FDA Approves First Gene Therapy for Cancer

In what it called a “historic action,” the US Food and Drug Administration approved the first gene therapy for cancer treatment in August. It is a genetically modified T-cell therapy pioneered by researchers at the Perelman School of Medicine and Children’s Hospital of Philadelphia in collaboration with Novartis [“The T-Cell Warriors,” Mar|Apr 2015]. Kymriah, as the chimeric-antigen-receptor (CAR) therapy will be branded, was cleared for use in certain patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL). It involves genetically engineering a patient’s own T-cells and infusing them back into his or her body, where they target and destroy cancerous cells.

“This is a turning point in the fight against B-cell ALL that opens up opportunities for patients across the world who desperately need new options,” said Carl H. June, the Richard W. Vague Professor in Immunotherapy at Penn’s Perelman School of Medicine and director of the Center for Cellular Immunotherapies in the Abramson Cancer Center. In early-stage clinical trials, more than 90 percent of patients who had failed to respond to conventional treatment achieved a complete remission one month after receiving the experimental therapy. That led to a global registration trial in 2015, in which 83 percent of patients achieved a complete remission.

The spectacularly effective therapy, whose personalized administration means it cannot be scaled up for mass production, will carry a list price of $475,000. Novartis is cooperating with the Centers for Medicare and Medicaid Pricing to implement “outcomes-based” reimbursement, whereby the company will only receive reimbursement if Medicaid patients respond to the therapy within one month of receiving it. The cost to privately insured patients is expected to vary depending on their insurance plans. Price transparency is not a common feature of the US health care system, but bone marrow transplants (which are another option for ALL patients) are estimated to cost between $350,000 and $800,000. Novartis will create a registry to follow Kymriah patients for 15 years after being treated to monitor their progress and any potential future side effects.

June’s team has other trials underway for the treatment of adult ALL patients, chronic lymphoblastic leukemia, and non-Hodgkin lymphoma. Penn and Novartis are also investigating the next generation of CAR therapies for multiple myeloma, and for solid tumors, through trials in glioblastoma, mesothelioma, and ovarian and pancreatic cancer. Other CAR trials at Penn are exploring the technique for prostate cancer, melanoma, and triple-negative breast cancer.

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