Gene therapy provides life-changing relief from hemophilia

Andrew Davidoff, MD

People with severe hemophilia B, a bleeding disorder, are at risk of serious health problems and early death. To manage their disease, they endure lifelong injections of a blood clotting protein.

Now, gene therapy developed at St. Jude Children’s Research Hospital, University College London (UCL) and the Royal Free Hospital has transformed life for 10 men with severe hemophilia B.

Years after receiving a single DNA treatment, the men continue to produce their own clotting factor with minimal side effects. This treatment has dramatically decreased their reliance on injections. Some now participate in sports like soccer without worrying about bleeding.

“The results so far have made a profound difference in the lives of study participants by dramatically reducing their risk of bleeding,” said Andrew Davidoff, MD, St. Jude Surgery chair. “This study provides the first clear demonstration of the long-term safety and efficacy of gene therapy.”

Injections of blood clotting protein can cost $250,000 a year. The researchers estimate that overall spending on injections of the missing protein has declined more than $2.5 million for study participants. The findings were published in the New England Journal of Medicine.

November 19, 2014

Read the news release