Enero 23, 2018

FDA approves gene-therapy leukemia treatment

31 Agosto 2017, 08:31 | Martinez Canez

Endpoints News

Endpoints News

At $475,000 a pop, the therapy isn't cheap, but most people would consider that a small price to pay for a cure from a awful, life-threatening disease.

Wednesday's announcement will "change the face of modern medicine and drug development", FDA Commissioner Dr. Scott Gottlieb said at a news briefing. That hasn't escaped the notice of big players and small, with a host of developers looking to do much better, more safely, with new drugs in the pipeline. The disease is a cancer of the bone marrow and blood that causes the body to make abnormal lymphocytes.

Dr. Carl June, director of the center for cellular immunotherapies at University of Pennsylvania's Abramson Cancer Center, who pioneered the therapy, was still emotional an hour after receiving the news of the approval in an email. The altered T-cells include a new gene that contains a protein called chimeric antigen receptor, or auto, that directs the T-cells to kill leukemia cells with the antigen CD19. It's important to note, however, that it's not clear how long that benefit lasts: Some patients did relapse months later, and the others still are being tracked to see how they fare over the long term. That drug is aimed at Lymphoma, another blood cancer.

This is a brand new way of treating cancer.

However, Anderson stressed that "this approval only pertains to a small population of children [with ALL]".

The therapy, also known as tisagenlecleucel, is a suspension meant to be used one time, with a patient's T calls. For its part, Novartis has said patients would only pay the full whack when patients respond to the therapy by the end of the first month, according to Marketwatch.

The FDA also approved a Risk Evaluation and Mitigation Strategy for Kymriah. Plus, there can be serious side effects, such as inflammatory problems that have the potential to be fatal.

Most patients suffered side effects that can be grueling, even life-threatening.

The therapy's cost will be another issue.

"I don't want to be an apologist for high drug prices in the U.S.", Grupp stressed. Now 12, she has been free of leukemia for more than five years.

Shares of Bluebird Bio Inc, which is developing a promising CAR-T treatment, were up 11.1 percent at $113.73.

The therapy consists of an individualized, one-time treatment that has shown an 83% remission rate in clinical trials with patients that have not responded to standard treatments.

Kymriah, marketed by Novartis, was granted several special designations, including "fast track", "priority review", and "breakthrough therapy" consideration.

"The science has reached a point of superiority, where enough of the components of these endeavors have worked out", he explained.

The promise of the CAR-Ts is enormous for patients with relapsed or refractory ALL, who otherwise face a death sentence after all other therapies have failed.

Thanks for your continued support.


"This is a major advance and is ushering in a new era", panel member Malcolm Smith, a pediatric oncologist at the US National Institutes of Health in Bethesda, Maryland, told Nature at the time.

We're thankful for your support in every way.

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