Existing drug may treat deadliest Childhood Brain Tumor

The drug restricted the tumor’s growth in a lab dish and improved the survival time of mice that had the tumor implanted into their brains, according to researchers at the Stanford University School of Medicine, in collaboration with colleagues at other institutions. The work is noteworthy because the disease, a brain stem cancer called Diffuse Intrinsic Pontine Glioma, is nearly always fatal and lacks an effective treatment.

“There have been over 200 clinical trials of chemotherapy drugs for DIPG, and none have shown any survival benefit,” said Michelle Monje, MD, PhD, assistant professor of neurology at Stanford and a senior author of the paper. “But those trials were conducted before we knew anything about the unique biology of this tumor.”

While the preclinical data in the new study are encouraging, Monje cautioned that the drug, Panobionstat, needs further testing in a closely monitored human clinical trial. The research team is now planning such a trial in children with DIPG. Panobinostat was recently approved by the Food and Drug Administration for treatment of a form of blood cancer.

The drug repairs a portion of the cellular machinery now known to be defective in DIPG tumor cells, the new research showed. “A key thing that is wrong with DIPG cancer cells gets corrected by panobinostat,” said Monje.

DIPG affects 200-400 school-aged children in the United States each year and has a five-year survival rate of less than 1 percent; half of patients die within nine months of diagnosis.