StemCell Therapy

Like a plate of poisoned cookies from Grandma, cancer could be coming from an unlikely place. Increasingly, some research is pointing to stem cells, usually thought of as a promising disease cure-all.

The term "stem cells" covers any cells capable of perpetually growing more of themselves. Most often, people refer to pluripotent, or embryonic, stem cells, which have the ability to become any cell in the body. But there are also adult stem cells, which are more limited in the cells they can create.

Now, according to some researchers, there are also tumor stem cells.

"They aren't the same thing as regular stem cells," said Dr. Allan Mufson, chief of Cancer Immunology/Hematology Branch Division of Cancer Biology at the National Cancer Institute. "But there seems to be a small population of cells within tumors that are responsible for keeping the tumor going. They're the only cells that can give rise to new tumors."

According to the tumor stem cell theory, the problem with common cancer treatments (chemotherapy and radiation) is that they focus on the whole tumor, when it's only the rare tumor stem cells that really matter. Doctors tend to use large doses of potentially deadly medications that weaken patients and aren't specifically aimed at killing tumor stem cells, increasing the risk that they'll be missed and the cancer will grow back.

This new theory of cancer is being studied, but questions still remain. One of the big ones: Where do tumor stem cells come from? Dr. John Kersey, a researcher at the University of Minnesota's Masonic Cancer Center, thinks he's found an answer: Tumor stem cells may be damaged versions of normal, potentially life-saving adult stem cells.

Adult stem cells come in two flavors: The highly specialized progenitor stem cells and an immature variety that are more flexible. For instance, a progenitor cell might only be able to grow white blood cells, while the immature adult stem cell could grow several different cells in the circulatory system. Researchers are still debating which type becomes a tumor stem cell, but Kersey's findings, detailed in the May issue of the journal Cancer Cell, suggest it is the immature adult stem cells, at least for certain types of leukemia.

To figure that out, Kersey and his team grew mice whose adult stem cells, both progenitor and immature, contained a gene that causes leukemia. Both types were then separated out and injected into healthy mice. The mice who got progenitor cells didn't get leukemia. The ones who got the immature adult stem cells did.

While this doesn't completely prove stem cells are the culprit behind leukemia, it does go a long way towards showing that low doses of cancer genes can transform a stem cell from something that creates life to something that takes it, Mufson said. Cancer researchers say that figuring out where tumor stem cells come from is the first step in turning them back into useful tools for health. If damaged stem cells really are the building blocks behind tumors, doctors might be able to figure out a way to target those cells, or even just a part of them, leading to safer and more effective cancer treatments.

"This is exciting stuff," Kersey said. "Understanding this will be essential to developing specific treatments for specific cancers, based on the part of the cancer that's actually growing."

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Stem Cells Might Cause Cancer - livescience.com

June 23, 2017 Assistant Professor Xu Jian (right) and Dr Hong Jing Han (left), together with their research team at the National University of Singapore, found that the Arabidopsis (in photo) may selectively kill part of their roots to survive under cold weather conditions. Credit: National University of Singapore

Plants adopt different strategies to survive the changing temperatures of their natural environments. This is most evident in temperate regions where forest trees shed their leaves to conserve energy during the cold season. In a new study, a team of plant biologists from the National University of Singapore (NUS) found that some plants may selectively kill part of their roots to survive under cold weather conditions.

This approach allows the plants to withstand chilling stress and to recover faster when the weather turns better. The discovery and understanding of this survival approach could pave the way for the development of novel strategies to improve the growth and yield of crops that undergo such environmental stress.

The study, led by Assistant Professor Xu Jian from the Department of Biological Sciences at the NUS Faculty of Science, was carried out using a small flowering plant called thale cress, known scientifically as Arabidopsis. This plant is a member of the Brassicaceae family, and its relatives include mustard greens, cabbage and kale.

The research was carried out in collaboration with scientists from the Novosibirsk State University, and the findings were reported in the online edition of the journal Cell on 22 June 2017.

Sacrificial mechanism of plants

Studies have shown that temperature can induce damage in the deoxyribonucleic acid (DNA) of plant cells, and has a profound effect on plant development and growth. However, its effects on plant stem cell behaviour and activity are still not well understood.

"The study of plant roots has been largely neglected by agricultural researchers in crop improvement until recently. Examining roots is important as they serve as the major interface between a plant and its soil environment, and are responsible for water and nutrient uptake - both resources which are critical for a plant's survival," said Asst Prof Xu, who is also from the Centre for BioImaging Sciences at NUS.

The research team conducted experiments on the roots of Arabidopsis, a plant often used as a "model organism" in plant biology and had its genome fully sequenced in 2000. To investigate the effect of chilling temperature on root development and growth, the team used the Arabidopsis root stem cell niche as an experimental model to perform in-depth studies at high spatial and temporal resolutions. The use of such experimental models provides deep insights on the survival strategies that plants employ when the odds are against them.

The research team found that a chilling temperature of four degree Celsius leads to DNA damage in the root stem cells of the Arabidopsis, as well as their early descendants. However, only the columella stem cell daughters die preferentially, and the death of these daughter cells allows maintenance of a functional stem cell niche. On the other hand, inhibition of the DNA damage response in these daughter cells prevents their death. Yet, this increases the probability that the other stem cells in the root stem cell niche will die due to the cold, leading to the plant's death.

Dr Hong Jing Han, who is the first author of the study, elaborated, "The sacrificial mechanism improves the root's ability to withstand other low temperature-related stresses. When optimal temperatures are restored, the plant stem cells can divide at a faster rate, which will in turn enhance recovery and survival of the plant." Dr Hong carried out the research as part of her doctoral thesis under the supervision of Asst Prof Xu.

