Milo’s Gene Therapy is a Hope for Destructive Muscle Disease
Follistatin gene therapy sparked hope for the treatment of a rare degenerative and inflammatory muscle disease – sporadic inclusion body myositis (sIBM). U.S. based Milo Biotechnology dedicated towards development of novel therapies for rare neuromuscular diseases announced promising safety and initial efficacy data of follistatin gene therapy treated sIBM patients.
The study was conducted in Nationwide Children’s Hospital on 9 patients for time period of 4 years. Six of them who received the therapy in both legs showed improvement of 3.1 meters per month in 6 minute walk test (6MWT) compared to the 12 patients who were untreated and showed declination of 2.3 meters per month in 6MWT.
sIBM is a non hereditary, rare muscle disease which shows progressive weakness and wasting of arms and leg muscles. It leads to inflammatory myopathy in adults above the age of 50. Follistatin is an autocrine glycoprotein encoded by FST gene in humans. During a research in 2009, follistatin resulted in the growth of muscle and improved strength in monkeys. Thereby, it indicated the potential for the treatment of muscle degenerative diseases.
Figure 1. Normal vs Mighty Mouse. An example of follistatin gene therapy. (photo credit: Se-Jin lee
Dr. Mendell and Dr. Brian Kaspar of Nationwide Children’s Hospital developed the Milo’s lead candidate, AAV1-FS344 which causes local expression of follistatin, a potent myostatin inhibitor. The gene therapy was based on adeno-associated virus (AAV) delivery of follistatin 344 (FS344) for prevention of muscle degeneration and improvement of muscle strength. Its intellectual property was exclusively licensed to Milo from Nationwide Children’s Hospital in 2012.
Other than sIBM, follistatin gene therapy can also find its application in the treatment of muscle wasting induced by cancer treatment and ageing, muscle dystrophies. Clinical studies for Duchenne muscular dystrophy treatment is going on.
Dr. Mendell, M.D., Director of Nationwide Children’s Hospital’s Center for Gene Therapy and PI on the trial, presented the data.
“From our observations, follistatin gene therapy is showing significant promise for patients with sIBM and other muscle-wasting disorders. We were gratified to discuss these encouraging findings with our colleagues at the ASGCT annual meeting, and we look forward to the day when this therapy becomes a standard treatment for patients affected by neuromuscular disease,” Dr. Mendell noted.
Currently, there is no standard treatment for sIBM. Symptomatic management can be done by physical exercises. Novartis reported BYM338 (bimagrumab)-a novel, fully human monoclonal antibody which is being developed for sIBM treatment and other pathological muscle loss conditions.
Featured image credit: Healthcare and Gene Therapy as a Science Concept © kentoh (Stock Photo ID: 118603256)