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Gene therapy achieves early success against hereditary bleeding disorder
Date:12/10/2011

ls inherit the mutation.

Previous efforts to ease hemophilia B symptoms by introducing a correct copy of the gene have been unsuccessful.

The current study used adeno-associated virus (AAV) 8 as the vector to deliver the Factor IX gene along with additional genetic material into the patient's liver. AAV8 was picked because the incidence of natural infection with AAV8 is low. It belongs to a family of viruses that target liver cells but do not cause disease in humans or integrate into human DNA. Participants in the study received no immune suppressing drugs prior to gene therapy.

This approach was jointly pioneered by St. Jude and UCL, initially in the laboratory of study co-author Arthur Nienhuis, M.D, a member of the St. Jude Department of Hematology.

For this study, each patient received a one-time infusion of the vector into a vein in the arm. Two patients each were treated with escalating doses of the vector. Following treatment, Factor IX levels rose in all six patients from less than 1 percent of normal levels prior to the gene therapy to between 2 and 12 percent.

Factor IX levels increased the most in the two study volunteers who received the highest dose of the experimental vector, researchers said. After treatment, levels of the protein ranged from 3 to 12 percent in those men. Even modest increases that raise Factor IX production to more than 1 percent of normal levels have the potential to dramatically affect a patient's quality of life and reduce bleeding episodes, said the study's senior author Andrew Davidoff, M.D., chair of the St. Jude Department of Surgery.

"The first patient has been followed for the longest time, and his levels have remained at 2 percent for more than 18 months. These results are highly encouraging and support continued research. More patients are scheduled to be enrolled in future trials scheduled to begin later this year," Davidoff said.

One of the participants who
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Contact: Carrie Strehlau
media@stjude.org
901-595-2295
St. Jude Children's Research Hospital
Source:Eurekalert

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