m3n4.net September 24, 2018

Leukemia : the Americans launched the first gene therapy

01 September 2017, 01:46 | Louis Holland

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- The US Food and Drug Administration approved a new leukemia treatment, which the agency considers the first gene therapy it has cleared to hit the market in the United States.

The FDA approved Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). In just the last few years, progress with auto T cells and other ACT approaches has greatly accelerated, with researchers developing a better understanding of how these therapies work in patients and translating that knowledge into improvements in how they are developed and tested.

Kymriah belongs to a new class of treatments called CAR-T therapies.

Kymriah, a cell-based gene therapy, is approved in the United States for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse. The FDA on Wednesday expanded the approval of Actemra, a rheumatoid arthritis drug that can also treat cytokine release syndrome.

"This is a turning point in the fight against B-cell ALL that opens up opportunities for patients across the world who desperately need new options", said Carl June, MD, the Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine in Penn's Perelman School of Medicine and director of the Center for Cellular Immunotherapies in the Abramson Cancer Center. The company is carefully training hospitals and staff to provide the treatment, which can cause a life-threatening immune reaction, as well as long-term complications.

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"While Novartis' decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive", said David Mitchell, founder and president of the advocacy group Patients For Affordable Drugs, and a cancer patient himself.

The drug Kymriah was developed by Novartis Pharmaceuticals and the University of Pennsylvania. Most ALL patients respond to initial therapy, but Kymriah will be an invaluable back-up option for relapsed and refractory patients who now have poor prognosis.

The FDA said it has granted 76 applications for trials involving experimental CAR-T therapies. The hope is that one day doctors might be able to prescribe a cell therapy and use it that same day, instead of waiting weeks to get the therapy back.

"The only potentially curative option for these pediatric and young adult patients is allogeneic stem cell transplant, which costs $500,000 to $800,000 for the first year", Cooper said.

Those updates could increase the number of patients that cell therapies can treat. That could expand to tens of thousands of people, but it'll require some major changes, Epstein said. They'll also have to be less toxic, so more people can consider it instead of only those who have very few other options.

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Other drugmakers have been developing Car-T products, including Kite Pharma, which was snapped up for $11.9 billionby Gilead Sciences earlier this week.

The high cost of the therapy reflects the fact that the CAR-T procedure is fairly intensive.

With the treatments available now, fewer than 10 percent of patients with the relapsed, hard-to-treat leukemia are alive five years after diagnosis. Penn and Novartis are also investigating the next generation of vehicle therapies for multiple myeloma, and for solid tumors, through trials in glioblastoma, mesothelioma, and ovarian and pancreatic cancer.

In clinical trials, CAR-T therapies have shown remarkable efficacy against blood cancers.

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