FSHD is a rare genetic disease that results in progressive muscle degeneration, atrophy, and weakness of selected muscles in the body. Image Credit: Allexxandar / Shutterstock.

Canadian venture philanthropy organisation Solve FSHD has awarded four grants to augment the understanding of facioscapulohumeral muscular dystrophy (FSHD) and the development of therapeutics for the condition.

FSHD is a rare genetic disease that results in progressive muscle degeneration, atrophy, and weakness of selected muscles in the body. Its prevalence is estimated to be four in 100,000 people with no cure for the disease, according to Muscular Dystrophy Association. The only treatment is symptom management to improve quality of life.

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There has been a renewed focus on gene therapies to treat muscular dystrophies following the US Food and Drug Administration’s (FDA) approval of the first-ever gene therapy for certain patients with Duchenne muscular dystrophy in June 2023.

The funding projects are worth a cumulative C$1.8m ($1.4m) spread across four groups. One of the Solve FSHD grant projects focuses on establishing trial outcomes for FSHD, by assessing skeletal muscle torque, as a functional outcome measure (to assess skeletal muscle strength) in an animal FSHD model.

Two other projects aim to evaluate therapeutics in the field. The first involves using a preclinical model to evaluate the effect of STAT3 inhibitors, a cell death-inducing cancer drug, on muscle pathology and fibrosis in FSHD. The second project will evaluate the use of fast skeletal muscle troponin activators (FSTAs); these drugs can slow the decline of muscle function, thereby improving muscle function in a preclinical FSHD model.

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The last grant project will work towards modelling FSHD developmental pathways by visualising interactions between the genome and the environment or epigenomic landscape, and defining the role of fibro-adipogenic progenitor cells—these differentiate into multiple cell types, including connective tissue— in the development of FSHD.

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Solve FSHD chairman Chip Wilson said: “We believe that fostering collaborations between leading minds across different scientific fields can only be beneficial in accelerating potential treatments to help those with FSHD.”

Other investigational drugs for FSHD that are at more advanced stages of development include miRecule/Sanofi’s anti-DUX4 RNA therapy, an antibody-RNA conjugate therapy, Fulcrum Therapeutics’ enzyme inhibitor (losmapimod), which is in a Phase III trial (NCT05397470), and Roche’s humanised monoclonal antibody at the Phase II stage (NCT05548556).