A $7 million federal grant will allow researchers to focus on solving immune-system obstacles to MD gene therapy
The National Institutes of Health (NIH) has awarded $7 million to Nationwide Children's Hospital in Columbus, Ohio, and designated the institution a Paul D. Wellstone Muscular Dystrophy Cooperative Research Center (Wellstone MDCRC).
|Jerry Mendell, co-director of the MDA clinic at Nationwide, will be co-director of the new Wellstone center.|
Neurologist and longtime MDA grantee Jerry Mendell, who also co-directs the MDA clinic at Nationwide, will share director responsibilities for the new center with Chris Walker, professor of Pediatrics, Molecular Virology, Immunology and Medical Genetics at Nationwide.
"We’re delighted by NIH’s decision," said Valerie Cwik, MDA medical director and executive vice president for research, noting that the MDA clinic at Nationwide is part of MDA’s DMD Clinical Research Network.
"This well-deserved designation and federal research grant will expand upon MDA’s longtime funding of Nationwide Children’s Hospital investigators, and will accelerate the lifesaving research efforts being led by Jerry Mendell and his colleagues."
Targeting the immune system in DMD
The new Wellstone MDCRC at Nationwide will focus on developing strategies to overcome immune-system barriers to gene correction in Duchenne muscular dystrophy (DMD).
Results reported in April 2010 from a small, MDA-supported clinical trial of gene therapy in DMD showed unwanted responses by the immune system to the muscle protein dystrophin (which is missing in DMD). These immune responses potentially pose a barrier not only to gene therapy-based treatments, but also to other experimental treatments designed to raise levels of the dystrophin protein. (For more, see Caution: Immune Response Seen in DMD Gene Therapy.)
Advocacy efforts fuel research
The Wellstone MDCRCs are named for the late Sen. Paul Wellstone (D-Minn.), a champion for the muscular dystrophy community and tireless advocate for MD research.
Wellstone, who died in a plane crash in 2002, was a driving force behind the passage in 2001 of the Muscular Dystrophy Community Assistance, Research, and Education (MD-CARE) Act, which significantly increased federal funding for muscular dystrophy research and lead to the creation of several MDCRCs. These "centers of excellence" work both independently and collaboratively on research into several forms of muscular dystrophy.
MDA — which is committed to collaborating with the NIH and to leveraging research funding through public-private partnerships — matched the $1.5 million awarded by NIH to the first three Wellstone MDCRCs in 2002.
In 2008, the MD-CARE Amendments were passed, expanding the focus on tracking disease progression and on “translational” research that seeks to move significant lab findings into human clinical trials.
This latest NIH grant and establishment of Nationwide Children’s as a Wellstone MDCRC “underscores the importance of continued research funding and the promise that current research efforts hold," said Annie Kennedy, MDA vice president for advocacy.
“We have great confidence that this NIH award will yield great therapeutic developments for individuals and families affected by DMD."