T cell therapy shows persistent benefits in young leukemia patients

IMAGE: Shannon L. Maude, MD, PhD, is a pediatric oncologist at Children’s Hospital of Philadelphia. view more 

Credit: Children’s Hospital of Philadelphia

Updated results from a global clinical trial of the CAR T-cell therapy, tisagenlecleucel, a landmark personalized treatment for a high-risk form of acute lymphoblastic leukemia (ALL), reveal that children and young adults continued to show high rates of durable, complete remission of their disease. Most side effects were short-lived and reversible, according to a study published in the Feb. 1 issue of the New England Journal of Medicine.

Based on previous findings, the U.S. Food and Drug Administration (FDA) approved tisagenlecleucel–brand name Kymriah–in August 2017 for children and young adults up to 25 years of age with B-cell ALL that is refractory or in second or later relapse. It was the first-ever FDA-approved gene therapy, as well as the first FDA-approved CAR-T cell therapy.

The updated results report new data from the Novartis-sponsored ELIANA trial, conducted in 25 centers in 11 countries worldwide.

Chimeric antigen receptor T (CAR-T) cell therapy genetically modifies a patient’s immune cells to make them seek out and kill leukemia cells. The approach was developed by a team led by Carl June, MD,


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