An independent data monitoring committee (DMC) took a look at the ongoing data for its pivotal phase 3 in patients with critical limb ischemia (CLI), a severe obstruction of the arteries which markedly reduces blood flow to the extremities and can lead to amputation.

Your Guide to Maximizing the Benefits of a Unified Clinical Data Capture and Management Platform

Learn how a unified platform can increase visibility across your trial. Adopt technology solutions driving faster, more accurate data interpretation for better decision-making to remain competitive in a new era of drug development.

The DMC said the test was unlikely to meet the primary endpoint, and that the CLI study population has experienced a substantial low number of events (major amputation of the index leg or death), different from what is known in clinical medicine for the rate of these events in this patient population. The lower than anticipated event rate in the placebo group reduced the statistical power of the study to meet its primary endpoint.

The biotech is now tossing out the therapy and will instead focus on other pipeline areas, including a long-shot stem cell attempt at treating COVID-19. The biotechs shares fell nearly 40% on the news.

We are deeply disappointed by the outcome of the CLI interim analysis. In light of the DMCs recommendation, we decided that it would be in the best interests of the company and its shareholders to terminate the CLI study and focus our resources and efforts on our other lead indications, said Pluristem CEO and President Yaky Yanay.

We expect to present topline clinical results during calendar year 2021, including our phase 3 study in muscle regeneration following hip fracture, phase 2 studies in Acute Respiratory Distress Syndrome associated with COVID-19 and our phase 1 study in incomplete hematopoietic recovery following hematopoietic cell transplantation. Pluristem is well positioned to advance and support future development of these indications.

Last year, Pluristem penned a deal with NASA to assess its cell therapies against the health problems caused by spending time in space, teaming up with NASAs Ames Research Center for the project, which focuses on using its PLX placenta-derived cell therapies to try to prevent or treat medical conditions that can occur during and after space missions, including conditions that affect the blood, bone, muscle, brain and heart.

See the original post here:
NASA-partnered Pluristem crashes to Earth as it axes leading therapy - FierceBiotech

High-dose melphalan (Evomela) followed by autologous stem cell transplant (ASCT) has demonstrated significant efficacy for the treatment of patients with multiple myeloma. The agent has improved progression-free survival (PFS) in patients when administered as upfront therapy to patients aged 66 years or younger.

The median PFS shown with melphalan plus ASCT in a phase IFM/DFCI 2009 study was 50 months versus 26 months when compared with the standard of care treatment regimen lenalidomide (Revlimib), bortezomib (Velcade), and dexamethasone (RVD) alone hazard ratio for disease progression or death, 0.65;P<.001). As the treatment strategy continues to be explored in patients with multiple myeloma, researchers have now begun to investigate an outstanding biological question of whether the alkylating agent causes an increased amount of DNA damage.

An analysis presented during the virtual 2020 American Society of Hematology (ASH) Annual Meeting showed that between the time of diagnosis and relapse, patients treated with high-dose melphalan had an increased number of point mutations. It was unclear from this research how this result translated to treatment selection and sequencing.

In an interview with Targeted Oncology, Mehmet Samur, PhD, senior research scientist, Dana-Farber Cancer Institute, discussed the investigation of high-dose melphalan following ASCT in patients with multiple myeloma and shared insights into how the ongoing questions can be explored in the future.

TARGETED ONCOLOGY: Can you explain what was demonstrated prior with high-dose melphalan followed by ASCT in patients with multiple myeloma?

Sumar: The clinical part of the phase 3 study was published previously. It showed that when you do RVD plus high-dose melphalan following by stem cell treatment, patients do significantly better than patients who get RVD alone. Adding high-dose melphalan increased the PFS benefit by around 12 months.

TARGETED ONCOLOGY: Can you provide background on your analysis of Melphalan for patients with multiple myeloma?

Sumar: Melphalan is an alkylating agent. Because of the way the agent works, we always think that it creates more DNA damage. The study that we presented at ASH was questioning whether this was true or not.

We collected DNA sequencing data from patients who were treated with RVD followed by high-dose Melphalan and a bone marrow transplant. We had a total of 25 patients, and we collected data at the time of diagnosis and the time of relapse. To compare this compilation, we also collected data from 43 patients from the IFM/DFCI 2009 study who only received RVD. We also collected data at diagnosis and relapse in the 43 patients. Genomic alterations were compared at diagnosis and relapse for patients who were injected with high-dose Melphalan and RVD versus patients who were only treated with RVD.

TARGETED ONCOLOGY: What were the findings from this study?

Sumar: We found that patients who got high-dose melphalan plus RVD followed by transplant accumulated more point mutations. To be precise, they accumulated around 10,000 new mutations between diagnosis and relapse at 5 years. For RVD patients, there were around 4500 new point mutations. The study showed that treating patients with high-dose Melphalan is increasing the mutational load by about 2.9-fold at the time of relapse.

TARGETED ONCOLOGY: What are the implications of these findings?

Sumar: There are a couple of things that we see from our study. One point is that we only saw point mutations. We didnt see any large-scale DNA alterations. This suggests that our patients who are treated with high-dose melphalan are more likely to experience changes.

In terms of the pathways that are mutated, the DNA damage repair pathway is more frequently mutated between diagnosis and relapse in patients treated with high-dose melphalan. We think that if we combine inhibitors that can overcome the selection of DNA damage repair pathway mutations, those patients may get additional benefit from the treatment.

We dont have clear data yet on whether this increased mutational load is something that is bad for patients. Even though these patients have more mutations, overall survival times are similar between the 2 arms. C outcomes are not impacted by the increased number of mutation so far.

TARGETED ONCOLOGY: What plan are underway to further this research?

Sumar: We are expanding our study in multiple ways. There is no clear data set we can get answers to our ongoing questions yet. We have reached out to our partners around the world to see if we can come up with a cohort to investigation. Also, we are looking at impact of these mutational load increase on other features like secondary cancer rate.

TARGETED ONCOLOGY: The understanding of gene mutations in myeloma is evolving. Can you discuss the current role of genomic testing?

Sumar: It has been shown in many studies that genetic testing at diagnosis can tell us which patients are high risk and which are low risk. Studies have also shown that patients who have loss of p53 or with deletion 17p will have bad outcomes.

There was a study published last year in the Journal of Clinical Oncology showing which patients with myeloma would have a lower risk. The study also shows that there are certain genomic features prolong survival time in patients.

We have different genomic tools that we can use to look at these different alterations and assess patient risk. Today, I think people are looking at these alterations from all different angles to plan stratification in upcoming clinical trials.

TARGETED ONCOLOGY: In your opinion, what change will we see in the myeloma treatment landscape in the next 5 years?

Sumar: There are a lot of studies looking at new treatment. Everyone is carefully watching out for data on new treatment options like chimeric antigen receptor T-cell therapy, bispecific antibodies, and monoclonal antibodies. It looks like these agents are providing benefit to patients, but they are at the very early stages of research.

Reference:Attal M, Lauwers-Cances V, Hulin C, et al. Lenalidomide, bortezomib, and dexamethasone with transplantation for myeloma. N Engl J Med. 2017; 376(14):1311-1320. doi: 10.1056/NEJMoa1611750

View post:
Exploring Increase of Point Mutations Associated with High-Dose Melphalan in Multiple Myeloma - Targeted Oncology

Take a look at a month-by-month recap of the biggest stories at the University of Miami during the past year.

From a pandemic that forced the migration of spring semester classes to an online environment, to a new dean for the College of Engineering, to a Super Bowl halftime performance by the Band of the Hour, to a record-breaking gift for the Sylvester Comprehensive Cancer Center, 2020 proved to be a very unusual year for the University of Miami.

January

The Planet Kreyol student organization and the Office of Multicultural Student Affairs commemorate victims of the 2010 Haiti earthquake with dance, song, poetry, and more on January 15.

With evidence mounting that climate change is worsening everything from wildfires to hurricanes, the University of Miamis Rosenstiel School of Marine and Atmospheric Science hosts the three-day Miami Climate Symposium 2020: Predicting and Living with Extremes. The summitheld Jan. 22-24explored how sea level rise, saltwater intrusion, extreme heat waves, and other conditions exacerbate tropical cyclones, storm surge, and coastal flooding, as well as analyzed adaptation policies and strategies.

A professor of jazz trumpet at the Frost School of Music and four alumni of the University of Miami receive Grammy Awards during the 62nd annual ceremony held Jan. 26 in Los Angeles. Brian Lynch, jazz trumpet professor, is honored in the category of Best Large Jazz Ensemble. Cristian Macelaru, B.M. 03; Julio Reyes Copello, M.M. 00; Carlos Fernando Lopez, B.M. 12; and Natalia Ramirez, M.A. 17, also win Grammy Awards.

Miami baseball alumnus Tommy Adams makes the largest gift in support of the baseball program by a former University baseball student-athlete, donating $1 million toward the University of Miamis Baseball Facility Enhancement Campaign.

February

Five months after Hurricane Dorian devastated the Bahamas, students enrolled in the semester-long action project interdisciplinary class offered by the Miami Herbert Business School travel to Freeport for a three-day visit, meeting with Bahamian students and local businesses to share ideas and inspire hope.

Building on the immense resources and expanding the vision of the Center for Computational Science, the University establishes the Miami Institute for Data Science and Computing to catalyze data-intensive research that will solve real-world problems and enhance the understanding of data science among students and the public.

The Lancet, one of the worlds leading medical journals, announces that Felicia Marie Knaul, director of the University of Miami Institute for Advanced Study of the Americas, will lead a new Lancet Commission examining gender-based violence and maltreatment of young people, two areas with a dearth of study and understanding.

Students from the University of Miamis Frost Band of the Hour and the color guard, as well as the Hurricanettes dancers, perform in the Pepsi Super Bowl LIV Halftime Show at Hard Rock Stadium on Feb. 2.

