Archive for March, 2017

A French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder, researchers report.

The first-in-the-world case is detailed in Thursdays New England Journal of Medicine.

About 90,000 people in the U.S., mostly blacks, have sickle cell, the first disease for which a molecular cause was found. Worldwide, about 275,000 babies are born with it each year.

Vexing questions of race and stigma have shadowed the history of its medical treatment, including a time when blacks who carry the bad gene were urged not to have children, spurring accusations of genocide, Keith Wailoo of Princeton University wrote in a separate article in the journal.

The disease is caused by a single typo in the DNA alphabet of the gene for hemoglobin, the stuff in red blood cells that carries oxygen. When its defective, the cells sickle into a crescent shape, clogging tiny blood vessels and causing bouts of extreme pain and sometimes more serious problems such as strokes and organ damage. It keeps many people from playing sports and enjoying other activities of normal life.

A stem cell transplant from a blood-matched sibling is a potential cure, but in the U.S., fewer than one in five people have a donor like that. Pain crises are treated with blood transfusions and drugs, but theyre a temporary fix. Gene therapy offers hope of a lasting one.

The boy, now 15, was treated at Necker Childrens Hospital in Paris in October 2014. Researchers gave him a gene, taken up by his blood stem cells, to help prevent the sickling. Now, about half of his red blood cells have normal hemoglobin; he has not needed a transfusion since three months after his treatment and is off all medicines.

Its not a cure but it doesnt matter, because the disease is effectively dodged, said Philippe Leboulch, who helped invent the therapy and helped found Bluebird Bio in Cambridge, Massachusetts, the company that treated the boy. The work was supported by a grant from the French governments research agency.

Bluebird has treated at least six others in the U.S. and France. Full results have not been reported, but the gene therapy has not taken hold as well in some of them as it did in the French teen. Researchers think they know why and are adjusting methods to try to do better.

Two other gene therapy studies for sickle cell are underway in the U.S. at the University of California, Los Angeles and Cincinnati Childrens Hospital and another is about to start at Harvard and Boston Childrens Hospital using a little different approach.

This work gives considerable promise for a solution to a very common problem, said Dr. Stuart Orkin, a Boston Childrens Hospital doctor who is an inventor on a patent related to gene editing.

The results are quite good in this patient, he said of the French teen. It shows gene therapy is on the right track.

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Gene therapy: http://ghr.nlm.nih.gov/primer/therapy/availability

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Marilynn Marchione can be followed at http://twitter.com/MMarchioneAP

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Gene therapy lets a French teen dodge sickle cell disease - The Seattle Times

Photo: Michael Cummo / Hearst Connecticut Media

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo inside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo inside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo outside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo outside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy CEO and President John Walter inside his office in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy CEO and President John Walter inside his office in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy Executive Director Margaret Cianci inside her office in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy Executive Director Margaret Cianci inside her office in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo inside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo inside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo outside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Alliance for Cancer Gene Therapy (ACGT) CEO and President John Walter and Executive Director Margaret Cianci pose for a photo outside the ACGT offices in Stamford, Conn. on Monday, Feb. 27, 2017.

Stamford-based ACGT sees success with support of cancer research

STAMFORD The Alliance for Cancer Gene Therapy recently announced two grants totaling $500,000. But its leaders are already filling the funding pipeline to support future recipients.

Describing itself as the only nonprofit in the country solely dedicated to funding and supporting research into cell and gene therapies for cancer, ACGT has built itself into a fundraising force since its founding in 2001. It has awarded 52 researcher grants totaling almost $27 million. Major events such as Swim Across Americas annual swim between Greenwich and Stamford and a gala held other every other year the next gala is set for April 19 at the Harvard Club in Manhattan are founts for the organizations giving.

We want to keep creating new interest in the organization, new donors and new dollars, ACGT President and CEO John Walter said in an interview Monday. For people who are interested in investing in cancer research, were a good place to turn to because of the success weve had.

Research backed by ACGT focuses on gene and cell mutations, a targeted approach that aims to reduce or eliminate debilitating side effects of traditional treatments.

The recipients of ACGTs latest Young Investigator Awards, the University of Calgarys Marco Gallo and the University of Pittsburghs Greg Delgoffe, each earned a $250,000 grant.

We play a very key role in helping young investigators, said Margaret Cianci, ACGTs executive director. They have wonderful ideas, and we want to help them fund that research and move into the next phase.

