T gene therapy for leukemia clears FDA hurdle

14 July, 2017, 00:36 | Author: Garry Little
  • FDA advisers back Novartis' game-changing personalized CAR-T for cancer, approval in sight

Although the FDA is not obliged to follow the recommendations of its advisory panels, it often does so.

Despite unknowns surrounding the therapy, "it is hard to argue with the unprecedented clinical success we have seen in this population of patients who do not have other viable treatment options", added Grzegorz S. Nowakowski, MD, of the Mayo Clinic in Rochester, Minn. But treatment gave their daughter a few extra months of happy life, Kappen said.

Approximately 620 pediatric and young adult patients with ALL relapse after achieving an initial response each year in the United States despite current treatments.

"We believe that when this treatment is approved it will save thousands of children's lives around the world", Whitehead told the meeting, his voice cracking as he looked at his daughter beside him.

Principal supporting data for the application came from a phase II trial of children and young adults with relapsed/refractory ALL, given a single infusion of CTL019.

Last year, when Kite hired Christine Cassiano its new SVP of corporate communications and investor relations, she said that communication around CAR-T therapies is "about making it relatable to patients".

The panel is reviewing the safety, effectiveness and production of the treatment.

The scalability of CAR-T treatments has, in fact, been one of the major challenges facing Novartis and its two biggest rivals in the space, Kite and Juno.

Novartis officials acknowledged the adverse events associated with auto T-cell therapy in general, including the potential for serious adverse events.

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The main side effect of CTL-019 is cytokine release syndrome, a blistering immune reaction to the drug and the detritus of dying cancer cells. Clinical trials of the medically advanced treatment were held in February 2016 by a group of scientists, when several dozen blood cancer patients, who barely had months to live, were administered the drug. In a lab, a gene is then inserted into the T cells that prompts them to grow a receptor that targets a special marker found on some blood cancer cells.

A harmless, disabled version of an old medical foe - HIV, the virus that causes AIDS - is employed to carry the new genetic material into the patient's T-cells to reprogram them.

The approval of the drug is likely, especially in light of the positive results of experiments in the field of cell modification to fight diseases.

In this pivotal study informing the committee's decision, roughly half of 68 patients receiving the drug experienced this complication, though none died from it.

This is perhaps one of the most significant things to happen to patients with this, but also with other types of cancer, as this might lead to other discoveries and more treatments can be applied in the future.

CAR-T therapy is a high risk approach, and little is known about any long-term toxicity effects of treatments. It's also unclear if patients whose cancer goes into remission will be cured or will have their cancer return eventually.

Patients with ALL who fail chemotherapy typically have a 16 to 30 percent chance of survival.

Other biotech and pharmaceutical companies are developing types of gene therapy to treat solid cancers and rare gene-linked diseases.

Could this drug be used for other cancers? That was approved previous year for a deadly condition called severe combined immunodeficiency and launched with a $670,000 price tag.

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