Redshirt senior David Dinsmore wins his fourth straight gold medal in the mens platform on Feb. 21, capturing top honors at the 2020 ACC Swimming and Diving Championships.

At the Dolphins Cancer Challenge, Team Hurricaneswith 1,250 strongjoined thousands of others on Feb. 29 to run, walk, and ride to fight cancer and support the Sylvester Comprehensive Cancer Center.

March

For the health and well-being of the campus community, the University extends spring break for students through March 22, announcing that classes will resume on March 23 but strictly in online environments through at least April 4. Shortly thereafter, with COVID-19 cases surging across the nation, the University, in accordance with public health guidance to reduce density on campus, extends online instruction through the remainder of the spring semester and implements partial closing of on-campus housing.

April

An international team of scientists led by Dr. Camillo Ricordi, director of the Diabetes Research Institute and Cell Transplant Center at the University of Miami Miller School of Medicine, is granted immediate FDA authorization for a 24-patient clinical trial to test the safety and exploratory efficacy of umbilical cord-derived mesenchymal stem cells to block the life-threatening lung inflammation that accompanies severe cases of COVID-19.

Joined by the Rapid Defense Network in New York, the Southern Poverty Law Center, and others, the School of Laws Immigration Clinic files a lawsuit on April 13 accusing U.S. Immigration and Customs Enforcement authorities of ignoring COVID-19 guidelines in three Florida detention centers.

Debbie Ajagbe is named the 2020 ACC Indoor Track Scholar-Athlete of the Year on April 17, with five other Miami women joining her on the All-ACC Academic Team for Indoor Track and Field. Earlier in the year, Ajagbe, a mechanical engineering major, earned both ACC Womens Field Performer of the Year and ACC Championship Field MVP honors, winning both the weight throw and shot put at the conference championships.

May

The 180 graduates of the Miller School of Medicines Class of 2020 celebrate their newly minted degrees during a virtual commencement on May 9.

Arva Moore Parks, a prominent historian and preservationist, who served on the University of Miami Board of Trustees for 26 years and wrote several books on Greater Miami, Coral Gables, and University history, passes away on May 10.

Nine University of Miami Athletics programsmens basketball, mens and womens cross country, mens diving, golf, rowing, mens and womens tennis, and womens track and fieldare recognized for perfect single-year scores of 1,000 in the 2018-19 Academic Progress Report released May 19 by the NCAA.

UMTVs first Black show, The Culture, is nominated by the Suncoast Chapter of the National Academy of Television Arts & Sciences for a student production award in the magazine program category.

Brian Van Belle and Chris McMahon, two of the Miami Hurricanes best pitchers, are named All-Americans by Collegiate Baseball Newspaper on May 26. Both Van Belle and McMahon earned spots on the second team after posting brilliant performances in the abbreviated 2020 season that was halted due to the COVID-19 pandemic.

June

Pratim Biswas, the Lucy and Stanley Lopata Professor in the McKelvey School of Engineering at Washington University in St. Louis and a pioneer in his field recognized for applying aerosol science and engineering to multiple areas, is named dean of the University of Miami College of Engineering.

The University of Miami Board of Trustees elects six new members to its ranks with expertise in business, finance, law, technology, and strategic planning. Patricia Menendez-Cambo, Adam E. Carlin, Jose R. Mas, Alice S. Vilma, Carolyn B. Lamm, and Jordan Rhodes were elected to the Board on June 19.

Dr. Judy Schaechter, chair of the Miller School of Medicine Department of Pediatrics, is named a 2020-21 Health Policy Fellow by the Robert Wood Johnson Foundation and the National Academy of Medicine. The prestigious one-year fellowship in Washington, D.C., will enable her to expand her longtime involvement in health policy and child policy at the local and state levels to the federal level.

July

The University becomes one of 89 locations around the nation, and one of only six in Florida, to enroll volunteers for the first Phase 3 clinical trial of a COVID-19 vaccine. The trial, part of the National Institutes of Health COVID-19 Prevention Trials Network, tested a vaccine developed by scientists at the NIHs National Institute of Allergy and Infectious Diseases and collaborators at biotechnology company Moderna, Inc. Vice President Mike Pence visited the Miller School of Medicine along with Florida Gov. Ron DeSantis on July 27 to thank the University for its participation in the trial.

The National Oceanic and Atmospheric Administration selects the Rosenstiel School of Marine and Atmospheric Science to host the Cooperative Institute for Marine and Atmospheric Studies, which will bring together the research and educational resources of 11 partner universities to increase scientific understanding of the Earths oceans and atmosphere within the context of NOAAs mission. The selectionmade through an open, competitive evaluationcomes with an award of up to $310 million over the course of five years, with the potential for renewal for another five years based on successful performance.

Amid ongoing nationwide protests against police brutality sparked by the tragic death of Minneapolis resident George Floyd in May, President Julio Frenk reaffirms his commitment to racial and ethnic equality, outlining in a letter sent to all students, faculty, and staff a 15-point plan the University will implement to support racial equality, inclusion, and justice across the institution and in the greater South Florida community.

Patti Herberta longtime University of Miami alumna and benefactor who, along with her husband Allan, donated millions of dollars to the institution, helping to transform academics and student lifepassed away on Monday, July 27. She was 84.

University of Miami Libraries launches Documenting COVID-19: South Floridas Pandemic Experience. Through community-generated and community-contributed content that will be made available through digital collections and by visiting the library, the initiative will chronicle how local communities are dealing with the crisis.

August

In a move to support a safe learning and working environment for students, faculty, and employees, the Butler Center for Service and Leadership establishes a new team of public health ambassadors to support the Universitys COVID-19 reopening and operating plan. The 75 students who made up the new Public Health Ambassadors Program during the fall semester enforced guidelines on the Coral Gables Campus by offering support and utilizing peer-to-peer influence to encourage members of the campus community to engage in the healthy behaviors of physical distancing, wearing face coverings, and hand washing/sanitizing.

Lakeside Village, a 12-acre facility on the shores of Lake Osceola in the heart of the Coral Gables Campus, officially opens its doors on Aug. 13 to greet the inaugural class of students to the transformative housing complex.

Featuring a mix of virtual and in-person instruction, the first day of fall semester classes begins on Aug. 17. On the Coral Gables Campus, a number of safety protocolsfrom mandatory mask-wearing to social distancinghelp ensure the well-being of students, faculty, and staff.

The Miller School of Medicine Class of 2024 launches the innovative NextGenMD Curriculum, which focuses on health system science and features an enhanced emphasis on mentorships. The students will be significantly better prepared to respond to COVID-19 and to the public health challenges that will follow.

The Miller School of Medicine becomes one of a few medical schools across the nation selected by the National Institutes of Health to test the effectiveness of treating COVID-19 patients with convalescent plasma.

Following a limited opening in April 2020, Canes Central, a new student-centered, service-oriented department, fully opens. It offers both in-person and online undergraduate and graduate students assistance on matters relating to registration and records, billing and payment, financial aid, and Cane Cards.

Legendary Miami Hurricanes baseball coach Jim Morriswho in his 25 seasons at the University of Miami won 1,090 games, made the NCAA postseason 23 straight years, reached the College World Series 13 times, and won national championships in 1999 and 2001is voted into the 2020 induction class of the National College Baseball Hall of Fame.

September

Sylvester Comprehensive Cancer Center at the University of Miami Leonard M. Miller School of Medicine receives a landmark gift of $126 million. The groundbreaking donationthe single largest in the University of Miamis 95-year historywill accelerate breakthrough advances in finding cures for cancer and expand innovative treatment options for cancer patients.

The University of Miami jumps eight spots to No. 49 in U.S. News & World Reports 2021 Best Colleges issue, placing the institution back among the 50 top-tier colleges and universities. This jump in the rankings reflects our commitmenteven in these unprecedented timesto comprehensive excellence and selective preeminence, said President Julio Frenk.

Sylvester Comprehensive Cancer Center, part of the University of Miami Health System, opens the Dwoskin Proton Therapy Center on Sept. 15. The new state-of-the-art facility treats patients with proton therapy, an advanced type of low-dose radiation that is extremely precise and two-thirds the speed of light.

Physician-researchers with the Miller School of Medicine begin a new Phase 3 clinical trial to test another investigational vaccine for COVID-19. Part of a large-scale international trial in partnership with Janssen Pharmaceuticals, the clinical trial to test the Janssen vaccine is the Miller Schools second human study of its kind.

October

The University of Miami becomes the first college testing site for a quick, easy, and cost-effective Israeli-produced COVID-19 breath analyzer that could revolutionize coronavirus testing if approved by the FDA.

In a Miami Herbert Business School webinar held Oct. 8, U.S. Secretary of Health and Human Services Alex Azar highlights the administrations effort to restructure the health care system to combat the COVID-19 pandemic and support countries in Latin America and the Caribbean.

November

Taking their dedication to fighting cancer to a new level, the Miami Dolphins pledge a transformational $75 million gift to Sylvester Comprehensive Cancer Center at the University of Miami Leonard M. Miller School of Medicine, South Floridas only NCI-designated cancer center.

Musicians from the Frost School of Music join Dean Shelly Berg and celebrity musicians to participate in a benefit concert on Thanksgiving Day in support of nurses. The livestreamed Nurse Heroes Live! concert raises funds for the Nurse Heroes Foundation, an initiative working to support and honor nurses.

December

Four extraordinary University of Miami alumniJose R. Mas, Jackie Nespral, Hilarie Bass, and Jaret L. Davisshare their advice with more than 5,000 students at four virtual commencement ceremonies held Dec. 10 and 11.

