NEWS BLOG from UPMC and the University of Pittsburgh Schools of the Health Sciences

FDA Approves First Gene Therapy for Cancer

by Cyndy Patton 0 Comments

The Food and Drug Administration (FDA) recently approved the very first cancer treatment that uses genetically engineered cells as therapy. The treatment is approved for use in children and young adults with a blood cancer known as acute lymphoblastic leukemia (ALL), and marks the start of a new way to treat cancer. The therapy, called CAR-T, is made by using a patient’s own white blood cells, then re-engineering them to find and attack cancer tumors.

FDA Approves First Gene Therapy For Cancer - CAR-TWe asked Dr. Alison Sehgal, UPMC Hillman Cancer Center oncologist and assistant professor of medicine at the University of Pittsburgh School of Medicine, about this important advancement in cancer treatment.

What is immunotherapy and what are CAR-T cells?

A. Immunotherapy is a type of cancer treatment that uses a patient’s own immune system to attack cancer tumors. CAR-T cell therapy is just one of the emerging immunotherapy treatments currently being researched. T cells are part of our immune system and have the ability to fight infections and kill cancer cells. CAR-T cell immunotherapy involves removing a patient’s T cells from the blood, modifying them with an inactivated virus so that they can find molecules on the surface of cancer cells, and returning the T cells to the body to directly attack the cancer cells. Until this recent FDA approval, CAR-T cell immunotherapy has been available only in clinical trials, including those here at UPMC.

Why is this therapy and approval such an important advancement?

A. This is the first approved adoptive cellular therapy that has shown significant results in clinical trials. The therapy is approved for the treatment of children and adults up to 25 years old who have ALL, which is one of the most common cancers in children. In clinical trials, CAR-T therapy has been effective for these kids who are essentially out of options to treat their cancer because the standard approaches have not worked for them. This will surely mark the start of other cellular immunotherapies that are showing promising results in treating additional types of cancer. It’s an exciting time to be involved in cancer research for scientists, physicians and patients.

How can cancer patients learn more or become involved?

A. UPMC Hillman Cancer Center has many clinical trials in cancer, including these novel immunotherapy trials. We are one of just 41 of the nation’s National Cancer Institute designated Comprehensive Cancer Centers, an elite group of institutions dedicated to cancer research by developing more effective approaches to prevention, diagnosis and treatment of cancer. Our research takes place right here in Pittsburgh, and these trials are made available to cancer patients throughout the more than 60 UPMC Hillman Cancer Centers. Any patient interested in clinical trials can find more information here.

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