Today the U.S. Food and Drug Administration (FDA) approved the first chimeric antigen receptor (CAR) T-cell therapy for the treatment of children with relapsed/treatment-resistant CD-19 positive acute lymphocytic leukemia (ALL). ALL is the most common type of pediatric cancer.
In CAR T-cell therapy, a patient’s own immune cells are collected and outfitted with a receptor to target a protein on the surface of cancer cells, creating a highly personalized cell army that is reintroduced into the body to attack the cancer.
In response to today’s approval, ASH President Kenneth C. Anderson, MD, of the Dana-Farber Cancer Institute, issued the following statement:
“The approval of CAR T-cell therapy for pediatric leukemia marks an important shift in the blood cancer treatment paradigm. We now have proof that it is possible to eradicate cancer by harnessing the power of a patient’s own immune system. This is a potentially curative therapy in patients whose leukemia is unresponsive to other treatments and represents the latest milestone in the shift away from chemotherapy toward precision medicine. Today’s approval is the result of over a decade of hematology research, including research funded by the National Institutions of Health (NIH).
“While the importance of CAR-T cannot be overstated, this approval only pertains to a small population of children. More research is needed to make this therapy more effective for a broader population, to reduce the severe side effects that patients experience during treatment, and ultimately to find a broader application beyond blood cancers. Continued research will also lead to improved manufacturing of large numbers of cells, which is necessary to make this therapy accessible to more patients.
“ASH is very excited to have contributed to the progress that has led to today’s approval and will continue to support research to refine CAR T-cell therapy. The Society believes that immunotherapies like this are among the most promising areas of hematology research and has made immunotherapy one of six major priorities in the Agenda for Hematology Research. ASH also has formed a taskforce on immunotherapy with the overall goal of improving these therapies and speeding their development from the bench to the bedside.”