The rest is here:
2020 at the U: The year in review - University of Miami

COVID-19 has many symptoms, including fever, coughing, and fatigue. But one of the more distinctive signs is the loss of the ability to smell. Were not talking about the usual stuffy nose that goes along with a cold, but an inability to process scent even when youre not congested. People have reported that not being able to smell their own perfume or finding no aroma in their cup of mint tea was their first clue that they might be infected.

James Schwob, a professor of developmental, molecular, and chemical biology at Tufts University School of Medicine, researches the olfactory system and the roughly 1,000 types of neurons that are involved in our ability to register odors both good and bad.

Right now, he is studying tissue from COVID-19 patients to better understand how the virus leads to anosmia, or loss of sense of smell. Tufts Now talked to Schwob about what we know about viruses and their effects on sense of smell.

Tufts Now: How does sense of smell work?

James Schwob: The sense of smell operates by chemicals wafting in on the air and reaching the upper and back parts of the nasal cavity. Those chemicals bind to receptors on sensory neurons in the epitheliumthe thin tissue that lines the nasal passages. Those neurons then send a signal up the olfactory nerve into the brain, where it registers as the delicious smell of coffee or fresh cut grass.

What causes you to lose it?

There are a number of pretty well-known causes for loss of sense of smell. One is via post-viral infection, and we think that has something to do with the immune system causing inflammation. It also happens with head injuryin that case, its likely the part of the brain that receives the smell signals that is damaged. Toxin exposurefrom cadmium, formaldehyde, or methyl bromide, for examplewill make you lose your sense of smell. Chronic sinus infections and simply the aging process can also cause anosmia.

How common is loss of sense of smell among COVID-19 patients?

We have long known that people can lose their sense of smell after other viral infections, such as the flu, but the percentage of people who have had this problem with COVID-19 is quite remarkable. One study from Iran reported 98 percent of hospitalized patients had an objective problem with their sense of smell.

How does COVID-19 cause people to lose their sense of smell?

We dont really know why this happens with COVID-19. It could be that the virus is harming the neurons that send smell signals to the brain, or that the bodys immune system, in trying to deactivate the virus, is killing other, supporting cells that are part of that pathway.

There is some evidence that certain cells in the lining of the nasal passages express a protein receptor, called ACE2, that the coronavirus uses it as a way to infiltrate the body. That is one of the things Im hoping to investigate.

Back in 2000, colleagues and I published two papers looking at a different coronavirus, called mouse hepatitis virus. We looked at what effect that had on the peripheral olfactory nerve in the central olfactory system. What we found was that this virus would pass up that nerve into the brain and cause problems in the brain.

One of the other things that has been described is that there have been some neurological symptoms due to the infection with the SARS-CoV2-virus. And one of the questions we have is whether the virus is crawling up the nerve in some fashion in these patients who have died of the infection.

The good news is that the olfactory epithelium contains stem cells that can give birth to new neurons throughout life as long as they remain intact. So the system has a capacity to repair itself. Some COVID-19 patients have recovered their sense of smell within a couple weeks. Thats actually quicker than you would expect new neurons to be created, so there could be some sort of functional disruptionrather than neuron deathgoing on.

With all the more serious symptoms associated with COVID-19, why is sense of smell worth investigating?

Sometimes loss of smell is a COVID-19 patients only symptom. Any symptom that can be tied directly to the disease becomes an important one to be aware of, so that it can be used to guide testing and keep people from unknowingly spreading the disease. That is part of the reason I think its important to figure this out.

An intact sense of smell is also critical to good nutrition. If smell is lost so is most of foods flavor. As a consequence, patients may overeat (to try to get the pleasure back), undereat (why bother?) or over-salt or -spice their food, because those aspects of food flavor can still be detected by nerves and taste buds in the oral cavity.

Loss of sense of smell can be very disturbing, because eating is of our great pleasures in life. We dont want to lose that when we have so few pleasures left to us now that were stuck at home.

How can a person know if their sense of smell is really hampered, and they arent just imagining it?

One of the things that can be done pretty easily, pretty objectively by someone at home would be to take some ground coffee and see how far away you can hold it and still smell it. Or do the same with rubbing alcohol or your shampoo. If your nose is not congested and you have trouble recognizing those or other scents that are familiar to you, you might want to call your doctor about getting tested.

Julie Flaherty can be reached at julie.flaherty@tufts.edu.

Link:
Something Wrong With Your Sniffer? It Could Be the Coronavirus - Tufts Now

I

m going to turn 40 this month, a milestone Im approaching with nine-tenths equanimity, one-tenth mild existential dread.

On the one hand, with a kid and a job, Im too busy to spend much time peering at my newly middle-aged navel. Plus, if youre lucky enough to be healthy, griping about simply growing older is churlish. On the other, there is an undeniable psychic wrench in the realisation that I now tick the aged 40-60 box. Somehow, I seem to have lurched from young and clueless to the age where people will make jokes about the number of candles on my cake, without an intervening period of solid competence. I still run for the bus. I can still never find the lids for the Tupperware. I am older than the prime minister of Finland, but I cant reliably pair my own socks. I dont feel fully grown up, and yet there is the creeping sense that, as Sue Townsend put it via her timeless mouthpiece Adrian Mole, Im on a pathetic slide towards gum disease, wheelchair ramps and death.

But is this slide inevitable? Thats the central question of Ageless: The New Science of Getting Older Without Getting Old, an ambitious and energetic new book by the scientist and writer Andrew Steele. While hes not peddling some holy grail of immortality, he does give a startling round-up of the biological factors that make us age and the emerging techniques to tackle them, offering the prospect of both longer and healthier lives.

To start with some perspective: human lifespans vary hugely based on time and place, with life expectancy under the age of 40 the norm everywhere at the start of the 19th century, largely due to high child mortality. Drawing examples from the animal kingdom, Steele points out that innings vary from five minutes for a mayfly to 400 years for a Greenland shark. We are, of course, neither flies nor sharks. But if we accept the idea that a human life isnt capped at 82 and pursue all the opportunities of frontier research, perhaps we could become more like tortoises. As Steele explains, the Galapagos tortoise, along with a handful of other blessed oddballs in the natural world, are negligibly senescent meaning they have no obvious impairments of movement or senses as they get older, and they experience no age-related declines in fertility.

They dont live forever. But referring to a giant tortoise discovered by Charles Darwin who lived until 2006, Steele writes: Harriet was likely pretty much as sprightly at 170 as she was at 30, at the height of Queen Victorias reign which is to say, not very; she was a giant tortoise, after all.

This liveliness of tone helps to carry the reader through an unsparing account of the toll ageing takes on the human body. The comic writer Nora Ephron may have called her mature memoir I Feel Bad About My Neck, but ageing emerges from this book as less aesthetic challenge, more devastating, systemic collapse. The gloomiest statistic of the lot? Your chance of death doubles every eight years, as a range of processes make us progressively more susceptible to big killers like cancer, heart disease, stroke, dementia and diabetes.

Steele gives a jauntily accessible account of the mechanisms behind our decline. One overarching driver is disposable soma theory: the evolutionary logic that prioritises the health of our reproductive cells over the soma cells which make up our own bodies. I had always suspected that having a child somehow drained me of my life force: here is the evidence. And as our neglected soma cells age, things go wrong. Our telomeres, protective caps on our chromosomes which are essential for healthy cell division, get shorter. Autophagy the process by which cells clear out their own junk declines. Proteins misfold into sticky clumps that risk forming the plaques behind Alzheimers disease. Frail old cells linger: They stick around, no longer dividing aged, zombie cells which refuse to commit cell suicide, known as senescent cells. Our mitochondria which give cells energy misfunction. Stem cells falter. Chronic inflammation ups the risk of everything from diabetes to cancer. Its a startling wake-up call that there are worse things bubbling away in your biology than a few grey hairs, or the frown lines that make every Zoom call of this pandemic a harrowing ordeal for the over-35s.

Independent Culture NewsletterThe best in film, music TV & radio straight to your inbox every week

Independent Culture NewsletterThe best in film, music TV & radio straight to your inbox every week

But what if there was another way? Dont book your return trip to the moon for 2125 just yet. The science has a long way to go: as Steele points out, most of the research is in its early stages, bristling with the potential for pitfalls and unintended consequences. But there are a number of approaches that hold promise, with todays huge advances in computing spurring new therapies. Steele posits that senolytics, or drugs that destroy senescent cells, could be with us in the next few years, albeit to tackle age-related conditions rather than ageing itself. After that, more advanced treatments like gene and stem cell therapies could be available on timescales measured in decades. Ultimately, instead of tackling the individual symptoms of ageing a creaky knee here, a furred-up artery there we will move towards systems medicine that stops us falling to bits in the first place:

The first ageless generation probably wont realise their luck at first theyll grow up expecting to die at 100, or 150, or whatever old is for their society but, one after another, lifesaving medical breakthroughs will push their funerals further and further into the future.

Steele writes with the maverick confidence of the outside perspective. He took a PhD in physics, then moved into computational biology before pursuing his interest in ageing as a writer. In this career switch, he has something in common with Aubrey de Grey, the bearded high priest of the anti-ageing movement who began with a background in computer science. While Steele stops short of making outlandish predictions de Grey famously ruffled academic feathers with his claim people could live to be 1,000 the logic is the same.

While we would all merrily jettison our wrinkles and give bowel cancer a miss, are we really ready for an ageless society, presuming the science stacks up? Steele writes quite reasonably that no one would invent the suffering of old age as a solution to an over-crowded world, but this research nonetheless opens up a fascinating Pandoras box of challenges. Rather than a utopia of dewy-faced centenarians hover-boarding to work, we would probably first see a deepening of the grotesque inequalities that already exist in health today. Theres currently a nine-year gap in lifespans between the richest and poorest parts of the UK. Add some fancy preventive drugs to the mix and you can guess who would get them first. Moreover, with ageism rife in the workplace, especially in the tech industry which provides an increasing number of jobs, who could afford not to quaff the pills that keep you looking fresh in your hoodie?

