FDA Approves First 'Living Drug' for Childhood Leukemia

"This is the first-ever true gene therapy treatment made available to the United States population and will help accelerate the speed at which we will see even more gene-based therapies come to fruition".

Known as gene therapy, the treatment involves removing immune cells called T cells from the patient's blood and then genetically altering them in a lab. The FDA itself called the approval a "historic action".

Shares of Juno Therapeutics Inc, which previous year reported a handful of patient deaths during trials of its CAR-T therapy, were down 8.9 percent at $39.92. Novartis has agreed a risk evaluation and mitigation strategy (REMS) with the FDA that amongst other things will see the therapy delivered only at certified treatment centres. The new cells would then continue to multiply to fight disease for months or years. The price is well below the $700,000 that was estimated before the approval of the drug. The new treatment can act as a lifesaver for those patients who did not recover through first line drug treatments. Although the treatment sometimes causes severe side effects, in a Novartis clinical trial the drug caused remission in 83% of 63 young patients with a certain type of leukemia, B cell acute lymphoblastic leukemia (ALL). "There are also over 800 cell therapy clinical trials now underway, and a considerable pool of research and pre-clinical work right across the cell therapy sector", said Bruce Levine, PhD, ISCT Commercialization and Immuno & Gene Therapy Committees, Barbara and Edward Netter Professor in Cancer Gene Therapy, University of Pennsylvania Perelman School of Medicine.

The treatment involves altering the patient's own immune system by removing T-cells and genetically modifying them to recognize, attack and kill cancer cells.

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Swiss pharmaceuticals group Novartis has said it will charge $475,000 (CHF458,000) a patient for its new cancer therapy, putting it among the most expensive drugs of all time. The clinical review was coordinated by the FDA's Oncology Center of Excellence, while CBER conducted all other aspects of review and made the final product approval determination. "The established path to commercialization taken by Novartis will provide considerable motivation for cell therapy scientists and development companies as well as investors looking to benefit from the ongoing success in the industry". The manipulated cells could cause a cytokine storm, which could present with high fever, low blood pressure, long congestion and other life-threatening issues. Instead of getting a better, updated therapy for a disease every decade or so, we might begin to see second-generation cell therapies in a few years.

For one, the treatments would have to treat more types of cancer than the one Kymriah was approved for on Wednesday. Penn and Novartis are also investigating the next generation of auto therapies for multiple myeloma, and for solid tumors, through trials in glioblastoma, mesothelioma, and ovarian and pancreatic cancer. CAR-T's side effects can be deadly.

Based on available data, patients on the treatment have had an 89% chance of surviving at least six months and a 79% chance of surviving at least a year, with most being relapse-free at that point.

(Copyright © 2015. All Rights Reserved.)
 
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