Duchenne Alliance Funds Follistatin Gene Therapy Trial with $500,000 Grant

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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Duchenne AllianceThe International Duchenne Alliance has announced that it will provide a $500,000 grant to researchers Jerry Mendell, M.D. and Milo Biotechnology to support their work in a first ever follistatin gene therapy trial for Duchenne muscular dystrophy. The study will be held at Mendell at Nationwide Children’s Hospital in Columbus, Ohio, and the scientist has recently published positive data about their research project, demonstrating its efficacy in increasing muscle strength and function in Becker’s muscular dystrophy patients.

The therapy is based on adeno-associated virus delivery of follistatin 344 and has been developed by Mendell, in collaboration with Brian Kaspar Ph.D., in order to increase the strength of the muscle, as well as prevent its wasting and fibrosis. In addition, since the mechanism of action of follistatin is not mutation specific, the researchers believe it may also be helpful in other forms of muscular dystrophy.

“This is the first gene therapy clinical trial to demonstrate functional improvement in any form of muscular dystrophy, and a major advance for those suffering with muscle disease,” explained Mendell. As a consequence of the positive data from the first tests, the researcher announced that the team is now advancing to examine if the therapy can also benefit children suffering from Duchenne muscular dystrophy.

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The funding was granted by a series of liaisons from Duchenne Alliance as a recognition of the potential of developing a gene therapy based on follistatin. “This has truly been a community effort. Fifteen foundations from three continents collaborated to fund this project,” stated Steve Dreher of Hope for Gus. In addition, the project may also be selected to receive additional funding to Milo Biotechnology for follow-up studies. “With unprecedented speed to funding, our member foundations continue to accelerate the pace of Duchenne biomedical research,” said the Scientific Director of DA, Carlo Rago Ph.D.

The foundations that contributed to financing the project are the Americans Charley’s Fund, Hope for Gabe, Hope for Gus, JB’s Keys, Jett Foundation, Little Hercules Foundation, Michael’s Cause, Nash Avery Foundation, Pietro’s Fight, Ryan’s Quest, Team Joseph, and Two Smiles One Hope Foundation, as well as the English Duchenne Now and Harrison’s Fund, and the Australian Save Our Sons.