Writing with the vim of a Bill Bryson and the technical knowledge of a scientist, Steele at least gives us a chance to grasp whats at stake in this dazzling, daunting age where big data meets human biology. Negligible senescence may remain a stretch for my New Years resolutions, but Id happily progress at a more tortoise-like pace towards the next milestones of decrepitude.

Read the rest here:
Science can stop us ageing, according to a new book. But do we want it to? - The Independent

Three University of Pennsylvania researchers have been honored by The Sanford and Sue Greenberg Prize to End Blindness by 2020 for their research, which led to the first Food and Drug Administration-approved gene therapy for a genetic disease. Gustavo D. Aguirre of the School of Veterinary Medicine and Jean Bennett and Albert M. Maguire of the Perelman School of Medicine, together with William Hauswirth of the University of Florida, are recipients of the Outstanding Achievement Prize, to be awarded in a virtual ceremony today.

The four scientists share a $1 million prize, funds that will go to support further laboratory and clinical research that advances vision science. Together, their workgoing from an animal model of disease to human clinical trialsled to an FDA-approved gene therapy for Leber congenital amaurosis (LCA) caused by a mutation in the RPE65 gene, a retinal disease that causes visual impairments beginning in infancy. Now commercialized and used routinely, this treatment has reversed blindness and spared the vision of children and adults around the world.

The researchers are among 13 recipients of a total of $3 million from the End Blindness by 2020 initiative, originated by Sanford Greenberg, an

inventor, entrepreneur, and investor who serves as chairman of the board of the Johns Hopkins University Wilmer Eye Institute and who lost his sight at the age of 19 due to disease.

Gustavo D. Aguirre is professor of medical genetics and ophthalmology in the School of Veterinary Medicine. His internationally renowned research, generated during more than four decades, has investigated the genetic basis of a variety of inherited vision disorders, including LCA, Best disease, achromatopsia, and retinitis pigmentosa. His work on novel gene therapy approaches to treatment, which deliver to the eye a functional copy of a gene that is otherwise dysfunctional, has restored vision in animal models of X-linked retinitis pigmentosa and LCA. Aguirre, who earned his V.M.D. and Ph.D. from Penn, is a fellow of the Association for Research in Vision and Ophthalmology and a member of the National Academy of Medicine, College of Physicians of Philadelphia, and American Association for the Advancement of Science. He is also a recipient of the Louis Braille Award, Proctor Medal, and Foundation Fighting Blindess Board of Directors Award, among other honors.

Jean Bennett is the F.M. Kirby Professor of Ophthalmology in the Perelman School of Medicine. She is an internationally-recognized pioneer in gene therapy and has dedicated her career to restoring eyesight in the blind. Bennett earned a Ph.D. in zoology and cell and development biology from the University of California, Berkeley, and a medical degree from Harvard University, where she met her future husband and research collaborator, Albert M. Maguire. She has developed a number of strategies for gene therapy-mediated treatments for retinal disease. Her research is focused on the molecular genetics of inherited retinal degenerations in order to develop rational approaches for treating blindness. In addition to the eye, projects in Bennett's laboratory target other diseases/organs suffering from mutations in cilia proteins, including the ear and the kidney.

Albert M. Maguire is a professor of ophthalmology in the Perelman School of Medicine and an attending physician in the Division of Pediatric Ophthalmology at Children's Hospital of Philadelphia. He earned his medical degree from Harvard University, completed an internship in surgery at Yale-New Haven Hospital, a residency at Johns Hopkins Hospital, and a fellowship at the William Beaumont Hospital in Royal Oaks, Michigan. Maguire specializes in the diagnosis and treatment of pediatric retinal diseases. His research interests involve the development of treatments for incurable retinal degenerative disease, including LCA.

The hourlong streamed ceremony, freely accessible at http://www.EndBlindness2020.com, will feature Art Garfunkel, Margaret Atwood, Al Gore, Michael Bloomberg, U.S. Sen. Chris Coons, and musical performances. It will also feature a tribute to the late U.S. Supreme Court Justice Ruth Bader Ginsburg, a longtime supporter of the End Blindness movement, including exclusive footage of Ginsburg reading from Hello Darkness, My Old Friend, the memoir of Sanford Greenberg.

More here:
Vision researchers honored by End Blindness 2020 | Penn Today - Penn Today

"Kodi's inspirational tenacity and spirit is exactly the empowerment the Foundation wants to provide our community."

Kodi's Music to Our Eyes performance will feature a wide variety of covers from his musical repertoire, including You Are The Reason by Calum Scott and Don't Let The Sun Go Down On Me by Elton John. Kodi will also perform holiday favorites, Winter Wonderland by Johnny Mathis and Santa Claus Is Coming to Town by Michael Bubl.

In between sets, Jason Menzo, chief operating officer at the Foundation Fighting Blindness, will interview Kodi and his siblings, Derek and Kayla, about their experiences with Kodi's vision loss and discuss his determination to achieve his dreams no matter what obstacles come his way. Event registration is free, but attendees will have the opportunity to contribute, as all net proceeds will go towards the Foundation's mission.

"We're excited to be collaborating with Two Blind Brothers again for a livestream musical performance with Kodi Lee," says Jason Menzo, COO at the Foundation. "Kodi's inspirational tenacity and spirit is exactly the empowerment the Foundation wants to provide for the blind and low vision community."

For more information and to register for a reminder about this event, visit: https://bit.ly/3m84Wkr

About the Foundation Fighting BlindnessEstablished in 1971, the Foundation Fighting Blindness is the world's leading private funding source for retinal degenerative disease research. The Foundation has raised more than $800 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the entire spectrum of retinal degenerative diseases including: retinitis pigmentosa, age-related macular degeneration, Usher syndrome, and Stargardt disease. Visit FightingBlindness.org for more information.

Media Contacts:Chris AdamsVice President, Marketing & Communications[emailprotected]tingblindness.org (410) 423-0585

SOURCE Foundation Fighting Blindness

http://www.FightBlindness.org

Here is the original post:
Kodi Lee Teams up with Foundation Fighting Blindness and Two Blind Brothers for Music to our Eyes Livestream Music Series - PRNewswire

Glaucoma refers to the group of conditions characterized by optic nerve damage, visual field loss, and secondary to retinal ganglion cell damage, which may lead to death. According to the International Agency for the Prevention of Blindness: 2016, glaucoma is second leading cause of blindness and leading cause of irreversible blindness worldwide. The most common types of glaucoma are Primary Open Angle Glaucoma (POAG) and Primary Angle Closure Glaucoma (PACG). Moreover, PACG is most common in South-East Asian population while POAG is most common in white Caucasians and individuals of African origin. PACG is associated with a high risk of blindness as compared to POAG.

Glaucoma is a group of eye diseases which result in damage to the optic nerve and cause vision loss. The most common type is open-angle (wide angle, chronic simple) glaucoma, in which the drainage angle for fluid within the eye remains open, with less common types including closed-angle (narrow angle, acute congestive) glaucoma and normal-tension glaucoma. Open-angle glaucoma develops slowly over time and there is no pain. Peripheral vision may begin to decrease, followed by central vision, resulting in blindness if not treated. Closed-angle glaucoma can present gradually or suddenly. The sudden presentation may involve severe eye pain, blurred vision, mid-dilated pupil, redness of the eye, and nausea. Vision loss from glaucoma, once it has occurred, is permanent. Eyes affected by glaucoma are referred to as being glaucomatous.

Apply For Sample Report (Get Full Insights in PDF) with Tables, Charts, and Graphs: https://www.worldwidemarketreports.com/sample/311182

By Top Industry Players:Allergan ,Merck ,Novartis ,Pfizer ,Santen Pharmaceuticals ,Valeant Pharmaceuticals

Risk factors for glaucoma include increasing age, high pressure in the eye, a family history of glaucoma, and use of steroid medication. For eye pressures, a value of greater than 21 mmHg or 2.8 kPa is often used, with higher pressures leading to a greater risk. However, some may have high eye pressure for years and never develop damage. Conversely, optic nerve damage may occur with normal pressure, known as normal-tension glaucoma. The mechanism of open-angle glaucoma is believed to be slow exit of aqueous humor through the trabecular meshwork, while in closed-angle glaucoma the iris blocks the trabecular meshwork. Diagnosis is by a dilated eye examination.Often, the optic nerve shows an abnormal amount of cupping.

If treated early, it is possible to slow or stop the progression of disease with medication, laser treatment, or surgery. The goal of these treatments is to decrease eye pressure. A number of different classes of glaucoma medication are available. Laser treatments may be effective in both open-angle and closed-angle glaucoma. A number of types of glaucoma surgeries may be used in people who do not respond sufficiently to other measures. Treatment of closed-angle glaucoma is a medical emergency.

Product Type Coverage (Market Size & Forecast, Major Company of Product Type etc.):

ProstaglandinsAlpha AgonistBeta BlockersCarbonic Anhydrase InhibitorCholinergicCombined Medication

Application Coverage (Market Size & Forecast, Different Demand Market by Region, Main Consumer Profile etc.):

HospitalsOphthalmic ClinicsAmbulatory Surgical Centers

Thanks for your visit. If you have any other questions, please contact us. Our team will prepare a report tailored to your needs.

https://www.worldwidemarketreports.com/quiry/311182

Contact Us:

Mr. ShahWorldwide Market ReportsSeattle, WA 98154,U.S.Email:sales@worldwidemarketreports.com

More:
Glaucoma Therapeutics The market is expected to see a surge in growth | Allergan , Merck , Novartis - The Courier

Night blindness, also known as nyctalopia, is a type of vision impairment that makes it difficult to read or see in dim light or at night. It is not a disease itself, but rather a symptom of an underlying medical condition. Although the name suggests blindness during the night, it usually means difficulty in reading or driving in a dusky environment, which can happen any time during the day.