Engineering cold tolerance in plants

"Our discovery of how the Arabidopsis plant slays its columella stem cell daughters shed light on the plant's unique strategy to survive harsh weather conditions, and demonstrates that the potential of engineering cold tolerance in plants to help them withstand harsh environmental conditions. The ability to do so will certainly allow farmers to extend the growing season of crops and the land area in which to grow them, increasing both yield stability and production capacity," said Asst Prof Xu.

The next step for Asst Prof Xu and his team would be to uncover the gene regulatory network that has underpinned the successful adaptation of plants and their stem cells to cold environments.

Explore further: The origin of stem cells

More information: Jing Han Hong et al. A Sacrifice-for-Survival Mechanism Protects Root Stem Cell Niche from Chilling Stress, Cell (2017). DOI: 10.1016/j.cell.2017.06.002

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Plants sacrifice 'daughters' to survive chilly weather - Phys.Org

By John Stanton

(June 22, 2017)He led what many would call a charmed life.The son of a prominent Cleveland businessman, he played hockey at Harvard, served in theU.S. Navy, and started down the road to a life in banking. Then came a day when he was struck by a disease called retinitis pigmentosa.By the time he was 30 years old, Gordon Gund was blind.

To the casual observer, Gunds blindness has hardly slowed him down. He was a successful businessman with a summer home on Nantucket. He raised a family. He owned several sports teams, including the Cleveland Cavaliers. Through it all he has fought to find a cure for the disease that took his sight.

Gordon is past the point of no return with his blindness, filmmaker Tom Scott said. But it is almost impossible to stop his passion for finding a cure.

Gund is the subject of Scotts 20-minute documentary profile,The Illumination. The film will play at the Nantucket Film Festival Friday at 4:45 p.m. at the Dreamland Theater. It will be followed by a conversation between Gordon Gund, his wifeLulie and Scott.

To read the complete story, pick up the print edition of this weeks Inquirer and Mirror or register for the I&Ms online edition byclicking here.

For up-to-the-minute information on Nantuckets breaking news, boat and plane cancellations, weather alerts, sports and entertainment news, deals and promotions at island businesses and more, Sign up for Inquirer and Mirror text alerts.Click Here.

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NFF doc examines one man's refusal to give in to blindness - Nantucket Island Inquirer

Telemedicine is not a new concept. Physicians in Australia were using two-way radio to treat patients in rural Australia in the 1920s. Almost a century later, glaucoma is going remote.

In the glaucoma clinic of the future, patients will be checking their own eye pressure, predicted Louis Pasquale, MD, professor of ophthalmology, Harvard Medical School, and director of Glaucoma Service and Teleretinal Program, Massachusetts Eye and Ear Infirmary, Boston. They will be doing their own visual fields and imaging their own discs. This will convert the glaucoma clinic to focus on the patients who really need to be seen.

Dr. Pasquale moderated New Horizons in Telemedicine session and set the scene for changes that are already underway. The session was part of the New Horizons Forum at the 2017 Glaucoma 360 meeting.

Teleglaucoma is feasible and can play a major role in blindness prevention, said Lama A. Al-Aswad, MD, MPH, associate professor of ophthalmology, Columbia University College of Physicians and Surgeons, New York. Telemedicine and teleglaucoma are going to be an important part of how we take care of patients in the United States and worldwide.

Telemedicine is practicing medicine over a spatial or temporal distance by using electronic communications, Dr. Aswad explained. Glaucoma is an ideal candidate for telemedicine because patients tend to be older and less mobile. The disease is chronic and the technology exists for remote screening, diagnosis, and treatment.

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Teleglaucoma redefining role in future of blindness prevention - ModernMedicine

LE MARS | Hours before Marty Kurth won his 500th game as a baseball coach at Gehlen Catholic High School in Le Mars, he walked through his house with a black fungo bat, the kind he's used thousands of times to hit fly balls and grounders to his players.

"I use the bat as my cane," Kurth said. "It helps me get around the house."

Coach Kurth is going blind. In layman's terms, he has suffered a stroke in each eye the past 11 months, resulting in a sudden loss of blood flow to the optic nerve. The first stroke, which afflicted his left eye, happened on July 25, 2016. The stroke to his right eye took place on June 3, just 19 days ago.

Kurth is still coaching, doing so from the dugout, not in his coaching box on the field. He relies on assistant coaches Solomon Freking and Ty Kurth (his son) and Jays players such as Cooper Davis to describe action on the field. The Jays won 10-0 at Hartley-Melvin-Sanborn on Monday night, giving Kurth his 500th victory. With that victory level and a pair of state championships (1995 and 1999) among his six state tournament appearances, the Westmar College graduate is a lock for a spot in the Iowa Baseball Coaches Association Hall of Fame.

The accolades matter little right now, if they ever did. Kurth remains focused on his 2017 team, a club that began the season 0-4 and has ripped off 11 wins in the last 13 games. When he's not studying lineups or opponents, he's pondering a somewhat uncertain future, one that for the first time in his adult life doesn't include teaching or coaching full-time, as he recently resigned.

"I was at the point of my career where I thought maybe after next year I'd retire," said Kurth, a native of Remsen and a second-baseman on Remsen St. Mary's state championship baseball team in 1983. "Now what? I have no idea what the good Lord has planned for me."

Kurth hasn't been one to run from challenges in the past. A physical education teacher who was toiling as Gehlen athletic director several years ago, Kurth was charged with finding a head coach to direct the girls' basketball program. When his search turned up empty, Kurth told school officials he'd lead the team for a maximum of two years.

"I ended up coaching eight years," he said.

Not only that, Kurth piloted the 2012 Jays basketball team to the school's first state basketball tournament. And, he surpassed the 100-win total, all for a guy who was awfully "green" when it came to high school girls' basketball.

The news of his failing eyesight came as a shock to me. I didn't realize it until Barry Poe mentioned it in a Sunday story in the Journal, a wrap-up of Gehlen's title in the J-Club Tournament on Saturday. I was there that day and saw Kurth sitting in the dugout, an oddity for a hands-on coach who was always prepping the field and his players for another game.

