Ines Batinic-Haberle, PhD, professor emeritus of radiation oncology at Duke University School of Medicine, created hundreds of compounds before she hit upon the right one.
More than 30 years later, her drug, BMX-001, has improved survival and lessened cognitive decline for people with advanced brain cancer enrolled in a phase 2 clinical trial. The drug is also under study in clinical trials for several other cancers.
BMX-001 mimics the action of an enzyme naturally made by the body called superoxide dismutase, which was discovered in the 1960s by Irwin Fridovich, PhD, a Duke biochemist who was internationally renowned as “the father of free radical biology.”
Free radicals are unstable forms of oxygen that can damage cells. Fridovich discovered that superoxide dismutase neutralizes free radicals and thus is “the enzyme that we can’t live without,” Batinic-Haberle said.
Batinic-Haberle began designing a synthetic form of the enzyme in the 1990s, while working as a postdoctoral associate in Fridovich’s lab. She wanted to create a small molecule that, unlike superoxide dismutase, is “very stable, water soluble, and positively charged, therefore able to cross cellular membranes and reach the brain,” she said.
Katherine B. Peters, MD, PhD, professor of neurosurgery and neurology at the Preston Robert Tisch Brain Tumor Center at Duke, led the multi-institutional trial of BMX-001 in people with high grade glioma (brain cancer) and presented the results at the Society for Neuro-oncology Annual Meeting in November 2023, as the plenary presentation.
The trial included 160 patients with high-grade glioma at nine clinical centers. People who took BMX-001 combined with radiation and standard-of-care chemotherapy (temozolomide) had an increase in median survival time of 6.6 months compared to those who received radiation and chemotherapy alone.
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