FDA Approves First Gene Therapy Treatment For Cancer

FDA Approves First Gene Therapy Treatment For Cancer
From TIME - August 30, 2017

The Food and Drug Administration (FDA) followed the advice of its advisory committee and approved a breakthrough treatment for children with acute lymphoblastic leukemia (ALL) on Wednesday. Its the first gene therapy approved in the U.S.

Dr. Carl June, director of the center for cellular immunotherapies at University of Pennsylvanias Abramson Cancer Center, who pioneered the therapy, was still emotional an hour after receiving the news of the approval in an email.

Its unbelievable, June told TIME, his voice wavering. Its an amazing feeling. We worked on it and worked on it and finally to have such a discrete, palpable event like this is such an amazing thing.

Read more: FDA Panel Recommends Approval of the First Gene Therapy Treatment

June and his team have been developing the treatment, called CAR T cell therapy (for chimeric antigen receptor T cells), for nearly three decades. Also called Kymriah, the treatment isnt actually a drug in the traditional sense, but a collection of the patients own cells that have been removed from the body and genetically engineered to recognize and destroy cancer cells circulating in the blood. Each treatment is a living drug, a bespoke therapy designed specifically for individual patients.

This is an ultra-personalized therapy that actually works, says June. Patients are involved in making their own drug.

Not only does the approval represent a landmark in being the first green-light for a gene therapy, it marks a sea change in the way cancer can be treated. Until now, treatments have focused on bringing in drugs, surgery or radiation to weaken the cancer. With CAR T cell therapy, the treatment starts from within, by enhancing the bodys own immune system to fight cancer with a genetic boost. "It's a paradigm shift," says Dr. Kevin Curran, from the pediatric bone marrow transplantation service at Memorial Sloan Kettering Cancer Center. "These are actually living cells that we pull out of the system, modify in the lab, teach how to find cancer, turn back on and put back into patients. To do that definitely shifts everything."

Read more: Cancer's Newest Miracle Cure

And there is hope that the therapy might even provide more cures for leukemia than existing options. The first patient to be treated with CAR T cells is currently in remission seven years after his treatment. We now have proof that it is possible to eradicate cancer by harnessing the power of a patients own immune system," Dr. Kenneth Anderson, president of the American Society of Hematology and a physician at Dana-Farber Cancer Institute, said in a statement. "This is a potentially curative therapy in patients whose leukemia is unresponsive to other treatments and represents the latest milestone in the shift away from chemotherapy toward precision medicine.


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