"When I lost my vision in my left eye in July 2016, I woke up that morning and closed my right eye and could not see myself in the mirror," he said.

He began worrying at that point, not only about his left eye, but his right eye, too. Kurth's sister, Cheri Hoebelheinrich, who resides in Florida, lost vision in one eye when she was 37. She lost the vision in her other eye one decade later. Kurth's father, who died at age 56, began losing vision in one eye at age 37, too.

"We hoped that after I lost the one eye that I'd have time, like maybe 10 years," Kurth said. "But not even 11 months later, I woke up on June 3 and knew something wasn't right."

Kurth hit infield to his Jays that weekend in the CYO Classic, which played out on fields in Carroll and Glidden, Iowa. Before the second game at Glidden, a 10-0 victory over St. Edmond High School of Fort Dodge, Kurth had trouble catching a toss from his catcher as he hit ground balls. It's the kind of catch he's made a million times, second-nature.

"I couldn't see the ball," he said.

Jen and Marty Kurth visited the Truhlsen Eye Institute at the University of Nebraska Medical Center two days later. Doctors there identified the cause, the same affliction that struck his left eye last July: non-arteritic ischemic optic neuropathy, or "NAION" for short.

"There's no cure," he said. "It's what my sister had, too."

Jen said that while the condition isn't genetic, it can be familial. Researchers continue to study it. The Kurths continue to pray.

Marty Kurth tried to qualify for a "NAION" study, but his participation was ruled out because he has too many red blood cells.

"We got opinions from Duke University and Johns Hopkins Medicine and they didn't want to give me the medication in the study because they didn't know what the ramifications might be with my blood disease," he said.

Jen Kurth, who works in the business office at Floyd Valley Healthcare in Le Mars, said that "NAION" typically affects smokers, diabetics and those with high blood pressure. Marty, she noted, fits none of those descriptors.

Marty Kurth said he can currently see a little out of the upper right hand corner of his right eye. He also has some peripheral vision in his left eye. "I told Ty that if you closed your eyes so that your eyelids were touching and you tried to see, that's kind of what it's like for me right now."

He hasn't lost his sense of humor, though, and it showed on Saturday as the Jays battled Newell-Fonda. When Gehlen pitcher Collin Buden got ahead in the count before hitting one batter and walking the next, the old head coach became anxious on Saturday: "I hollered out to the mound and said, 'Alex, don't make me come out there. You know, I will find you!'"

The players and Budden got a kick out of it, their longtime coach making the best of a difficult, life-changing predicament.

Kurth knows he's fortunate to have Jen, their children Kendra, Mitchell and Ty, and Jen's parents offering love and support, as well as a world of friends and current and former Gehlen students, players and competitors throughout Plymouth County and Northwest Iowa.

"I'm 52," the Gehlen legend said. "I feel good. The good Lord has a plan. We hope to find out what it is soon."

In the meantime, researchers will continue to work, as will the baseball players sporting the Gehlen green and gold. And the wise, old coach in the dugout? He'll lean on his fungo, listening, feeling, smelling for the optimum time to call a pitch-out or a hit-and-run. Maybe Marty Kurth is becoming visionary, in a figurative sense.

"My daughter wanted to make a shirt after Monday's victory," he said. "It was going to say, '500 wins. Not so hard. My dad did it. The last six with his eyes closed.'"

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Le Mars baseball coach rallies while battling blindness - Mason City Globe Gazette

Boston Business Journal

Watertown's pSivida files for European approval of treatment to ... Boston Business Journal After unveiling positive data last week from a late-stage trial of its experimental, long-acting treatment for a leading cause of blindness, Watertown-based pSivida ... pSivida Submits Marketing Authorization Application (MAA) for ...Nasdaq all 3 news articles »

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Watertown's pSivida files for European approval of treatment to ... - Boston Business Journal

Eli Lilly has added a new state-of-the-art laboratory and 180,000 square feet of additional working space to its Biotechnology Center in San Diego, CA. Reporting completion of the $90-million expansion, Lilly said the 145% increase in working space will help to foster collaborations with external partners, and allow closer partnerships between its biotechnology, discovery chemistry, and research technology teams. The aim is to accelerate drug discovery within therapeutic areas including immunology, diabetes, oncology, neurodegeneration, and pain.

To further exploit its expertise in automated organic synthesis, and build on its Automated Synthesis Laboratory in Indianapolis, Lilly is also establishing the Lilly Life Science Studio at the San Diego site. The firm says the new facility will give researchers worldwide the ability to remotely test new ideas, and design, synthesize, and screen molecules.

"Investing in drug discovery and development is critical to maintaining an ecosystem that encourages and promotes innovation, commented Jan Lundberg, Ph.D., executive vice president for science and technology and president of Lilly Research Laboratories. "Expanding our presence in San Diego will not only help us discover and deliver innovative medicines faster, but will also help us achieve our goal of launching 20 new medicines in 10 years."

"This year we announced a commitment to invest $850 million in our U.S. operations based on our potential for growth and the company's long-standing investment in the U.S. market, added David A. Ricks, Lilly's chairman, president, and CEO. "This investment doesn't come without risk. America's biopharmaceutical leadership is driven by a free-market economy that rewards innovation. Today, there are multiple public policy threats to our business that would discourage or reduce our investment in the U.S. and the state. We are committed to working with policymakers and stakeholders to ensure our efforts to deliver new innovative medicines to patients are not threatened."

Lilly set up the San Diego Biotechnology Center, which is sited close to the University of California, San Diego, in 2009, having acquired Applied Molecular Evolution back in 2004.

Earlier this month Lilly reported that it will acquire a pipeline of dual amylin calcitonin receptor agonists (DACRAs) from KeyBiosciencefor the potential treatment of type 2 diabetes and other metabolic disorders.