Difficulty to see in a dark room is usually normal but in nyctalopia, it is more strenuous. It can even prevent you from pointing out the stars in the night sky. Night blindness may make it difficult to recognize faces or locate obstacles in a dimly-lit room. Your vision could also take longer than usual to adjust to the transition from darkness to bright light.

Causes of night blindness

Night blindness may be caused by a variety of underlying causes including the following:

Treatment for night blindness

It is important to know the exact cause of the condition for an appropriate intervention to be planned. The treatment for nyctalopia may vary from simply getting a new pair of prescription glasses or switching glaucoma medications to surgery if the cause is cataracts.

A proper balance diet may also be advised if the condition is caused due to a dietary imbalance. Vitamin and mineral requirements may be fulfilled by supplementation, depending on your doctors recommendation. If a retinal disease is discovered, the treatment will be carried out by an ophthalmologist depending on the type and severity of the condition.

Unfortunately, treatment of genetic conditions such as retinitis pigmentosa and Usher syndrome that cause nyctalopia are yet to be developed.

Prevention and precautions

There is no way to prevent night blindness when it is caused by genetic conditions or birth defects. However, in the case of other causes, you can take the following measures to reduce the risk of developing the condition:

For more information, read our article on Night Blindness.

Health articles on News18 are written by myUpchar.com, Indias first and biggest resource for verified medical information. At myUpchar, researchers and journalists work with doctors to bring you information on all things health.

Link:
Nyctalopia: All You Need to Know About Night Blindness - News18

PETALUMA, Calif., Dec. 15, 2020 /PRNewswire/ --RetinalGeniXTM Technologies, Inc. https://retinalgenix.com/ announced today the engagement of Amato and Partnersto provide Investor Relations services. RetinalGeniXTM Technologies is a private California-based medical technology company focused on prevention of blindness through mass medical retinal screening and patient home monitoring.

RetinalGeniX is moving forward with a strategic investor relations plan that will be supported by Amato and Partners. Amato and Partners is an independent Investor Relations firm headquartered in New York City. The firm has a strong and well-recognized brand in the U.S. capital markets. The Amato team has over 40 years of experience developing and executing investor relations programs and a proven track record of achieving results both for private and public companies.

"Amato and Partners provides experienced advice and established Wall Street relationships that will cultivate company visibility with market participants including equity research analysts, investment banks and appropriate potential investors," said Jerry Katzman, M.D., RetinalGeniX's chief executive officer. "We very much look forward to working together."

About RetinalGeniX Technologies, Inc. RetinalGeniX Technologies, Inc. is a privately held emerging medical device company focused on preventing blindness through a cost-effective Mass Retinal Screening Device and a Patient Real-Time Home Monitoring Imaging & Physician Alert System. (A) The Mass Retinal Screening device provides retinal imaging that captures a 200 FOV without pupil dilation and detects the earliest stages of diabetic retinopathy to prevent blindness. This device requires submission to the FDA's 510(k) approval process. (B) RetinalGeniX's second product is RetinalCam, the first in-home, self-monitoring device providing real-time ocular and retinal imaging and streams video to the physician. The technology enables high-risk individuals to send their physician digitized retinal images and streaming video easily from their home. This second device does not require submission to the FDA's 510(k) approval.

Safe Harbor Statement This press release includes "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements in this press release include, but are not limited to, statements that relate to the advancement and development of the Mass Retinal Screening Device and a Patient Real-Time Home Monitoring Imaging & Physician Alert Systemand other information that is not historical information. When used herein, words such as "anticipate", "being", "will", "plan", "may", "continue", and similar expressions are intended to identify forward-looking statements. In addition, any statements or information that refer to expectations, beliefs, plans, projections, objectives, performance or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking. All forward-looking statements are based upon RetinalGeniX's current expectations and various assumptions. Voltron believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain. RetinalGenix may not realize its expectations, and its beliefs may not prove correct. Actual results could differ materially from those described or implied by such forward-looking statements as a result of various important factors, including, without limitation, market conditions. Consequently, forward-looking statements should be regarded solely as RetinalGeniX's current plans, estimates and beliefs. Investors should not place undue reliance on forward-looking statements. RetinalGenix cannot guarantee future results, events, levels of activity, performance or achievements. Voltron does not undertake and specifically declines any obligation to update, republish, or revise any forward-looking statements to reflect new information, future events or circumstances or to reflect the occurrences of unanticipated events, except as may be required by law.

Contact:Jerry Katzman, M.D.Chief Executive Officer(415) 578-9583[emailprotected]

SOURCE RetinalGeniX Technologies, Inc.

https://retinalgenix.com/

Link:
RetinalGeniX Has Engaged New York Investor Relations Firm Amato and Partners, LLC - PRNewswire

Many neurodegenerative conditions, from glaucoma to Alzheimers disease, are characterized by injury to axons the long, slender projections that conduct electrical impulses from one nerve cell to another, facilitating cellular communications. Injury to axons often leads to neuronal impairment and cell death.

Researchers know that inhibiting an enzyme called dual leucine zipper kinase (DLK) appears to robustly protect neurons in a wide range of neurodegenerative diseases models, but DLK also inhibits axonal regeneration. Until now, there have been no effective methods to modify genes to improve both the long-term survival of neurons and promote regeneration.

In a paper published December 14, 2020 in PNAS, a multi-university team led by researchers at University of California San Diego School of Medicine and Shiley Eye Institute at UC San Diego Health identified another family of enzymes called germinal cell kinase four kinases (GCK-IV kinases) whose inhibition is robustly neuroprotective, while also permitting axon regeneration, making it an attractive therapeutic approach for treating at some neurodegenerative diseases.

We basically figured out that there are a set of genes that, when inhibited, allow optic nerve cells to survive and regenerate, said senior author Derek Welsbie, MD, PhD, associate professor of ophthalmology in the Viterbi Family Department of Ophthalmology at Shiley Eye Institute.

Example of retinal ganglion cells with axons and dendrites in the retina of a healthy eye.

Prior to this work, the field knew how to get these cells to survive, but not regenerate. Conversely, there are ways to promote regeneration, but then the survival was rather modest. Of course, for a successful strategy of vision restoration, you need both and this is a step in that direction.

The researchers conducted a series of screens after first creating retinal ganglion cells (RGC) from human stem cells. RGCs are a type of neuron located near the inner surface of the retina of the eye. They receive visual information from photoreceptors and collectively help transmit that information to the brain.

The first screen involved testing a group of well-studied chemicals to assess their ability to increase the survival of RGCs; the second to measure the ability of chemicals to promote regeneration.

We then used a machine-learning technique to understand why certain compounds were active while others were not and it identified these key genes, said Welsbie.

The discovery that these genes improved RGC survival was not surprising, he said. However, you would have predicted that they (like DLK) would have blocked regeneration when inhibited, not promote regeneration. That was definitely a surprise. It highlights one of the advantages of discovery-based science using high-throughput screening: By testing many agents at once, we can find identify overlooked genes that might not have been thought to play a role.

Welsbie and colleagues focused their work on RGCs because they are interested in optic neuropathies, such as glaucoma. Most people think only about glaucoma in terms of eye pressure, Welsbie said. But eye pressure is only part of the problem. At its core, glaucoma is a neurodegenerative disease characterized by progressive loss of RGCs and their axons, leading to measurable structural and functional damage to the optic nerve, visual impairment and blindness.

The U.S. Centers for Disease Control and Prevention estimate 3 million Americans have glaucoma. It is the second leading cause of blindness worldwide.

Welsbie cautioned that its not yet known whether these findings extend to other neuron types, but he noted that the work suggests strong therapeutic possibilities.

Co-authors include: Amit K. Patel, Risa M. Broyer, Cassidy D. Lee, Tianlun Lu, Mai T. Vu, Karl J. Wahlin and Robert N. Weinreb, all at UC San Diego; Mikaela J. Louie, Anna La Torre and Yang Hu, UC Davis; Hassan Al-Ali, John L. Bixby and Vance P. Lemmon, University of Miami; Katherine L. Mitchell and Vinod Jaskula-Ranga and Donald J. Zack, Johns Hopkins University; Xin Duan, UC San Francisco; Santiago Vilar, Truvitech, Miami.

Funding for this research came, in part, from the National Institutes of Health (grant 1RO1EY029342), Research to Prevent Blindness, E. Matilda Ziegler Foundation, Brightfocus Foundation, Fight for Sight Foundation, the Glaucoma Research Foundation Catalyst for the Cure and the Tushinsky family.

Go here to read the rest:
Researchers Discover Clue to How to Protect Neurons and Encourage Their Growth - UC San Diego Health

Jos-Alain Sahel was on a rare vacation in Portugal in the spring of 2018 when his phone rang with grim news: The gene therapy he had worked on for a decade, a potential cure for a rare form of blindness, had failed in a pivotal trial.

In the first minute, I was very disappointed, Sahel says. I said, well OK, its not working.

A failed trial in drug development is crushing but not unexpected, a tradeoff of doing business in biology. You examine the full data, go back to the drawing board and either abandon the effort or tweak and try again. Sahel, founder of four companies and the longtime head of the Vision Institute of Paris, was used to the process. But this time, when the full data came, he was bewildered.

Unlock this story instantly and join 95,300+ biopharma pros reading Endpoints daily and it's free.

SUBSCRIBE SIGN IN

More here:
They thought their gene therapy failed. Instead, it spawned a medical mystery - Endpoints News

Of all the hardships on Little House on the Prairie, this Mary Ingalls (Melissa Sue Anderson) going blind was one of the saddest. When it comes to how the real Mary Ingalls went bind, though, the story is still a little bit muddled.

