New drug combination shows promise against childhood brain cancer

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March 17, 2016

Researchers at Sanford Burnham Prebys Medical Discovery Institute (SBP) in La Jolla, California have identified a new combination therapy for the most aggressive form of medulloblastoma, a fast-growing brain cancer occurring mostly in pediatric tumors. “Our goal was to identify drugs with minimal toxicity that we can move quickly from the laboratory to the clinic, where new therapeutic options are desperately needed,” said Robert Wechsler-Reya, Ph.D., one of the senior authors. The research team developed a compound to treat Group 3 medulloblastoma tumors, the group with the worst prognosis – only 40 percent of these patients become long-term survivors. The combination involved two drugs, histone deacetylase inhibitors (HDACIs) and phosphatidylinositol 3-kinase inhibitors (P13K1s) in mice to kill mouse and human medulloblastoma cells with minimal toxicity to normal cells.

According to Yanxin Pei, Ph.D., co-first author of the study, “Our initial screen identified several HDACIs that killed MYC-activated medulloblastoma cells without harming normal cells.” The findings of this study highlight the value of drug screening to identify therapies effective for specific types of disease and how combinations of drugs can be more effective than single agents.

To read the full study, click here