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Lilly Completes $90M Expansion of San Diego Biotechnology Center - Genetic Engineering & Biotechnology News

Kansas City Star

Shawnee Mission West High student wins international biotechnology competition Kansas City Star Hosted by the Biotechnology Institute, the competition challenges high school students from across the world to find solutions to health care, sustainability and environmental needs through biotechnology. Earlier this year, Smith was chosen along with ...

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Shawnee Mission West High student wins international biotechnology competition - Kansas City Star

A publication in Science Translational Medicine shows that Roche has a rising star in the 15 million patient market of age-related macular degeneration (AMD). In a Phase II trail US and German researchers showed efficacy in geographic atrophy, an advanced stage of AMD, which has currently no treatment.

One week prior to the publication, Roche announced it has intitiated two Phase III trails (CHROMA and SPECTRI) enroling 936 patients with the advanced form of AMD that affects 5 million AMD patients and has currently no cure. Primary endpoint is slowing for disease progression at 12 months, secondary endpoint is visual acuity at 24 months. However, rumors say the FDA could accelerate patient access through granting breakthrough status to the treatment.

In a multi-center, randomized, 18 month Phase study that recruited 129 AMD patients ( MAHALO), lead author Brian Yaspan observed a 20% reduction in lesion area progression in patients receiving Roche/Genentechs antibody drug candidate lampalizumab at acceptable safety profile. Lampalizumab zeroes in on complement D, part of the innate immune defenses alternative complement pathway

Genome analysis of participants identified a patient subgroup with complement D variants who showed a 44% reduction in geographic atrophy area progression. The authors say targeting the alternative complement pathway has potential to be a viable treatment option for patients with secondary geographic atrophy.

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Roche's lampalizumab halts geographic atrophy - European Biotechnology

Kawartha Media Group

Peterborough biotechnology startup targeting $50 million in equity financing Kawartha Media Group Peterborough biotechnology startup targeting $50 million in equity financing. Community Jun 22, 2017 02:20 by Todd Vandonk Peterborough This Week. Share. Noblegen Inc. CEO and founder Adam Noble and CCO and co-founder Dr. Andressa Lacerda ... Noblegen Announces the Opening of Its Second Round of Financing: Peterborough-based biotechnology startup ...ForexTV.com all 4 news articles »

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Peterborough biotechnology startup targeting $50 million in equity financing - Kawartha Media Group

German oncologists have unveiled that market-approved inhibitors of DNA methyltransferases (DMNTi) and histone deacetylases (HDACi) act through expression of cancer neoantigens. They already have a biomarker test to identify responders.

The findings, reported in Nature Genetics, are good news for drug developers who want to broaden the scope of current immune checkpoint modulators through combination therapies that trigger cancer cells to release immunogenic neoantigens.

Following addition of epigenetic inhibitors to cancer cell cultures thousands of atypical transcripts with altered frameshift were expressed form previously ignored endogenous promotors of retroviral origin, resulting in profound tumour cell death, group leader Plass from German Cancer Centre told European Biotechnology. His team has already a biomarker assay that could identify responders to the treatment as it measures activation of the normally silent treatment induced, not-annotated transcription start sites (TINATs).

Up to now, pharma majors such as Roche/Genentech have largely relied on combination of their checkpoint inhibitors with personalised mRNA cancer vaccines. The new findings might open the avenue to a broader activation of cancer neoantigens than these vaccinesas over 2,500 TINATs have been identified in the human genome by the researchers. The retrotransposons (elements of human endogenous retroviruses, HERVs), which are located in long terminal repeats, have entered the human genome millions of years ago.

However, application of the findings might be hampered by the fact that TINATs, so far, have only been identified in the human genome, which might complicate preclinical in-vivo testing. Its unclear if they also exist in animals, says Plass, who is currently deciphering the exact mechanism that normally stops TINATs from being activated. First findings suggest that acetylation of transcription factors might play an important role.

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Epigenetic drugs set to boost immunoncology - European Biotechnology

The center features a new technologically-advanced laboratory and an additional 180,000 square feet of working space, which is an increase of 145 per cent compared to the former facility.

Eli Lilly and Company has announced completion of a $90 million expansion of its Biotechnology Center in San Diego, California. Lilly's new space will help foster and accelerate the discovery of medicines within the company's core therapeutic areas of immunology, diabetes, oncology and neurodegeneration, as well as the emerging area of pain.

The center features a new technologically-advanced laboratory and an additional 180,000 square feet of working space, which is an increase of 145 per cent compared to the former facility. In addition to the center's established presence in preclinical and clinical immunology research, the new space allows for closer partnership between Lilly experts in biotechnology, discovery chemistry and research technologies while also fostering external collaborations.

As a pioneer in automated organic synthesis, Lilly is creating the Lilly Life Science Studio in San Diego. Building upon Lilly's Automated Synthesis Laboratory in Indianapolis, the new facility will allow researchers across the globe to remotely design, synthesize and screen investigational molecules in an unprecedented manner. Using the power of automation, the Lilly Life Sciences Studio will shape the next generation of drug discovery and expand the reach of individual scientists to test new ideas, while reducing the cost and minimizing the environmental impact of our research activities.

San Diego has long been an important location for Lilly. In 2004 Lilly acquired Applied Molecular Evolution, Inc. before establishing the Lilly San Diego Biotechnology Center in 2009, located near the University of California, San Diego, among other prominent biomedical research institutes. Since its establishment, the center has created more than 100 jobs with more than 200 scientists currently working in various research activities.

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Eli Lilly expands Biotechnology center - BSI bureau (press release)

GETTY

Most cases of bone cancer develop in the long bones of the legs or upper arms which can cause symptoms of the disease to be mistaken for arthritis.

