A phase I clinical trial of PAC-1, a drug that spurs programmed cell death in cancer cells, found only minor side effects in patients with end-stage cancers. The drug stalled the growth of tumors in the five people in the trial with neuroendocrine cancers and reduced tumor size in two of those patients. It also showed some therapeutic activity against sarcomas, scientists and clinicians report in the British Journal of Cancer.
The drug was first identified and developed as an anti-cancer agent by scientists at the University of Illinois Urbana-Champaign.
The findings from the clinical trial are noteworthy because the drug was tested in a small number of patients with advanced disease, said study clinical director Dr. Arkadiusz Dudek, an oncologist with the HealthPartners Cancer Center at Regions Hospital in St. Paul, Minnesota, and at Mayo Clinic in Rochester, Minnesota. Phase I clinical trials are designed to test whether a new drug compound has worrisome side effects or toxicities in human patients, Dudek said. But scientists also can look for early evidence of therapeutic benefits. The trial enrolled cancer patients with advanced disease who had run out of other treatment options.
“We had patients with colon cancer, breast cancer, pancreatic cancer, adenocarcinoma, melanoma and others,” he said.
The clinical trial — and another testing PAC-1 against brain cancer — involves patients and clinicians at three institutions: Regions Hospital, the University of Illinois Chicago and Johns Hopkins University.
Phase I clinical trials track side effects in patients who first are given very low doses of the compound being tested. If the drug is well tolerated and causes no discernible toxicities over the course of a month, the dose is incrementally increased. This process can take several months before a potentially therapeutic dose is given, said Dr. Oana Danciu, a medical oncologist and associate director for clinical research at the University of Illinois Cancer Center in Chicago, who led the clinical trial.
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