St. Jude's Publishes Gene Therapy Study

Dec 12, 2011

MEMPHIS, Tenn. -- A study outlining a new gene therapy treatment for Hemophilia, developed at Saint Jude’s in Memphis, appears in this month’s issue of the New England Journal of Medicine.

In a news release, St. Jude’s says the findings of a six-person study mark the first proof that gene therapy can reduce bleeding episodes for those suffering from the inherited blood disorder. Four of the six participants have reportedly had no bleeding episodes since undergoing the new therapy.

Dr. Andrew Davidoff is spokesman for St. Jude’s.

"The early results from our trial suggest that a simple, safe, single intervention may, in fact, cure patients with Hemophilia B and that its wide-spread availability is hopefully coming in the very near future."

The gene therapy was developed at St. Jude’s while clinical trials were conducted in England at the University College in London. 

The study notes that at least one participant recently ran in a marathon without suffering any problems with bleeding.