The most common symptoms of the disease is a persistent pain which gets worse over time, and like arthritis, can get worse at night.

Other symptoms of bone cancer can include swelling and redness over a bone which can make movement challenging.

This could also be mistaken for gout, an uncomfortable, inflammatory condition that occurs when needle-like crystals of uric acid form within certain joints or soft tissues.

Macmillan Cancer Support said another signs of the disease could be difficulty moving a joint.

GETTY

It said: If the cancer is near a joint, this can make it more difficult to move the joint. It can affect the movement of the whole limb.

If the affected bone is in the leg, it may cause a limp. If the tumour is in the spine, it may press on nerves, causing weakness or numbness and tingling in the limbs.

Other symptoms include a high temperature and unexplained weight loss.

Osteosarcoma is the most common form of primary bone cancer in children and young adults, usually affecting people aged 10 to 24 years.

However, there has not been a new treatment for osteosarcoma in almost 40 years, in spite of extensive research.

More than 160 new patients are diagnosed with osteosarcoma in the UK each year, of which around one third cannot be cured.

The current treatment for osteosarcoma is chemotherapy followed by surgery, where the bone tumours are removed.

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Leukaemia 31% - This cancer of the blood is the most common type of childhood cancer

Osteosarcoma is difficult to treat.

Now experts have revealed a subgroup of patients with osteosarcoma could be helped by an existing drug, scientists from the Wellcome Trust Sanger Institute and their collaborators at University College London Cancer Institute and the Royal National Orthopaedic Hospital NHS Trust have suggested.

In the largest genetic sequencing study of osteosarcoma to date, scientists discovered that 10 per cent of patients with a genetic mutation, in particular growth factor signalling genes may benefit from existing drugs, known as IGF1R inhibitors.

In the study, scientists analysed the genome of 112 childhood and adult tumours double the number of tumours studied previously.

Dr Sam Behjati, first author from the Wellcome Trust Sanger Institute and University of Cambridge, said: Osteosarcoma is difficult to treat.

Despite extensive research over the past 40 years, no new treatment options have been found.

In this study we reveal a clear biological target for osteosarcoma that can be reached with existing drugs.

GETTY

Professor Adrienne Flanagan, senior author from the Royal National Orthopaedic Hospital NHS Trust and University College London Cancer Institute, said: By sequencing the whole genome of the tumours, we have unpicked the mechanism behind osteosarcoma for the first time.

Dr Peter Campbell, lead author from the Wellcome Trust Sanger Institute, said: Currently, there are no new osteosarcoma treatments on the horizon.

Genomic sequencing has provided the evidence needed to revisit clinical trials of IGF1R inhibitors for the subset of patients that responded in the past.

The mutations of patients tumours may enable clinicians to predict who will, and will not respond to these drugs, resulting in more efficient clinical trials.

The drugs could be effective for 10 per cent of osteosarcoma patients.

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What is bone cancer? Symptoms could be similar to arthritis | Health ... - Express.co.uk

Takeda says it is assessing manufacturing options ahead of potential European approval later this year of the Crohns disease stem cell therapy licensed from TiGenix.

Following its acceptance for review by the European Medicines Agency (EMA), Takeda and TiGenix announced this week Swissmedic has accepted for review the file for compound Cx601, an allogeneic expanded adipose-derived stem cell (eASC) therapy for the treatment of complex perianal fistulas in patients with Crohns disease.

The therapy is being made from TiGenix site in Madrid, Spain but CEO Eduardo Bravo told Biopharma-Reporter its partner Takeda which holds the rights for Cx601 in non-US markets following a licensing agreement inked last year will take responsibility for its manufacture from 2021 from a purpose built manufacturing facility in Europe.

Takeda spokesman Luke Willats told this publication: After a transition period for technology transfer during which TiGenix will manufacture Cx601, Takeda will assume responsibility for manufacturing the compound.

But while Willats added the firm is exploring how itcan best meet manufacturing responsibilities for Cx601 following a potential European Commission (EC) approval decision for the compound in 2017, he could not comment further on specific plans or CAPEX investments.

The Japanese pharma firm has its European headquarters in Switzerland, with production sites in Austria, Belgium, Denmark, Estonia, Germany, Ireland, Italy, Norway, Poland and Russia.

Fat chance

Cx601 is produced by TiGenix in plastic flasks in incubators at a one-litre scale, using stem cells taken from healthy volunteers who have undergone liposuction for cosmetic reasons, Bravo told us.

The fat gets sent to our facility in Madrid and is processed to extract the stem cells, which account for about 2% of the material. These are placed in plastic flasks with serum to multiply the number. This is repeated until there is a large population and then the cells are frozen, creating the master cell bank (MCB).

According to Bravo, one liposuction when expanded produces upwards of 360 billion cells, enough to treat 2,400 patients.

While TiGenix is considering using bioreactors for its future pipeline, it will continue making the product as it does now due to not needing to increase volume and the challenges of making production changes in the middle or end of development.

For cell therapies, the process defines the product. Anything you change could change the cells themselves, effectively changing the product.

US deal with Lonza

TiGenix holds the US rights to Cx601 and is discussing with the US Food and Drug Administration (FDA) whether it can file using EMA data, something Bravo said would be decided in the next six-to-eight months.

US trial material will be produced by TiGenixs contract manufacturing organisation (CMO) Lonza , which is undergoing tech transfer at its site in Maryland.

But looking ahead to commercialisation, Bravo said it is not yet decided whether we continue using a CMO or build our own [US] facility.

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Takeda prepping to take over EU production of stem cell therapy from 2021 - BioPharma-Reporter.com

IANS | Mumbai June 23, 2017 Last Updated at 00:16 IST

Raising hopes of new and less painful treatment for burn injuries, a 26-year-old patient with Grade 2 burn injuries was successfully treated using stem cell therapy at a city-based hospital, doctors said on Thursday.

