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Scientists first synthesized the drug, DFMO, in 1979, but since then it has developed a reputation as a jack of all trades and master of few. In the 1980s, DFMO showed efficacy in treating African sleeping sickness and eventually got regulatory approval for that use. It also gained clearance as a skin cream to fend off sun-related skin cancer. Otherwise, DFMO seemed to have been lost in the shuffle.
Part of the problem was a curious side effect of the drug. In some patients, it caused subtle, temporary hearing loss.
Now, oncologist Frank Meyskens Jr. of the University of California, Irvine and his colleagues have completed nearly two decades of testing very low doses of DFMO in people who are at high risk for colorectal cancer. Early on, the work showed that at doses only one-fiftieth the amount used to treat cancers, DFMO was safe and patients tolerated it well — and the hearing side effect didn’t show up.
In the recent study, researchers recruited 375 people who had already had precancerous colorectal growths called polyps removed. The scientists randomly assigned some to receive placebo pills and others to get DFMO plus the long-standing anti-inflammatory drug sulindac — both at very low doses. After three years, all participants underwent colonoscopy to determine their polyp status.
The results were so clear that the trial was stopped. About 41 percent of participants receiving placebo pills showed a polyp recurrence, compared with only 12 percent receiving the two-drug treatment. Moreover, 17 people getting placebos had developed more than one polyp while only one receiving the drugs had.
“We’re looking for commercial partners to try to move this
drug forward,” says Meyskens, who presented the findings in April in
DFMO, or difluoromethylornithine, inhibits the synthesis of polyamines, basic compounds in cells. “Elevation of polyamines leads to increased growth, and almost anything like that leads to cancer opportunities,” Meyskens says.
In another study presented at the meeting, researchers in
Molecular biologist Michelle Haber of Children’s Cancer
Institute in
Old drugs can have new uses, says Roy Herbst, a medical
oncologist at the
Found in: Body & Brain