Study Points to Possible Treatment for Lethal Pediatric Brain Cancer

(National Institutes of Health) – Using brain tumor samples collected from children in the United States and Europe, an international team of scientists found that the drug panobinostat and similar gene regulating drugs may be effective at treating diffuse intrinsic pontine gliomas (DIPG), an aggressive and lethal form of pediatric cancer. The study, published in Nature Medicine, was partially funded by the National Institutes of Health, the Department of Defense, and more than 25 nonprofit foundations devoted to finding cures for childhood brain cancer.

“Our results provide a glimmer of hope for treating this heartbreaking disease,” said Michelle Monje, M.D., Ph.D., assistant professor of neurology and neurological sciences, Stanford University School of Medicine, California, a senior author of the study and a specialist in DIPG. “Caring for DIPG patients drives me to find new ways to treat them.”

DIPG typically attacks children 4 to 9 years of age. Children progressively lose muscle control as the tumor rapidly attacks the pons, a region deep inside the brain that connects the brain to the spinal cord, and is difficult to reach and surgically remove. Despite radiation treatment, children usually survive for about nine months, and less than 1 percent survive longer than five years.

Six years ago, Dr. Monje started to create and share cell cultures of patients’ DIPG cells that could be studied in labs. In this study, she and her colleagues used cell cultures collected from 16 patients in the United States and Europe to search for drugs that could kill or stop the growth of DIPG cells. By performing experiments in petri dishes and with mice, they found that panobinostat, a drug designed to change the way cells regulate genes, may be effective at inhibiting DIPG growth and extending survival rates.

Pin It on Pinterest

Scroll to Top