Dana-Farber/Boston Children's responds to FDA approval of CAR-T cell therapy

August 30, 2017


Following today's historic FDA approval of CAR-T therapy for refractory pediatric and young adult ALL, please find the below statement from Lewis Silverman, MD, Clinical Director, Pediatric Hematologic Malignancies Center at the Dana-Farber/Boston Children's Cancer and Blood Disorders Center:

"We are very encouraged by today’s news that CAR-T cell therapy will now be available for pediatric and young adult patients with relapsed and refractory acute lymphoblastic leukemia (ALL). This new therapeutic approach provides a promising option for some of the most challenging cases we face in pediatric ALL. We are actively preparing to offer this novel treatment at our center to patients at Dana-Farber/Boston Children's Cancer and Blood Disorders Center."

For more information and further expert reaction to this FDA approval, please visit:

FDA Gives First-Ever Approval to CAR T-Cell Therapy

Landmark moment for science as the FDA approves a gene therapy for the first time

About CAR-T

Wednesday's FDA decision means that the drug can be used as standard therapy for the treatment of pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL) that has relapsed or hasn't responded to previous treatments. The approval, which follows the drug's designation as a "breakthrough" therapy by the FDA, underscores the potential of CAR T-cell therapies to treat a wide range of cancers, researchers say.

The FDA ruling in favor of CTL019 is based on the results of a series of clinical trials that showed the therapy to be safe and effective in the patients tested. The largest such trial, known as the ELIANA trial, found that 83 percent of patients treated with CTL019 achieved complete remission — the disappearance of all signs of cancer — within three months of treatment.

CAR T-cell therapy, like all forms of cancer immunotherapy, seeks to sharpen and strengthen the immune system's inherent cancer-fighting powers. It involves treating patients with modified versions of their own immune system T cells — white blood cells that help protect the body from disease.

Looking Ahead

Dana-Farber Cancer Institute is also currently involved in CAR T-cell clinical trials for adult patients with ALL, mantle cell lymphoma, diffuse large B-cell lymphoma, follicular lymphoma, and multiple myeloma. For more information on these programs, visit Dana-Farber's Cellular Therapies Program.

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