Cancer

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Hailing a breakthrough in fighting cancer, FDA approves gene therapy that functions as a ‘living drug’

In a step that heralds a new era in cancer treatment, the U.S. Food and Drug Administration said Wednesday it has approved a form of gene therapy that is highly effective at fighting an aggressive form of leukemia in young patients with no other options.

The treatment, to be marketed under the name Kymriah, is neither a pill nor an injection, but a personalized medicine service that functions as a “living drug.” Patients would have their body’s own disease-fighting T cells fortified and multiplied in a lab, then get the cells back to help them fight their cancer.

In clinical trials of 88 patients with a relapsing or treatment-resistant form of acute lymphoblastic leukemia, 73 went into remission after receiving the experimental treatment.

FDA Commissioner Scott Gottlieb, himself a survivor of blood cancer, predicted that this new approach to cancer treatment will “change the face of modern medicine.”

Cancer researchers and physicians outside the agency shared Gottlieb’s enthusiasm.

Dr. Crystal L. Mackall, associate director of Stanford University’s Cancer Institute, called Kymriah “a transformative therapy. … It represents an entirely new class of cancer therapies that holds promise for all cancer patients.”

Acute lymphoblastic leukemiais the most common form of pediatric cancer, affecting some 3,000 children and young adults yearly in the United States. Though it is considered highly curable in most patients, about 600 each year either do not respond to chemotherapy or see their leukemia return after an initial round of successful treatment.

http://www.sandiegouniontribune.com/newsletters/la-sci-sn-car-t-cancer-drug-20170830-htmlstory.html#nws=true