Clinical trial successfully repurposes cancer drug for hereditary bleeding disorder
www.sciencedaily.com
A drug approved for treating the blood cancer multiple myeloma may offer a safe and effective way to reduce the risk of severe nosebleeds from a rare but devastating bleeding disorder. Hereditary hemorrhagic telangiectasia (HHT), the world's second-most-common inherited bleeding disorder, affects approximately 1-in-5,000 people and can have life-threatening complications, but there are currently no U.S. FDA-approved drugs to treat HHT.
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The original was posted on /r/upliftingnews by /u/No-Zucchini3759 on 2024-09-19 18:59:41+00:00.

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“Hereditary hemorrhagic telangiectasia (HHT), the world’s second-most-common inherited bleeding disorder, affects approximately 1-in-5,000 people and can have life-threatening complications, but there are currently no U.S. FDA-approved drugs to treat HHT.”

“The results of our trial demonstrate the clear safety and efficacy of pomalidomide to treat bleeding in HHT, giving these patients a much-needed effective treatment option," said first author Hanny Al-Samkari, MD, the Peggy S. Blitz Endowed Chair in Hematology/Oncology at Massachusetts General Hospital.