(medicalnewstoday.com) – Researchers at Sanford Burnham Prebys Medical Discovery Institute (SBP) have identified a new combination therapy for the most aggressive form of medulloblastoma, a fast growing type of pediatric brain cancer. The study was published online in Cancer Cell, and is expected to lead to a clinical trial to confirm the benefits of the novel drug combination.
“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, PhD, director of the Tumor Initiation and Maintenance Program at SBP and senior author of the study. “Using high-throughput drug screening, we identified a compound that cooperates with a second drug to inhibit tumor growth in vitro and in vivo.”
Medulloblastoma, a fast-growing cancer that arises in the cerebellum, is diagnosed in about 400 children annually in the U.S., making it the most common malignant brain tumor in children. Success rates for treating this cancer are improving, but lag behind those for other pediatric cancers.
Of the four distinct forms of medulloblastoma, patients with Group 3 tumors have the worst prognosis – only 40 percent of these patients become long-term survivors, compared to 80 percent of other medulloblastoma patients. Most Group 3 medulloblastoma cancers have high levels of the MYC oncogene, which causes cells to divide uncontrollably and form tumors.
Using mice with Group 3 medulloblastoma tumors, the research team demonstrated that the combination of two drugs, histone deacetylase inhibitors (HDACIs) and phosphatidylinositol 3- kinase inhibitors (PI3KIs), potently kill mouse and human medulloblastoma cells with minimal toxicity to normal cells.
The research was supported by funding from the National Cancer Institute, Alex’s Lemonade Stand Foundation, CureSearch for Children’s Cancer, and the California Institute for Regenerative Medicine.