A new gene therapy could change the lives for people with haemophilia A
Imagine living with a condition where even a minor injury could lead to life-threatening bleeding. This is the reality for people with severe haemophilia A. But a groundbreaking study from India may offer a life-changing solution.
Researchers tested a new gene therapy on five men, aged 22 to 41, who had frequent bleeding episodes—more than 20 a year. This therapy used their own blood stem cells, modified to carry a special gene that helps produce the clotting protein factor VIII, which people with haemophilia A lack. Unlike traditional treatments, they underwent a gentler pre-treatment with a drug called treosulfan, and some received a special "boost" called a transduction enhancer.
The outcomes were truly remarkable. Bleeding episodes stopped completely for all participants, and they showed stable levels of factor VIII. Those who got the "boost" had much higher levels of the clotting protein, reaching up to 40 times that of those who did not.
Even better, this treatment didn't seem to disrupt their overall health—most participants maintained normal sperm counts, an encouraging sign of safety.
For people with haemophilia A, this could mean freedom from constant bleeding and endless treatments. While the therapy is still in its early stages, it offers hope that life with haemophilia might soon look very different. Scientists are optimistic this innovation could lead to a brighter future for millions worldwide.
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