Heres what we know about the differences between Mary Ingalls blindness in the television series, the novel, and real life.

Shes the eldest of the Ingalls girls. Although shes smart and beautiful, Mary doesnt have it easy. On the television series, Little House on the Prairie, Mary Ingalls falls deathly ill in a two-part episode, needing surgery (To Live With Fear.)

As she got older, this character had dreams of becoming a teacher. After studying for hours by candlelight for the state teachers exam, and complaining that her eyes are tired, Mary gets a shocking piece of news shes going blind.

She completely loses her sight in the two-part episode, Ill Be Waving as You Drive Away. The reason for this, Charles Ingalls is told, is her scarlet fever. After that, Mary Ingalls attends a school for the blind, where she meets her husband, a teacher named Adam.

According to Little House on the Prairies website, though, in real life, Mary Ingalls never married or taught at this school. The reason for her blindness, however, may also different from the television series.

RELATED: Little House on the Prairie: Melissa Gilberts Grandfather Helped Create This Television Comedy Classic

In real life, Mary Ingalls went blind at the age of 14, her parents then sending her to the Iowa School for the Blind. The characters blindness was somewhat delayed in the television series, according to one website, for fear it would limit storyline options.

Even the reason behind Mary Ingalls blindness mightve been altered in different tellings of her story. One scientific journal states that the reason behind permanent blindness caused by scarlet fever is uncertain. It could be a postinfectious autoimmune phenomenon. Its more likely, however, that Mary Ingalls went blind for another reason.

CNN reports that Wilder wrote letters to her daughter, Rose, making reference to some sort of spinal sickness. The letter also mentions that Mary saw a specialist in Chicago who said the nerves of her eyes were paralyzed and there was no hope.

RELATED: Little House On The Prairie: What Is Melissa Gilberts Net Worth and What Is She Working On Now?

Similar to the television series, in the Little House on the Prairie novel, the author tweaks Mary Ingalls illness, making it scarlet fever. This change could be because of the common illnesses of the time period.

According to CNN, it could be because Wilder and her editors thought scarlet fever would be more relatable to her readers. Scarlet fever is mentioned in other books from the period, including Little Women and Frankenstein.

Here is the original post:
'Little House on the Prairie:' Here's the Reason Why the Real Mary Ingalls Went Blind - Showbiz Cheat Sheet

News Release

Tuesday, December 15, 2020

NIH study finds that some patients need both treatments for long-term improvements in vision

Surgical and injectable drug approaches are equally effective for treatment of bleeding inside the eye from proliferative diabetic retinopathy (PDR), according to a National Eye Institute (NEI)-supported clinical study from the DRCR Retina Network (DRCR.net). A consequence of diabetes, PDR involves the growth of new, abnormal blood vessels in the light-sensing retina. These blood vessels are prone to bleeding into the gel-like vitreous that fills the eye, causing vision loss. NEI is part of the National Institutes of Health.

Researchers compared anti-vascular endothelial growth factor (VEGF) eye injections versus removal of blood via vitrectomy surgery and laser photocoagulation. Both treatments improved central vision for the majority of participants, although approximately one-third of the participants needed both anti-VEGF injections and surgery. The findings will help guide treatment for people with bleeding in the eye from PDR. The results were published in the Journal of the American Medical Association.

This clinical trial was an opportunity to compare two commonly used treatments for vitreous hemorrhage from proliferative diabetic retinopathy head-to-head. The results provide useful guidance for clinicians who are managing patients with this condition, said Adam Glassman, Jaeb Center for Health Research, director of the DRCR.net coordinating center.

Vitrectomy surgically removes the blood from inside the eye. To prevent more blood vessels from forming, vitrectomy is usually paired with a laser treatment (laser photocoagulation). This treatment often restores central vision, but the laser treatment may reduce peripheral vision. More recently, NEI-supported trials found that anti-VEGF injections into the eye help control PDR and other diabetic eye complications. These drug treatments decrease the growth of blood vessels in the eye and prevent rebleeds while the blood in the vitreous is being absorbed.

Over the past 15 years, the DRCR Retina Network has performed trials that have helped set the standard of care for diabetic eye disease, said Jennifer Sun, M.D., Harvard University, Cambridge, Massachusetts, chair of Diabetes Initiatives for the Network. There have been major advances in surgical technology and technique since the initial studies that evaluated surgical treatment of PDR. This latest DRCR Retina Network study lets us understand how outcomes in the modern era of retinal surgery compare to treatment with intraocular anti-VEGF injections for vitreous hemorrhage from PDR.

In this new clinical study, the DRCR Retina Network team compared these two standard treatments to see if either was more effective in improving visual acuity over two years. Of 205 participants, 100 were assigned an anti-VEGF drug, aflibercept (brand name Eylea), and 105 were assigned prompt vitrectomy and laser photocoagulation. The researchers tracked best corrected visual acuity over two years. At four weeks, visual acuity in the surgery group was significantly higher than the anti-VEGF group, but by 24 weeks, the visual acuity in the anti-VEGF group had caught up to the surgery group. Visual acuity was similar between the two groups out to two years. Approximately one in three participants in each group eventually received both types of treatments to help control their PDR during that time.

This is a very common disease for patients with diabetes particularly after living with diabetes over several decades. For patients who are experiencing visual loss from bleeding due to PDR, these strategies are both excellent treatments and can improve and then preserve visual acuity over six months to two years, said Sun. But there are some subtleties to this study that will help clinicians tailor their treatment plans for an individual patient.

For people with severe bleeds or who need to improve their vision quickly, surgery results in more rapid improvement, Sun said. But for people who cannot receive surgery or prefer to avoid surgery if possible, anti-VEGF treatment still leads to equivalent visual outcomes over the long term.

About a third of our study participants ended up needing both treatments, said Glassman, so we think its critical that people continue to follow up with their clinicians after starting treatment for PDR, because they may need to adjust their treatment along the way to get the best outcome.

An estimated 30 million Americans have diabetes. Blood vessel abnormalities, including the growth of new blood vessels, are common in people with diabetes. PDR can lead to retinal tissue death, permanent vision loss, and eventually blindness in some patients. Prior to the development of surgical and laser treatments to remove blood and regress abnormal blood vessel growth, PDR was a leading cause of blindness in the United States.

The Clinicaltrials.gov identifier for this study is NCT02858076. The study was supported by NEI and the National Institute of Diabetes and Digestive and Kidney Diseases through a cooperative agreement (EY14231). Regeneron provided aflibercept for the study and funds to DRCR.net to defray the studys clinical site costs.

NEI leads the federal governments research on the visual system and eye diseases. NEI supports basic and clinical science programs to develop sight-saving treatments and address special needs of people with vision loss. For more information, visithttps://www.nei.nih.gov.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

Antoszyk AN, Glassman AR, Beaulieu WT, Jampol LM, Jhaveri CD, Punjabi OS, Salehi-Had H, Wells JA III, Maguire MG, Stockdale CR, Martin DF, and Sun JK, for the Diabetic Retinopathy Clinical Research Network. Effect of Intravitreous Aflibercept Versus Vitrectomy with Panretinal Photocoagulation on Visual Acuity in Patients with Vitreous Hemorrhage From Proliferative Diabetic Retinopathy: A Randomized Clinical Trial. JAMA. Dec 15, 2020

###

See the original post:
Surgical and drug treatment options lead to similar outcomes for diabetic eye disease - National Institutes of Health

In a development that could restore sight to thousands of people with an inheritable condition calledLeber's Hereditary Optic Neuropathy (LHON), scientists have been able to improve vision in both eyes in a majority of patients even though only one eye was actually treated.

The treatment is an experimental type of gene therapy, where tailored genetic material is injected to counteract genes that are defective or malfunctioning. With LHON, a m.11778G>A mutation in the MT-ND4 gene is the target.

In a phase 3 clinical trial, 37 patients were treated with a modified viral vector rAAV2/2-ND4 in one eye only, leading to an average vision improvement of 15 letters on the standard ETDRS chart you might have spotted at an optician's clinic.

"We expected vision to improve in the eyes treated with the gene therapy vector only," says neuro-ophthalmologist Patrick Yu-Wai-Man, from the University of Cambridge.

"Rather unexpectedly, both eyes improved for 78 percent of patients in the trial following the same trajectory over two years of follow-up."

The eyes that didn't get the gene therapy were given a sham treatment instead, and while the improvement wasn't as great, it was still substantial. Those in the earlier stages of LHON typically saw a bigger improvement in their vision from the treatment.

LHON is the most common form of mitochondrial blindness transmitted from a mother to her children and attacks the retinal ganglion cells, damaging the optic nerves. Around 1 in 30,000 people are thought to be affected, usually men in their 20s or 30s.

The replacement MT-ND4 gene treatment seems to rescue the retinal ganglion cells from their fate, causing results that can be "life-changing" according to the researchers. Normally less than 20 percent of those affected get their sight back.

"As someone who treats these young patients, I get very frustrated about the lack of effective therapies," says ophthalmologist Jos-Alain Sahel, from the University of Pittsburgh.

"These patients rapidly lose vision in the course of a few weeks to a couple of months. Our study provides a big hope for treating this blinding disease in young adults."

While scientists know what causes the loss of vision, finding a way to stop it has proved difficult. LHON is a good candidate for gene therapy though, because it has a clear starting stage and genetic targets that are relatively straightforward to hit.

What's not clear yet is why and how the gene therapy is spreading from one eye to the other. Follow-up experiments in macaque monkeys, which have vision systems similar to humans, suggested the injected viral vector can spread to other tissue via some means of interocular diffusion, but more research is going to be needed to understand the mechanisms at work.

