Gene Therapy for Hemophilia A

Medical science is constantly evolving. I remember studying hemophilia A during my first year at the University of Pennsylvania School of Medicine in 2004. The treatments we learned about were primitive – factor transfusions. Eighteen years later (!), the New England Journal of Medicine has published a new article, Gene Therapy for Hemophilia A – clearly science is advancing.

Gene therapy! That caught my attention, probably because of the Jesse Geslinger case. Geslinger was the first person publicly identified as having died in a clinical trial for gene therapy, in 1999, also at Penn. He had suffered from ornithine transcarbamylase deficiency, and there were ethical lapses in the trial.

Anyway, in this new study, which was funded by BioMarin Pharmaceutical, patients with severe hemophilia A received valoctocogene roxaparvovec treatment. This increased endogenous factor VIII production and reduced bleeding.

Medical science will continue to progress, and we must make sure trials are always conducted ethically.

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