Synopsis

Source of validity: Scientific
Rating (out of 5): 2

Insulin-like Growth Factor (IGF) and Muscular Dystrophy: Evidence Synopsis

Insulin-like growth factor 1 (IGF-1) has been investigated as a potential therapeutic agent for muscular dystrophy, particularly Duchenne Muscular Dystrophy (DMD). Scientific interest stems from IGF-1’s role in promoting muscle growth, regeneration, and reducing muscle atrophy. Preclinical studies in mouse models of DMD have shown that overexpression or administration of IGF-1 can increase muscle mass, improve muscle strength, and reduce fibrosis. These beneficial effects are attributed to IGF-1’s ability to stimulate satellite cell proliferation and differentiation, as well as its anti-apoptotic properties.

Despite promising results in animal models, clinical evidence in humans remains limited. Early-phase clinical trials and small pilot studies have explored IGF-1’s safety and efficacy, but results have been inconclusive or mixed. Some reports suggest mild improvements in muscle strength, while others do not demonstrate significant clinical benefit. Additionally, concerns about side effects such as hypoglycemia and abnormal growth have limited the widespread clinical adoption of IGF-1 therapy in muscular dystrophy patients.

In summary, the use of IGF-1 to support or treat muscular dystrophy is based on scientific rationale and supported by animal research, but currently lacks robust, high-quality evidence from large-scale human clinical trials. As such, IGF-1 use in this context is considered experimental, with moderate preclinical but weak clinical validation.

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