An experimental drug that attacks brain tumor tissue by crippling the cells' energy source called the mitochondria has passed early tests in animal models and human tissue cultures, say Houston Methodist scientists. As reported on the cover of the April 2015 ChemMedChem (early online), Houston Methodist Kenneth R. Peak Brain & Pituitary Tumor Center Director David S. Baskin, M.D., and Peak Center Head of Research Martyn Sharpe, Ph.D. designed a drug called MP-MUS that destroyed 90 to 95 percent of malignant glioma cells, yet in other experiments did not seem to adversely affect healthy human brain cells (in vitro). This compliments a soon to be published extensive study showing the same drug can treat human brain cancer grown in the brains of mice. Researchers hope to begin testing the drug in human clinical trials in 2016 or 2017

"We are very optimistic that we'll get there," said Baskin, also Vice Chair of the Department of Neurosurgery at Houston Methodist Hospital. "Our past work has shown that MP-MUS has very low toxicity until it gets into tumor cells. Once it arrives, it is changed to its active form, doing a lot of damage where we want it to, leaving healthy brain cells alone -- a bit like a 'smart bomb.' To our knowledge, this is the first known example of selective mitochondrial chemotherapy, which we believe represents a powerful new approach to brain cancer."

Medical options for brain tumor patients are woeful, Baskin said. "It's a horrible diagnosis. Because of where the tumors are located, and because of the way they can infiltrate healthy tissue, surgery is often not helpful long term. The most effective chemotherapy drug available right now, temozolomide, only extends life from 9 to 15 months, and patients' quality of life during that period isn't very good."