ACGT-supported research has produced four companies that are developing major new treatments, according to ACGT officials. They said they hope the first gene-therapy drug will launch in the U.S. later this year. One of the drugs that could soon enter the market is based on Car T cell research by an ACGT fellow, Dr. Carl June, who is an immunotherapy professor at the University of Pennsylvania.

Buoyed by recent advances, ACGT officials said that they want to keep supporting research of emerging treatments.

While you have these excellent immunotherapies, how can we combine them with some of the gene therapy going on? Cianci said. The goal is to be sure that the cancers dont come back.

All of ACGTs donations fund research. The nonprofits board covers administrative expenses for the nonprofits staff of four.

Headquartered on Cummings Point Road in the citys Waterside section, ACGT has made several leadership changes recently. Walter and Barbara Gallagher, national director of philanthropy, both arrived last year. Walter succeeded Barbara Netter, who co-founded ACGT with her late husband, Edward Netter.

Barbara Netter, a Greenwich resident, maintains an important role in the organization as honorary chairman. She said that the organizations work is fulfilling Edward Netters vision.

With the promise of new drugs coming on the market, it should really make a statement about gene therapy, she said. Im very optimistic.

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Stamford-based ACGT sees success with support of cancer research - The Advocate

Posted Feb. 28, 2017 at 3:39 p.m.

Published: 2017-02-28 15:39:55 Updated: 2017-02-28 15:39:55

By JIM SHAMP, NCBiotech Writer

Raleigh, N.C. The North Carolina Biotechnology Center has announced that Pfizer has committed to providing funding in the amount of $4 million which will enable the Center to establish and administer a multi-year academic fellowship program to help advance North Carolinas fast-growing expertise in gene therapy.

The new program, to be managed by NCBiotech, will support distinguished postdoctoral fellowships in North Carolina university research laboratories providing advanced scientific training in gene therapy-related research.

Absent or faulty proteins linked to genetic mutations cause numerous devastating diseases, making gene therapy an increasingly important treatment strategy.

Pfizers portfolio in North Carolina has grown in recent years. The company already operates a pharmaceutical manufacturing facility in the Lee County community of Sanford, and in August 2016, it acquired leading-edge gene therapy company Bamboo Therapeutics, Inc. in Chapel Hill.

With that acquisition, Pfizer gained the expertise of Bamboos world-renowned co-founder, R. Jude Samulski, Ph.D., director of the Gene Therapy Centerat the University of North Carolina at Chapel Hill. The deal also included an 11,000-square-foot facility for the highly specialized manufacturing of recombinant adeno-associated viral vectors.

Pfizer is one of several biopharmaceutical companies that have added high-profile gene therapy acquisitions, and several partnerships with biotechnology companies and leading academic institutions, to its R&D portfolio. Numerous other North Carolina scientists and companies are also making significant inroads into gene therapy, gene editing and related applications, many with NCBiotech support. For example, Samulski was recruited to UNC in 1993 as part of a $430,000 NCBiotech grant. Additionally, Bamboos former parent company received more than $700,000 in Biotech Center grants and loans.

Gene therapy advances require specific skills in addition to deep scientific knowledge. The fellowship program being established with Pfizers funding aims to boost that talent pipeline, with talent that has already proven to be exceptional in North Carolina. Such funding will enable NCBiotech to provide two-year fellowship support to postdoctoral scientists. The funding will afford the Center the ability to cover salaries, benefits, materials, professional development and travel for such postdoctoral scientists. The Center will encourage competitive applications from scientists interested in establishing research careers in gene therapy and related research activities.

The Biotech Center will also create and manage a related gene therapy Exchange Group. It will join some 25 other exchange groups designed to unite North Carolina-based academic and industry scientists with shared professional interests. The Gene Therapy EG will include these new postdoctoral fellows, their mentors, and others interested in the burgeoning gene therapy sector.

The field of gene therapy research has made tremendous strides in recent years, and we are pleased to be able to further enhance our leadership position in this area through this unique fellowship program, said Mikael Dolsten, M.D., Ph.D., president of worldwide research and development at Pfizer. We believe that gene therapy may hold the promise of bringing true disease modification for patients suffering from devastating diseases, and North Carolina is uniquely positioned to help us take advantage of collaborative opportunities that can develop the specialized talent well need.

Doug Edgeton, president and CEO of the Biotech Center, said he was deeply honored that Pfizer targeted North Carolina, and the Center, for the groundbreaking fellowship program.

