Using gene therapy, researchers have treated six patients with severe hemophilia B. When given a single infusion of gene therapy, patients with the inherited blood disorder showed an improved ability to form blood clots.
Researchers at St. Jude Children’s Research Hospital and the University College London demonstrated that it’s possible to treat the inherited blood disorder with gene therapy — with long-term results.
Normally, people with hemophilia B are missing a gene called Factor IX that is needed to form blood clots so they must inject themselves several times a week with an expensive protein therapy.
Researchers used an adenoid-associated virus (AAV) 8 to transport Factor IX gene to the liver. After being injected with gene therapy, patients no longer had to receive protein injections.
UCL’s Amit Nathwani said in a statement:
“This is a potentially life-changing treatment for patients with this disease and an important milestone for the field of gene therapy. It could have ramifications for the treatment of hemophilia A, other protein and liver disorders and chronic diseases such as cystic fibrosis”
The study was published in the New England Journal of Medicine. While the sample size was small, the results offered a long-term solution for people with the inherited blood disorder.
But gene therapy would certainly change the market for treating hemophilia. ISI Group analyst Mark Schoenebaum said to Reuters:
“This clearly presents a curveball to our (and much of Wall Street’s) assumptions around the future of the hemophilia market.”
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