Gene therapy is now being used to tackle a wide range of diseases and health issues, including those inherited from parents. Many other eye problems are in the sights of researchers too, and advances in one area can quickly help research in another something that the team behind the current study is excited about.

"Our approach isn't just limited to vision restoration," says Sahel. "Other mitochondrial diseases could be treated using the same technology."

The research has been published in Science Translational Medicine.

See the rest here:
Experimental Therapy Injected in One Eye Unexpectedly Improves Vision in The Other - ScienceAlert

Lineage Cell Therapeuticsis a clinical-stage biotechnology company developing novel, off the shelf cell therapies for unmet medical needs. Lineages programs are based on its proprietary cell therapy platform and in-house development and manufacturing capabilities. Their lead program features the manufacture and transplant of retinal pigment epithelium cells for the treatment of dry age-related macular degeneration, one of the leading causes of blindness. The therapy recently completed enrolment in a Phase 1/2a clinical trial.Here we share a Q&A with CEO Brian Culley. In this interview, Brian discusses age-related macular degeneration and retinal pigment epithelium transplant therapy.Q: What is age-related macular degeneration and what is the current treatment landscape?A: Age-related macular degeneration (AMD) is a leading cause of blindness in adults over the age of 50. Progression of the disease occurs as a result of the death of specialized retina cells in the area of the macula, which gradually leads to a decrease or complete loss of vision, particularly affecting the central vision, which is needed to use a cell phone, recognize faces, drive a car, etc. The causes of AMD are multifactorial and include age, genetics, smoking, and environmental influences. There are two forms of AMD. Dry AMD is the more common of the two types, accounting for approximately 85-90% of cases, while wet AMD is the less common, accounting for approximately 10-15% of cases.Wet AMD, or neovascular macular degeneration, occurs due to an abnormal growth of blood vessels beneath the macula, resulting in spots in the visual field or straight lines appearing wavy to the patient. Unlike dry AMD, which develops slowly, wet AMD can rapidly progress and cause a loss of central vision. There are a number of effective U.S. Food and Drug Administration (FDA) approved treatment options available for wet AMD. These treatments are designed to stop the development or growth of blood vessels and are known as antiangiogenic or "anti-vascular endothelial growth factor (VEGF) therapies. Anti-VEGF therapies can slow the progression of wet AMD and in some cases improve the patients vision.

In the early to intermediate stages of dry AMD development, retinal cells have died off and as a result, yellow deposits of metabolic waste products known as drusen begin to accumulate in the back of the eye. Drusen can vary in size and number and are considered a natural part of the eyes aging process. However, in dry AMD, the retinal pigment epithelium (RPE) cells are dysfunctional or dying and unable to clear the metabolic waste. Over time, the disease will inevitably progress, sometimes into advanced dry AMD with geographic atrophy (GA), meaning total and irreversible loss of cells in an area. There are currently no FDA or European Medicines Agency approved treatment options for dry AMD. This is likely because dead cells cannot be restored. Humans lack the ability to regenerate retinal tissue and replace lost retina cells which has led to a presumption that progression of GA may someday be slowed or halted but cannot be reversed.Q: Could you tell us about the allogeneic RPE cell replacement therapy Lineage is developing for the treatment of dry AMD with GA?A: One of the most promising potential therapies for advanced dry AMD with GA is cell replacement therapy, because cell therapy means transplanting cells which cannot be generated any other way. Most traditional approaches seek to slow or halt a disease process, but cannot reverse it. Cellular therapies are often aimed at regenerating or replacing absent cells and/or improving bodily functions. Therefore, cellular therapies may be shown to have broader or more suitable applicability than pharmaceuticals in the treatment of advanced dry AMD and other serious diseases. The benefits to the patient include drusen reduction, photoreceptor recovery and preserved or improved sight. At Lineage Cell Therapeutics we are exploring these potential benefits through the clinical development of an RPE cell replacement therapy called OpRegen. OpRegen is a formulation of allogeneic RPE cells, designed to ultimately be used as an off-the-shelf or thaw-and-inject treatment for advanced dry AMD with GA. That means were generating the cells outside of the body from established cell lines. The hypothesis of this therapy is that newly transplanted, healthy RPE cells may replace the patients dead RPE and support dysfunctional or dying RPE that would otherwise continue to progress to death. This is similar to a bone marrow transplant, except it is happening in the eye. The objective of OpRegen therapy is to slow, halt or even reverse the progression of dry AMD with GA. OpRegen is currently in a Phase 1/2a dose escalation safety and efficacy clinical study of a single injection of transplanted cells in patients with advanced dry AMD with GA. The primary objective of the study is to evaluate the safety and tolerability of OpRegen, while secondary objectives are to evaluate the preliminary efficacy of OpRegen treatment by assessing the changes in ophthalmological parameters, such as visual acuity and reading speed.We recently reported evidencefrom a patient with atrophic end-stage disease, who received a transplant of allogeneic RPE cells and showed substantial restoration of retinal tissue within the area of GA. Specifically, the area of GA assessed at 9 months was approximately 25% smaller than the patients pre-treatment baseline and it grew approximately 50% slower than its historical rate during the subsequent six months. This patients area of GA remained below baseline for 23 months and counting. Lineage is not aware of any other company or academic lab which has shown such a dramatic reversal of the disease in a human. These unprecedented findings were initially observed by an independent external advisor using multiple imaging technologies and were subsequently confirmed by the reading center and additional experts in the field of retinal imaging.To our knowledge, this is the first time any experimental treatment for dry AMD has demonstrated a reduction, rather than expansion, of an area of atrophy over a clinically meaningful time period.This finding supports the view that dry AMD is not an irreversible, degenerative condition and that some portion of diseased retinal tissue may be recoverable in atrophic end-stage disease patients.Q: What are the manufacturing capabilities at Lineage, and could you touch on the purity of the RPE cells?A: As we aim to pioneer a new branch of medicine, based on transplanting specific cell types into the body, relying on our competitive advantage of differentiating an unlimited supply of pluripotent stem cells into specialized cell types like retinal cells. Lineage has extensive experience directing the lineage of pluripotent cells into terminally differentiated, specialized cell types and scaling them up in preparation for commercialization. OpRegen cells are greater than 99% pure RPE cells manufactured from a pluripotent cell line that is NIH approved and established 20 plus years ago. The cells have undergone extensive characterization and karyotyping, and there are no genetic modifications made to the RPE cells. Commercial scale-up has already been achieved, with a current production scale of around 5 billion cells per 3-liter bioreactor, or the equivalent of greater than 2,500 clinical doses per batch. Further scale-up can be performed in larger or additional (i.e. parallel) reactors.

Read the original here:
Cell Replacement Therapy A Promising Treatment for Age-Related Macular Degeneration - Technology Networks

Taljat David/Shutterstock

On Tuesday, Novartis announced the first interpretable results from its Phase III KESTREL study of humanized single-chain antibody fragment (scFv) Beovu, demonstrating significant improvement in central subfield thickness in Diabetic Macular Edema (DME).

DME occurs when consistently high blood sugar levels cause damage to small blood vessels in the eye, resulting in an accumulation of leaked fluid, or edema, in the macula, a pigmented area near the center of the eye crucial for the human brain to process light and register a sharp, detailed picture. DME is the leading cause of blindness among young adults in developing nations, 40% of whom have either type 1 or type 2 diabetes.

Beovu (brolucizumab, RTH258), is the most clinically advanced humanized scFv and is currently approved in more than 50 countries, including the U.S. and the EU for the treatment of wet AMD (macular degeneration), a condition that causes blurred vision or a blind spot in the visual field.

According to Suying Dang et al in the journal Hybridoma, single-chain variable fragment of antibody (scFv Ab) provides many advantages over monoclonal antibodies, especially for therapeutic purposes, including enhanced tissue penetration due to their small size and abrogated immunogenicity.

In the KESTREL study, Beovu met the primary endpoint of non-inferiority to aflibercept 2 mg in change in best-corrected visual acuity (BCVA) at the one-year mark and demonstrated significant improvement at the 6 mg does in the change of central subfield thickness (CST) from baseline between weeks 40 and 52.

The results affirm the positive topline results Novartis announced in September from its pivotal KITE Phase III study of Beovu with DME.

Central subfield thickness [CST] is a key indicator of fluid in the retina, which is an indicator for active disease. For many DME patients, frequent anti-VEGF injections are needed to reduce the fluid in the eye and control the disease. The data from KITE and KESTREL suggest Beovu may have the potential to provide DME patients with better disease management through extended dosing intervals resulting in less overall injections, Dirk Sauer, Global Development Unit Head Opthalmology, told BioSpace.

Beovu also met its secondary endpoint, as more than half of participants in the 6 mg arm were maintained on a three-month dosing interval through year one, after the loading phase. Overall, the study showed that Beovu was well-tolerated.

Novartis is interested in determining whether the same level of efficacy can be achieved with an extended dosing interval. The Phase III KITE and KESTREL trials were designed to show non-inferiority in change from baseline in best-corrected visual acuity. The studies looked at Beovu 6 mg on a flexible dosing regimen, allowing for three and two-month dosing intervals versus aflibercept 2 mg on a two-month dosing interval. More than half of Beovu patients in the 6 mg arm were maintained on a three-month dosing interval through year one, following the loading phase, Sauer said. These data suggest Beovu may have the potential to provide DME patients with better disease management through extended dosing intervals, which could ultimately lead to better treatment adherence.

Sauer also stated that Novartis intends to submit the data from both KITE and KESTREL to health authorities in H1, 2021 and looks forward to working with regulators worldwide to make Beovu available to DME patients in need.

Most Read Today

More here:
Novartis' KESTREL Study Reaffirms Positive Results of Beovu in DME - BioSpace

Photo by Brandon Kopp

From an email:

Washington National Cathedral will toll its bells 300 times on Tuesday, Dec. 15 starting at 5 p.m. ET, in memory of the 300,000 Americans who have died of COVID-19.

