Repurposed Cancer Drug Shows Effectiveness in Treating Rare Blood Vessel Disorder

Posted onOctober 10th, 2024byDr. Monica M. Bertagnolli

Developing a new drug from scratch can take a decade or more. But sometimes promising treatment options come from repurposing existing drugs for completely different medical conditions. Im happy to share a new example of this: a cancer drug calledpomalidomidethat was found in a clinical trial to be safe and effective for treating a blood disorder calledhereditary hemorrhagic telangiectasia(HHT).

HHT is an inherited blood vessel disorder that can cause excessive or even life-threatening bleeding. The disease is rare, affecting about 1 in every 5,000 people worldwide, but because HHT is poorly understood and often misdiagnosed, its true incidence is likely greater. Most people with HHT experience recurrent severe nosebleeds, often in combination with mental health disorders such as depression and post-traumatic stress disorder, as well as other health conditions. HHT can also worsen with age and impact quality of life.

However, recent findings from an NIH-supported clinical trial, reported in theNew England Journal of Medicine, show that daily treatment with pomalidomide in people with HHT led to a significant reduction in nosebleed severity. Compared to trial participants taking a placebo, those taking pomalidomide needed fewer blood or iron transfusions and reported improvements in their quality of life. Because of these results, the trial was stopped months ahead of schedule, having found sufficient evidence that the treatment was safe and effective.