Pomalidomide, a bone marrow cancer drug, may be safely repurposed to treat a rare genetic blood disorder, according to a recent clinical trial. The Cleveland Clinic-led clinical trial was stopped early after demonstrating safety and effectiveness in treating hereditary hemorrhagic telangiectasia (HHT), a condition that currently has no cure. Dr.
Keith McCrae, professor of molecular medicine at the Cleveland Clinic who led the clinical trial, told The Epoch Times that he was motivated to find a drug for a particular patient he had 15 years ago. “This particular client was extremely ill..
.was requiring tens of thousands of dollars worth of blood products every week and was essentially advised to have most of his intestine resected because he was bleeding there,” McCrae said. McCrae dug into the research and found that the cancer drug thalidomide helped patients with similar symptoms.
“I treated him with thalidomide, and within two or three weeks, his bleeding had essentially stopped,” he said. The study , published in the New England Journal of Medicine, assigned adults with HHT in a 2:1 ratio to receive either pomalidomide or a placebo for 24 weeks. The trial was planned to run for four years but was stopped at 24 weeks after a second analysis showed that pomalidomide had met the threshold for efficacy.
Sarrah Hein wasn’t aware of HHT until she was diagnosed at 26 while pregnant with her son, Anthony, who was also diagnosed with the condition. “Looking back, I had .