The Very Rev. Randolph Marshall Hollerith, the Cathedrals dean, also released this statement:

We have reached the point in America where the death toll from COVID-19 for just one day was the equivalent of 16 fully loaded 737 jets falling from the sky.

Think about that. We would never allow that kind of disaster to take place day after day in this country, and we certainly would never allow such tragedy to become normal or expected. And yet, the climbing death toll from this pandemic seems disturbingly routine. How awful that is.

As the Cathedral marks 300,000 lives lost to COVID-19, I have grown weary of tolling this bell. I dont want to toll this bell any more. I dont want to lose any more lives. I dont want us to think this is normal, or that it is just the price we must pay for living in a free society. God forgive us if we find ourselves tolling this bell again at 400,000 lives lost.

The Christian faith teaches that each person is a beloved child of God, and that my well-being is deeply connected to your well-being. We are not lone individuals free from responsibility; rather, we are dependent upon one another for our very lives and commanded to love our neighbors as ourselves.

There are simple things we can do wear a mask, keep our distance, adjust our holiday plans to show our mutual respect and concern for one another. Yes, we are tired from the confines and struggles of this pandemic, and yes, its been a long nine months. But now, more than ever, we have to protect each other because there has been far too much death. A vaccine is coming and we will get through these difficult days, but we will only succeed if we do it together.

Lord, in your mercy, heal us from this disease. Remove the blindness from our eyes that keeps us from seeing your face in our neighbor. Soften our hearts, give us strength to endure and the will to act for the common good.

Lord, in your mercy, hear our prayer.

Originally posted here:
Washington National Cathedral will toll its bells 300 times starting at 5 pm "in memory of the 300000 Americans who have died of COVID-19."...

DUBLIN, Dec. 14, 2020 /PRNewswire/ -- The "Vision Care: Global Markets" report has been added to ResearchAndMarkets.com's offering.

The current report provides a detailed analysis of the vision care devices market. This report will highlight the current and future market potential of vision care devices along and provide a detailed analysis of the competitive landscape. The report covers market projections for 2025 along with current market estimates. Epidemiology trends of major eye disorders, drivers, restraints, and opportunities will also be covered in the current report along with company profiles of the key market players.

The report segments the market for vision care devices based on device, distribution channel, and geography. Based on device type, the market is broadly segmented into eyeglasses, contact lenses, intraocular lenses (IOLs), and artificial tears. Eyeglasses are further segmented into a single vision, bifocal and progressive. Contact lenses are further segmented into spherical lenses, toric lenses, multifocal contact lenses, and others. For market estimates, data will be provided for 2019 as the base year, with estimates for 2020 and forecast values for 2025.

By geography, the market has been segmented into North America, Europe, the Asia-Pacific region, Latin America, and the Middle East and Africa. Detailed analysis of major countries such as the U.S., Canada, Germany, the U.K., Italy, France, Spain, Japan, China, India, Australia, Brazil, and Mexico will be covered in the regional segments.

The Report Includes:

Market growth is attributed to factors such as the growing prevalence of blindness and vision impairment, a rise in the world's geriatric population, and growing collaborations between vision care companies and technology companies for the advent of technologically advanced products to address current unmet needs.

According to the IAPB, nearly 1.1 billion people are considered to be affected by some form of vision loss: 43 million people are categorized as blind, about 553 million people are estimated to suffer from some form of moderate to severe visual impairment (MVSI), about 258 million people experience mild vision impairment and about 510 million suffer from near vision impairment. In addition, at least 1 billion more people need ongoing access to eye care services. High prevalence and the increasing burden of vision impairment is likely to drive the vision care market during the forecast period.

Reasons for Doing This Study:

Visual impairment is a global health concern that has a negative impact on both physical and mental wellbeing. Preventive blindness, a common vision disorder, represents a major challenge to the global healthcare system. Visual impairment is associated with loss of global productivity and high disability-adjusted life years (DALYs) and is responsible for significant healthcare spending. According to the International Agency for the Prevention of Blindness (IAPB), in 2020, nearly 1.1 billion people were considered to be affected by some form of vision loss; 43 million people are categorized as blind, about 553 million people are estimated to suffer from some form of Moderate and Severe Vision Impairment MSVI, about 258 million people experience mild vision impairment and about 510 million suffer from near vision impairment. In addition, at least 1 billion more people need ongoing access to eye care services.

Globally, the leading causes of vision impairment are uncorrected refractive errors and cataracts. Refractory disorders, particularly myopia, are one of the common ailments seen across all age groups. The incidence of refractory disorders is estimated to have doubled in the last decade compared to past generations, with the rise driven by rapid changes in lifestyles, genetic disorders, and malnutrition. Refractory disorders, although increasingly prevalent, can be treated and are considered preventable ailments if addressed quickly.

Market growth has been spurred by the introduction of technologically advanced products and the rise in the global visually impaired population. This report is designed to provide the reader with a background on vision care products, an analysis of the current factors influencing the market, and the tools to make decisions regarding expansion and penetration in this market.

Key Topics Covered:

Chapter 1 Introduction

Chapter 2 Summary and Highlights

Chapter 3 Clinical Overview

Chapter 4 Epidemiological Insights

Chapter 5 Supply Chain Overview

Chapter 6 Vision Care Market Dynamics

Chapter 7 Impact of COVID-19 Pandemic on the Vision Care Market

Chapter 8 Market Breakdown by Product Type

Chapter 9 Market Breakdown by Region

Chapter 10 Competitive Landscape

Chapter 11 Company Profiles

Chapter 12 Appendix: Acronyms

For more information about this report visit https://www.researchandmarkets.com/r/k467nz

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716

SOURCE Research and Markets

http://www.researchandmarkets.com

Go here to see the original:
Insights on the Vision Care Global Market to 2025 - Increase in Uptake of a New Generation of Contacts Lenses is Driving Growth - PRNewswire

New York, Dec. 15, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Cell and Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change" - https://www.reportlinker.com/p05996809/?utm_source=GNW

The global cell and gene therapy market is expected to decline from $6.68 billion in 2019 to $6.92 billion in 2020 at a compound annual growth rate (CAGR) of 3.61%. The slow growth is mainly due to the COVID-19 outbreak that has led to restrictive containment measures involving social distancing, remote working, and the closure of industries and other commercial activities resulting in operational challenges. The entire supply chain has been disrupted, impacting the market negatively. The market is then expected to recover and reach $13.23 billion in 2023 at a CAGR of 24.10%.

The cell and gene therapy market consists of sales of cell and gene therapies by entities (organizations, sole traders and partnerships) that develop cell and gene therapies. Cell therapy refers to the transfer of intact, live cells that are originated from autologous or allogenic sources and gene therapy refers to the introduction, removal, or change in the genome for treating diseases. The market consists of revenue generated by the companies developing cell and gene therapy products by the sales of these products.

North America was the largest region in the cell and gene therapy market in 2019. It is also expected to be the fastest-growing region in the forecast period.

In December 2019, Roche, a Swiss multinational healthcare company, acquired Spark Therapeutics for $4.3 billion. The acquisition supports the commitment of Roche to bring transformational therapies and innovative approaches to people with serious illnesses. Spark Therapeutics will continue to work within the Roche Group as an independent company. Spark Therapeutics, headquartered in Philadelphia, is a fully integrated commercial company involved in the discovery, production, and distribution of gene therapies for genetic disorders including blindness, hemophilia, lysosomal storage, and neurodegenerative diseases.

The cell and gene therapy market covered in this report is segmented by product into cell therapy; gene therapy and by application into oncology; dermatology; musculoskeletal; others.

Limited reimbursements preventing patients from receiving treatments are expected to limit the growth of cell and gene therapy (CGT market. In 2019, Trinity Life Sciences, a life sciences solution provider, researched national and large regional commercial health insurance plans in the US. It found that the confluence of increasing price, patient volume and number of CGTs on the market is likely to change the reimbursement model for CGTs and impact payer budgets by 5-10%. Payers realize that financing needs to be generated for cost management due to the uncertainty surrounding reimbursement of ancillary costs. Limited reimbursements and uncertain insurance plans are preventing patients from receiving high-cost CGT, which is expected to limit market growth.

Chimeric antigen receptor (CAR) T-cell therapy is shaping the cell and gene therapy (CGT) market. (CAR) T-cell therapy is a combination of cell and gene therapy in which T cells are collected from the patients blood and are genetically engineered to produce modified receptors at their surface, known as chimeric antigen receptors (CARs). These modified T cells with special structures (receptors) are reinfused into the patient. Then, the modified receptors of T cell help in targeting the surface antigen of the cancer cell that ultimately results in the killing of tumor cells in patients. In 2020, the US-FDA approved Bristol-Myers Squibbs two CAR-T cell therapies to treat lymphoma and multiple myeloma and is set to be launched. Currently, FDA approved CAR-T cell therapy treatments like Tisagenlecleucel for the treatment of B-cell precursor acute lymphoblastic leukemia (ALL) in children and Axicabtagene ciloleucel for the treatment of adult patients with relapsed or refractory large B-cell lymphoma.

Steady investment and consolidation in cell and gene therapies contributed to the growth of the cell and gene therapy (CGT) market. After recognizing the potential of the CGT market, 16 out of the 20 largest biopharma companies by revenue, added CGT products to their portfolio. For instance, Merck invested $109 million in viral vector and gene therapy manufacturing in April 2020. Moreover, 12% of industrial clinical pipeline products and at least 16% of preclinical pipeline products consist of CGT. Steady investment and consolidation in CGT production capacity led to an increase in production capacity and also contributed to the growth of the market.Read the full report: https://www.reportlinker.com/p05996809/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

__________________________

View original post here:
Cell and Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change - GlobeNewswire