Timothy Fan and Paul Hergenrother
A drug that spurs cancer cells to self-destruct has been cleared for use in a clinical trial of patients with anaplastic astrocytoma, a rare malignant brain tumor, and glioblastoma multiforme, an aggressive late-stage cancer of the brain. This phase Ib trial will determine if the experimental drug PAC-1 can be used safely in combination with a standard brain-cancer chemotherapy drug, temozolomide.
The trial is approved for patients who have seen their cancer progress after first-line therapy. This is an extension of an ongoing human phase I clinical trial of PAC-1 alone in patients with various late-stage cancers. Phase I trials are designed to test the safety of new drugs in human patients
PAC-1 is unusual in that it is able to cross the blood-brain barrier, a formidable obstacle to most anti-cancer drugs. The drug targets procaspase-3, an enzyme that is overexpressed in many cancer cells, said University of Illinois chemistry professor Paul Hergenrother, who discovered PAC-1’s anti-cancer effects more than a decade ago. After tests in human cell lines and rodents proved promising, Hergenrother and veterinary oncologist Dr. Timothy Fan, a professor of veterinary clinical medicine at Illinois, tested PAC-1 in pet dogs with a variety of naturally occurring cancers.