CAR T-Cell Therapy Charts a New Course in U.S. with FDA Approval

FDA approves groundbreaking therapy to fight ALL

On Wednesday, the U.S. Food and Drug Administration (FDA) approved a new treatment designed to treat relapsed/refractory B-cell precursor acute lymphoblastic leukemia (ALL).

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According to Mikkael Sekeres, MD, MS, Director of the Leukemia Program at Cleveland Clinic Cancer Center, chimeric antigen receptor (CAR)-T cell therapy is a form of therapy in which immune cells are removed from a patient’s blood, re-engineered ex vivo to include a gene for CAR, and reintroduced into the bloodstream to attack leukemia cells with the CD19 antigen.

“This therapy is truly remarkable,” says Dr. Sekeres. “This is a vanguard of cancer therapy that we’re going to be seeing more and more of in the future.”

The treatment, tradenamed Kymriah and known as CTL019 or tisagenlecleucel, is the first therapy of its kind available in the United States. The FDA unanimously recommended for approval and then approved the immunocellular therapy for use in children and young adults with relapsed/refractory B-cell precursor ALL.

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The single-arm, international ELIANA trial showed an overall remission rate of 83 percent within three months of treatment, with 63 percent of patients experiencing complete remission. One limitation of the trial was the short median follow-up of 4.8 months.

Other limitations to the therapy include severe side effects, including cytokine release syndrome (CRS); a certification the FDA requires in order for providers to use CAR-T cell therapy, thus limiting availability to large medical centers; and a hefty price tag.

Still, says Dr. Sekeres, “this is a paradigm shift, a real step forward in personalized treatments for cancer.” Though the FDA approval applies to a relatively small group of patients, oncologists “are very hopeful that this is just the beginning.”