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A Breakthrough for Pediatric Glioblastoma

POSTED BY: Florida Hospital

A promising experimental treatment for a dire childhood brainstem cancer aims to retrain our own immune system to recognize and fight off cancer cells. The cancer, called diffuse intrinsic pontine glioma (DIPG), afflicts a few hundred school-aged children in the U.S. each year. The median survival is less than one year, and there is no cure.

The innovative new therapy works by injecting engineered human immune cells – called chimeric antigen receptor T cells (or CAR-T cells) – into the body. Researchers at the Stanford University School of Medicine administered these CAR-T cells to mice that had been infected with DIPG. Their cancer tumors quickly began to shrink.

Sherif Makar, M.D., a fellowship-trained neuro-oncologist with Florida Hospital Medical Group who was not involved with the research, says immunotherapy has been used to combat cancers of the lung and skin, but is lately showing promise as a treatment of brain cancer. A similar therapy using CAR-T cells was recently approved by the FDA to treat pediatric leukemia.

“The problem with glioblastoma is it evades detection,” Dr. Makar says of the fast-growing, deadly tumor. But the study authors were able to engineer the immune cells of the mice to recognize and attack cancer cells, leaving only a handful alive.

Furthermore, because the treatment relies on our own immune system, it may avoid harmful side effects often seen in cancer care.

Based on the study’s success, the therapy may soon move into human clinical trials, but researchers must first work to put safeguards in place to minimize risks. For physicians like Dr. Makar, the promising early results offer renewed hope that the treatment may help bring an end to this devastating childhood cancer.

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To learn more about immunotherapy and the other innovative treatments Florida Hospital has to offer, visit Florida Hospital Cancer Institute.