New Follistatin Gene Therapy Improves Function in Becker Muscular Dystrophy

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

Follistatin Gene Therapy

Follistatin Gene TherapyThe results from Milo Biotechnology’s Follistatin Gene Therapy were published in the Molecular Therapy journal showing therapy efficacy in increasing muscle strength and function in Becker’s muscular dystrophy patients. The study is entitled “A Phase I/IIa Follistatin Gene Therapy Trial for Becker Muscular Dystrophy.”

The study followed six patients with Becker’s muscular dystrophy for six months (three patients) up to a year (three patients), treated with a potent myostatin antagonist, follistatin, delivered via adeno-associated virus (AAV). In the study, a modified version of follistatin was developed, an alternatively spliced FS344, to prevent potential off target-binding (i.e., binding of the molecule of therapeutic interest to a protein target other than the primary target for which it was designed). The therapy was designed to increase muscle strength and prevent muscle wasting and was developed at Nationwide Children’s Hospital by Dr. Mendell and Dr. Brian Kaspar.

In the trial, AAV1.CMV.FS344 was delivered by direct bilateral intramuscular quadriceps injections. The majority of patients exhibited a significant increase in six-minute walk distance and in stair climbing tests, that were accompanied with improved muscle fiber regeneration and reduced muscle fibrosis and central nucleation. No adverse effects were encountered.

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Jerry Mendell, M.D., Professor at Nationwide Children’s Hospital and the study’s lead author commented, “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.”

Brian Kaspar, Ph.D., Associate Professor at Nationwide Children’s Hospital, author and compensated advisor and scientific founder of Milo noted, “Upon evaluating the top-line data in this trial, we are particularly impressed with the effects of our gene therapeutic to express follistatin long term, not only for the functional improvement, but also in improving the state of muscle health in the patients analyzed.”

Milo Biotechnology CEO Al Hawkins added, “This significant and long lasting clinical effect provides the basis for planning pivotal clinical studies beginning in 2015; we want to speed delivery of this important therapy to patients with debilitating muscular dystrophies.”