Pfizer embraced the opportunity to work with us given weve proven for more than 30 years that we have the expertise and success metrics to maximize impact, said Edgeton. We not only have outstanding research institutions across our state, but we also have a well-respected culture of partnering and collaboration that allows us to be nimble and responsive. This is a wonderful example.

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Pfizer commits $4M to NC Biotechnology Center gene therapy ... - WRAL Tech Wire

An experimental gene therapy that turns a patient's own blood cells into cancer killers worked in a major study, with more than one-third of very sick lymphoma patients showing no sign of disease six months after a single treatment, its maker said Tuesday.

In all, 82 percent of patients had their cancer shrink at least by half at some point in the study.

Its sponsor, California-based Kite Pharma, is racing Novartis AG to become the first to win approval of the treatment, called CAR-T cell therapy, in the U.S. It could become the nation's first approved gene therapy.

A hopeful sign: the number in complete remission at six months 36 percent is barely changed from partial results released after three months, suggesting this one-time treatment might give lasting benefits for those who do respond well.

"This seems extraordinary ... extremely encouraging," said one independent expert, Dr. Roy Herbst, cancer medicines chief at the Yale Cancer Center.

The worry has been how long Kite's treatment would last and its side effects, which he said seem manageable in the study. Follow-up beyond six months is still needed to see if the benefit wanes, Herbst said, but added, "this certainly is something I would want to have available."

The therapy is not without risk. Three of the 101 patients in the study died of causes unrelated to worsening of their cancer, and two of those deaths were deemed due to the treatment.

It was developed at the government's National Cancer Institute and then licensed to Kite. The Leukemia and Lymphoma Society helped sponsor the study.

Results were released by the company and have not been published or reviewed by other experts. Full results will be presented at the American Association for Cancer Research conference in April.

The company plans to seek approval from the U.S. Food and Drug Administration by the end of March and in Europe later this year.

The treatment involves filtering a patient's blood to remove key immune system soldiers called T-cells, altering them in the lab to contain a gene that targets cancer, and giving them back intravenously. Doctors call it a "living drug" permanently altered cells that multiply in the body into an army to fight the disease.

Patients in the study had one of three types of non-Hodgkin lymphoma, a blood cancer, and had failed all other treatments. Median survival for such patients has been about six months.

Kite study patients seem to be living longer, but median survival isn't yet known. With nearly nine months of follow-up, more than half are still alive.

Six months after treatment, 41 percent still had a partial response (cancer shrunk at least in half) and 36 percent were in complete remission (no sign of disease).

"The numbers are fantastic," said Dr. Fred Locke, a blood cancer expert at Moffitt Cancer Center in Tampa who co-led the study and has been a paid adviser to Kite. "These are heavily treated patients who have no other options."

One of his patients, 43-year-old Dimas Padilla of Orlando, was driving when he got a call saying his cancer was worsening, chemotherapy was no longer working, and there was no match to enable a second try at a stem cell transplant.

"I actually needed to park ... I was thinking how am I going to tell this to my mother, my wife, my children," he said. But after CAR-T therapy last August, he saw his tumors "shrink like ice cubes" and is now in complete remission.

"They were able to save my life," Padilla said.

Of the study participants, 13 percent developed a dangerous condition where the immune system overreacts in fighting the cancer, but that rate is lower than in some other tests of CAR-T therapy. The rate fell during the study as doctors got better at detecting and treating it sooner.

Roughly a third of patients developed anemia or other blood-count-related problems, which Locke said were easily treated. And 28 percent had neurological problems such as sleepiness, confusion, tremor or difficulty speaking, but these typically lasted just a few days, Locke said.

"It's a safe treatment, certainly a lot safer than having progressive lymphoma," and comparable to combination chemotherapy in terms of side effects, said the cancer institute's Dr. Steven Rosenberg, who had no role in Kite's study. The first lymphoma patient Rosenberg treated this way, a Florida man, is still in remission seven years later.

There were no cases of swelling and fluid in the brain in this or any other study testing Kite's treatment, company officials said. That contrasts with Juno Therapeutics, which has had a CAR-T study put on hold twice after five patient deaths due to this problem.

Company officials would not say what the treatment might cost, but other types of immune system therapies have been very expensive. It's also being tested for some other types of blood cancer.

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Gene therapy to fight a blood cancer succeeds in major study - Fox News