Anand Tiwari suffered burns after accidentally falling in a boiler unit while at work. He sustained Grade 2 and early Grade 3 burns in all parts of the body below his neck.

When admitted to the city based StemRx Bioscience Solutions hospital, he had severe burning sensation and pain all over the body. Blisters and swellings were noticed in many areas of his chest and limbs.

According to doctors, after initial care and stabilisation of the patient, for treatment of burns, a treatment protocol was prepared by Pradeep Mahajan, a regenerative medicine researcher at Stemrx Bioscience Solutions Hospital.

Explaining the treatment procedure, Mahajan said: "This involved the use of growth factors and fibroblasts and collagen based gel. These biological agents stimulate natural healing mechanisms in the body."

"The advantage of these growth factors is that they can be obtained from the patients' own body and hence are safe and effective. Additionally, unlike conventional treatment options, biological agents promote faster recovery," he said.

Under the stem cell therapy, the treatment process has to be repeated continuously so as to get rid of the problem completely and accordingly the procedure was performed.

"During the entire treatment, the patient was not given any closed dressing. He also underwent blood and supplementary fluid transfusion as required to maintain systemic homeostasis," said Mahajan.

He said that changes in the patient were observed as early as two-three days after the initiation of therapy. Drying of superficial burns began and swelling started reducing.

"Gradually, dry crusts started peeling and by the end of the third week, initial healing of most areas was complete. There was no odour or oozing from any wound and he did not complain of pain or burning sensation anymore.

"After a month-long treatment, healthy skin formation is being observed and further healing is progressing at an impressive rate," said Mahajan, adding that in treatment through conventional modalities, it takes more than eight weeks for healing to happen and further several months for patient to be able to regain joint and facial movements.

--IANS

rup/nir

(This story has not been edited by Business Standard staff and is auto-generated from a syndicated feed.)

Raising hopes of new and less painful treatment for burn injuries, a 26-year-old patient with Grade 2 burn injuries was successfully treated using stem cell therapy at a city-based hospital, doctors said on Thursday.

Anand Tiwari suffered burns after accidentally falling in a boiler unit while at work. He sustained Grade 2 and early Grade 3 burns in all parts of the body below his neck.

When admitted to the city based StemRx Bioscience Solutions hospital, he had severe burning sensation and pain all over the body. Blisters and swellings were noticed in many areas of his chest and limbs.

According to doctors, after initial care and stabilisation of the patient, for treatment of burns, a treatment protocol was prepared by Pradeep Mahajan, a regenerative medicine researcher at Stemrx Bioscience Solutions Hospital.

Explaining the treatment procedure, Mahajan said: "This involved the use of growth factors and fibroblasts and collagen based gel. These biological agents stimulate natural healing mechanisms in the body."

"The advantage of these growth factors is that they can be obtained from the patients' own body and hence are safe and effective. Additionally, unlike conventional treatment options, biological agents promote faster recovery," he said.

Under the stem cell therapy, the treatment process has to be repeated continuously so as to get rid of the problem completely and accordingly the procedure was performed.

"During the entire treatment, the patient was not given any closed dressing. He also underwent blood and supplementary fluid transfusion as required to maintain systemic homeostasis," said Mahajan.

He said that changes in the patient were observed as early as two-three days after the initiation of therapy. Drying of superficial burns began and swelling started reducing.

"Gradually, dry crusts started peeling and by the end of the third week, initial healing of most areas was complete. There was no odour or oozing from any wound and he did not complain of pain or burning sensation anymore.

"After a month-long treatment, healthy skin formation is being observed and further healing is progressing at an impressive rate," said Mahajan, adding that in treatment through conventional modalities, it takes more than eight weeks for healing to happen and further several months for patient to be able to regain joint and facial movements.

--IANS

rup/nir

(This story has not been edited by Business Standard staff and is auto-generated from a syndicated feed.)

IANS

http://bsmedia.business-standard.com/_media/bs/wap/images/bs_logo_amp.png 177 22

Original post:
Patient with severe burns treated using stem cell therapy | Business ... - Business Standard

June 23, 2017

Imagine being able to take cells from your skin, transform them into other types of cells, such as lung, brain, heart or muscle cells, and use those to cure your ailments, from diabetes to heart disease or macular degeneration. To realise this, however, challenges still remain, Professor Janet Rossant, a pioneer in the field, says.

All across the world, scientists have begun clinical trials to try and do just that, by making use of the incredible power and versatility of stem cells, which are special cells that can make endless copies of themselves and transform into every other type of cell.

While human embryos contain embryonic stem cells, which help them to develop, the use of those cells has been controversial. The scientists are using induced pluripotent stem cells instead, which are other cells that have been reprogrammed to behave like stem cells.

"There are still significant challenges that we need to overcome, but in the long run we might even be able to create organs from stem cells taken from patients. That would enable rejection-free transplants," said Professor Janet Rossant, a pioneer in the field.

The mouse that changed everything

A speaker at the recent Commonwealth Science Conference 2017 held in Singapore and organised by Britain's Royal Society and Singapore's National Research Foundation, Prof Rossant gave an overview of stem cells' origins, history, uses and potential.

Now a senior scientist at The Hospital for Sick Children (also known as Sick Kids) in Toronto, Canada, after a decade as its chief of research, she was the first scientist to demonstrate the full power of stem cells in mice.

In the early 1990s, scientists believed that stem cells could only become certain types of cells and carry out limited functions. Based on her own research and that of others, however, Prof Rossant believed that they were capable of far more.

Working with other scientists, she created an entire mouse out of stem cells in 1992, upending the conventional wisdom. "We went on to create many baby mice that were completely normal, and completely derived from stem cells grown in a petri dish," she said.

"That was an amazing experiment, and it was instrumental in making people believe that human embryonic stem cells could have the full potential to make every cell type in the body," she added.

