FDA To Support Leukemia Gene-Therapy Treatment.
Novartis AG’s pioneering new cancer drug won enthusiastic support from a federal advisory panel, paving the way for approval of the first U.S. gene therapy. The panel unanimously recommended that the Food and Drug Administration approve the drug, tisagenlecleucel, for patients ages 3 to 25 with relapsed B-cell acute lymphoblastic leukemia (ALL), the most common form of U.S childhood cancer. The drug uses a new technology known as CAR-T, or chimeric antigen receptor T-cell therapy, which harnesses the body’s own immune cells to recognise and attack malignant cells. In a clinical trial, 83 percent of patients who had relapsed or failed chemotherapy achieved complete or partial remission three months post-infusion. After one year 79 percent of patients were still alive. Patients with ALL who fail chemotherapy typically have only a 16 percent to 30 percent chance of survival. “This is a potentially paradigm-changing type of benefit,” said Dr. Brian Rini, a panelist and physician at Cleveland Clinic Taussig Cancer Institute. The FDA is not obliged to follow the recommendations of its advisers but typically does so. The agency is expected to rule on the drug by the end of September. Approval of tisagenlecleucel would have significant implications not only for Novartis but for companies developing similar treatments, including Kite Pharma Inc, Juno Therapeutics Inc and bluebird bio Inc. It would also advance a technique scientists have been attempting to perfect for decades and help lift the entire field of cell therapy. “This will be a historic approval,” said Brad Loncar, chief executive of Loncar Investments which runs the Loncar Cancer Immunotherapy ETF. However, more than half of patients experienced a serious complication known as cytokine release syndrome which occurs when the body’s immune system goes into overdrive. Doctors were able to manage the condition and the syndrome caused no deaths. The FDA expressed concern that the drug could cause new malignancies over the long term. Panelists generally felt that risk was low and they said Novartis’s risk mitigation proposals were adequate. Credit: Reuters.
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