Friday, July 22, 2011

Children's Hospital, Genethon get FDA OK for gene therapy trial

By Rodney H. Brown

Children’s Hospital Boston has won approval from the U.S. Food and Drug Administration for a gene therapy trial in conjunction with French biotech Genethon to treat the rare immunodeficiency known as Wiskott-Aldrich syndrome (WAS).

Nonprofit laboratory Genethon and Children’s Hospital Boston announced their partnership on a gene therapy to treat WAS earlier this year. The condition is described in a release as “a severe immunodeficiency disease leading to death before adulthood” from “significant bleeding due to low platelet count and increased incidence of serious infections.” Funding for the Boston trial came from the National Heart, Lung and Blood Institute through its Gene Therapy Resource Program.

Principal investigators at Children’s are Sung-Yun Pai and Luigi Notarangelo, with David A. Williams, chief of the Division of Hematology/Oncology and director of Translational Research for Children’s Hospital Boston, serving as sponsor-investigator. The Dana-Farber Cancer Institute and Harvard Medical School will play a role in the trial – the gene insertion of patient cells will be done there.

Genethon is sponsoring parallel trials in London and Paris, and is the manufacturer of the gene therapy product being used.

This isn’t the first time Genethon and Children’s Hospital Boston have worked together. Early last year, the pair and a number of collaborators demonstrated on dogs that gene therapy could be used to treat myotubular myopathy (MTM), a neuromuscular disorder that affects proper muscle growth.