When scientists learned how to remove stem cells from human embryos in 1998, however, controversy ensued. Many lobbied against the cells' use in medical research and treatment due to the moral implications of destroying even unwanted embryos to gain the cells.

In Canada, Prof Rossant chaired the working group of the Canadian Institutes of Health Research on Stem Cell Research, establishing guidelines for the field. These guidelines helped to keep the field alive in Canada, and were influential well beyond the country's borders.

In 2006, Japanese researchers succeeded in taking skin cells from adult mice and reprogramming them to behave like embryonic stem cells. These revolutionary, induced pluripotent stem (IPS) cells allowed scientists to sidestep the ongoing controversy.

The challenges in the way

While stem cells have been used for medical treatment in some cases bone marrow transplants, for example, are a form of stem cell therapy there are several challenges that need to be overcome before they can be used more widely to treat diseases and injuries.

"We need to get better at turning stem cells into the fully mature cells that you need for therapy. That's going to take more work. Another issue is that of scale-up. If you're going to treat a patient, you need to be able to grow millions of cells," said Prof Rossant.

She added: "Safety is another concern. One of the most exciting things about pluripotent stem cells is that they can divide indefinitely in the culture dish. But that's also one of the most scary things about them, because that's also how cancer works.

"Furthermore, because we need to genetically manipulate cells to get IPS cells, it's very hard to know whether we've got completely normal cells at the end of the day. These are all issues that need to be resolved."

She noted that some scientists are working on making "failsafe" IPS cells, which have a built-in self-destruct option if they become dangerous. "Bringing stem cells into regenerative medicine is going to require interdisciplinary, international collaboration," she said.

In the meantime, stem cells have been a boon to medical research, as scientists can use them to create an endless supply of different cells to study diseases and injuries, and test drugs. "That's the biggest use of IPS cells right now," Prof Rossant said.

Sick kids and how to help them

At SickKids, which is Canada's largest paediatric research hospital, she has been using stem cells to study cystic fibrosis, a frequently fatal genetic disorder that causes mucus to build up and clog some organs such as the lungs. It affects primarily children and young adults.

SickKids discovered the CFTR gene that, when mutated, causes the disease. It was also the first to produce mature lung cells, from stem cells, that can be used to study the disease and test drugs against it.

Even better, Prof Rossant and her team were able to turn skin cells from cystic fibrosis patients into IPS cells and then into lung cells with the genetic mutation specific to each of them. This is critical to personalising treatment for each patient.

"Drugs for cystic fibrosis are extraordinarily expensive, and patients can have the same mutation and yet respond differently to the same drug," Prof Rossant explained. "With our work, we can make sure that each patient gets the right drug at the right time."

In 1998, Prof Rossant also discovered a new type of stem cell in mice, now called the trophoblast stem cell. These surround an embryo and attach it to the uterine wall, eventually becoming the placenta. She is using such cells to study placenta defects and pregnancy problems.

By using IPS cells to create heart cells and other cells, pharmaceutical companies can also test their new drugs' effectiveness and uncover potential side effects, as well as develop personalised medicines.

"There are still huge amounts of opportunities in pluripotent stem cells," said Prof Rossant, who has won numerous awards for her research, including the Companion of the Order of Canada and the 2016 Friesen International Prize in Health Research.

She is also president and scientific director of the Toronto-based Gairdner Foundation, which recognises outstanding biomedical research worldwide, and a professor at the University of Toronto's molecular genetics, obstetrics and gynaecology departments.

"Meetings like the Commonwealth Science Conference are a fantastic opportunity for scientists to come together, learn about each other's work and establish new relationships, which will help to push science forward, including in stem cell research," she said.

She noted: "The world of science is becoming increasingly interdisciplinary, so this kind of meeting of minds across nations, cultures and scientific fields is really the way of the future."

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Stem cells: the future of medicine - Medical Xpress

Sarepta Therapeutics has penned its second DMD gene therapy pact this year as it announces a tie-up with Frances Genethon, a nonprofit R&D org.

The research collaborationwill see the Franco-American pair jointly develop treatments for Duchenne muscular dystrophy and comes after Sareptas first FDA approval for DMD with its controversial med Exondys 51 (eteplirsen).

RELATED: FDA expert lashes out at 'worrisome' Sarepta approval in JAMA

Sarepta is looking to tap into Genethons preclinical microdystrophin gene therapy approach, which can target the majority of patients with DMD. Its current med can only treat certain patients, namely those with the mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13% of the population with DMD.

It is hoping that with new tie-ups, it could produce a gene therapy that could treat many more, if not all, patients with the disease, although this is still some years off. DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness. The disease primarily affects young boysand occurs in about one out of every 3,600 male infants worldwide.

This builds on the pacts announced at the start of the year at the JPM conference, which saw it sign a deal with the Nationwide Childrens Hospital, which also focuses on the microdystrophin gene therapy program, as well as another form of gene therapy.

An initial phase 1/2a trial for the microdystrophin gene therapy is slated to begin at the end of the year and will be done at Nationwide Childrens. It also penned an exclusive license agreement with Nationwide for their Galgt2 gene therapy program, originally developed by researcher Paul Martin. This early-stage program aims to research a potential surrogate gene therapy approach to DMD, whereby the gene therapy looks to induce genes that make proteins that can perform a similar function as dystrophin. The goal will be to produce a muscle cell that can function normally even when dystrophin is absent, Sarepta said at the time.

Under the terms of its latest collaboration, Genethon will be responsible for the early development work. Sarepta has the option to co-develop Genethons microdystrophin program, which includes exclusive U.S. commercial rights. Financial terms, as is becoming more common with these pacts, have not been disclosed.

