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FDA Panel Recommends Gene Therapy Approval

A US Food and Drug Administration panel has recommended that the agency approve a gene therapy treatment, the New York Times reports. If the agency does give the Novartis therapy the OK, it would be the first gene therapy to make it to the market, the Times adds.

Novartis' CTL019 is a customized CAR-T therapy to treat children and young adults with advanced leukemia, Stat News adds. It re-engineers patients' own T-cells so that they, when re-introduced into the patient, attack B-cells, which become malignant in leukemia. The Times notes that the treatment also kills healthy B-cells, leaving patients susceptible to infections, so they also receive immune globulins every few months to bolster their immune systems.

In a clinical trial, 83 percent of the patients treated were cancer-free after three months, Stat News adds.

The FDA panel has recommended the approach for treatment-resistant B-cell acute lymphoblastic leukemia in people between the ages of 3 and 25, the Times says. Stat News adds that patients who receive the therapy would be entered into a registry and followed for 15 years.

"It's a new world, an exciting therapy," Gwen Nichols, the chief medical officer of the Leukemia and Lymphoma Society, which funded some of the research into the treatment, tells the Times.

Stat News adds that Novartis is pursuing CAR-T therapies for lymphoma and that Kite Pharma has a CAR-T therapy for advanced and aggressive lymphoma under review at FDA.

FDA is to make its final decision on Novartis' CTL019 in early October, according to Stat News.