RELATED: With Exondys 51 approved, Sarepta chief Ed Kaye to bow out

Our agreement with Genethon strengthens our ongoing commitment to patients and is aligned with our strategy of building the industrys most comprehensive franchise in DMD, said Ed Kaye, Sareptas outgoing chief. This partnership brings together our collective experience in Duchenne drug development and Genethons particular expertise in gene therapy for rare diseases. We look forward to working with Genethon given their knowledge, large infrastructure and state-of the-art manufacturing capabilities to advance next generation therapies for DMD.

Frederic Revah, CEO of Genethon, added: Microdystrophin-based gene therapy is a very promising approach with potential application to a large majority of Duchenne patients. In order to accelerate the development of a treatment, we are very pleased to partner with Sarepta Therapeutics, which has demonstrated commitment and success for innovative therapies for Duchenne muscular dystrophy. This partnership brings together the highly complementary and synergistic expertises of Sarepta and Genethon, to the benefit of the patients.

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Sarepta signs another Duchenne gene therapy pact as it aims for wider treatment - FierceBiotech

San Francisco-based Vineti, a cell and gene therapy software and analytics company, has closed on Series A funding round that pulled together nearly $14 million.

Backing came from General Electric Ventures, Mayo Clinic and new investor Draper Fisher Jurvetson.

The company will use the funds to continue growing its team and to deliver cloud-based software to improve patient access. It also plans to speed its work on life-saving treatment delivery and to promote safety and FDA compliance for individualized cell therapies.

The Vineti platform integrates logistics, manufacturing and clinical data.

Physicians, medical researchers and pharmaceutical companies are working together to develop successful therapies, transitioning from a one-size-fits-all model to individualized treatments for each patient, Vineti CEO Amy DuRoss said in a statement. But, the process for administering these treatments is broken and outdated, restricting access to terminal patients and creating unnecessary risk.

DuRoss added that Vineti developed the platform to ensure treatments reach the patients who need them the most. She added that many patients who are excellent candidates dont have access to the most innovative therapies and discovery timelines are more challenging than necessary.

GE Ventures formed Vineti based on customer requests to bridge the technology gap between individualized cell therapies and production.

Modern technology solutions to address complex production and delivery processes are lacking. GE Ventures, Mayo Clinic and DFJ have invested in Vineti to rectify these problems.

Vineti is led by DuRoss, Chief Strategy Officer Heidi Hagen and CTO Razmik Abnous.

Twitter: @Bernie_HITN Email the writer: bernie.monegain@himssmedia.com

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Vineti to fast-track cell and gene therapy tech with $14 million first ... - Healthcare IT News

SAN FRANCISCO, Calif. Mayo Clinic Ventures has partnered with a California-based company to make cancer-fighting gene therapies available to the public.

Vineti, a pioneering cell and gene therapy software and analytics company, announced Tuesday that it had completed its initial round of funding raising $13.75 million aimed at delivering "the first cloud-based software solution to improve patient access, accelerate life-saving treatment delivery, and promote safety and regulatory compliance for individualized cell therapies."

The funding was provided by Mayo Clinic Ventures, GE Ventures, DFJ and LifeForce Capital. It's just the 15th company that Mayo Clinic Ventures has backed since it was formed, according to Andy Danielson, vice chairman of Mayo Clinic Ventures.

"One thing with Vineti that we liked is that we have a commitment to cell and gene therapies at Mayo," Danielson told TechCrunch.com. "Vineti will make the gene and cell therapy production process more efficient and as a result, less costly. It's all part of the equation of making these therapies more affordable and opening them up to a greater number of people."

The targeted cancer therapy under development by Vineti is part of a thriving field that conducted more than 800 clinical trials in 2016 while investing nearly $6 billion. It's all aimed at positively impacting the oncology field, the largest market in medicine that's expected to grow to $165 billion by 2021.

The first two cell therapies are expected to hit the market later this year.

Vineti touts its plans as one that "integrates logistics, manufacturing and clinical data to improve product performance overall and enable faster, broader access for patients."

"Physicians, medical researchers and pharmaceutical companies are working together to develop successful therapies, transitioning from a one-size-fits-all model to individualized treatments for each patient," said Amy DuRoss, CEO at Vineti. "Now, the process for creating and delivering these treatments can be as innovative as the therapies themselves. We are developing the Vineti platform to help these treatments reach the patients who need them the most, and are confident the partnership between our advances technologies and leading medical research will deliver better outcomes across the globe."

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Mayo Clinic Ventures funds new cancer-fighting cell, gene therapy ... - Post-Bulletin

British drugmaker GlaxoSmithKline made headlines last year when it won approval for its gene therapy Strimvelis in Europe. But, due to a small patient population and high price tag, the drug has only been used once. So far, despite higher levels of safety and efficacy than previous iterations, the new wave of gene therapies still face commercial hurdles.

Spark Therapeutics looks set to be the next company to take on this challenge in the U.S. The biotech is currently awaiting approval of its treatment for a rare genetic form of blindness a potential one-time cure. Yet pricing will be the most closely watched aspect of this therapy, likely serving as an early barometer of what might be sustainable for a pipeline of treatments still in development.

While gene therapy offers the promise of cures and new ways of revolutionizing treatment of genetic diseases, society remains a long way from fully realizing those advances.

After decades of setbacks, a slew of next-gen gene therapies are ready to hit the U.S. market, prompting questions about manufacturing and pricing. Read More >>

A pricing conundrum and ethical decisions are clouding an already hazy path to market for many gene therapy drugs and providers. Read More >>

With an approval of Spark Therapeutics' gene therapy for a rare eye disease rapidly approaching, new questions about pricing are being raised. Read More >>

In a field shaped by small patient populations and eye-popping cost considerations, understanding gene therapy's promise and challenges comes down, in part, to the numbers. Read More >>

While many are optimistic about gene editing's ability to cure disease, it seems not enough realize the more dangerous aspects of treatment. Read More >>

See more here:
Gene therapy: What you need to know - BioPharma Dive