MicroRNA-targeted Therapies for Muscular Dystrophies and Other Inflammatory Disorders

Summary

Researchers at Children's National Hospital have developed a strategy of targeting microRNAs (miRNAs) to treat muscular dystrophies and other inflammatory disorders. This miRNA-based approach can be used independently to treat inflammation in either muscle disorders or other inflammatory diseases, or as a complementary approach to improve efficacy and reduce side effects of current glucocorticoid-based treatments.

Description

Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness, and chronic inflammation. The onset of DMD symptoms such as muscle weakness begins as early as age 3. The current standard-of-care for DMD is the chronic treatment with high dose of corticosteroids (prednisone, deflazacort). This increases patient strength, prolongs movement, and reduces scoliosis. However long term corticosteroid use is also associated with detrimental side effects such as weight gain, diabetes, stunted growth, bone fragility, mood disturbance and adrenal suppression. CNMC inventors have characterized two sets of microRNAs: 1) an “inflammatory set” that is elevated in DMD as well as other inflammatory and autoimmune diseases, and 2) a “steroid side effect set” that is elevated in DMD and becomes further increased with steroid treatment. Moving forward, CNMC researchers are will “block” these microRNAs as a novel therapeutic strategy to 1) treat inflammation in DMD and in other inflammatory disorders, and 2) counteract the side effects of chronic corticosteroid use. The following are the advantages offered by this therapeutic approach:

1. Effective anti-inflammatory treatment for muscular dystrophy and other inflammatory disorders.
2. Safer treatment option compared to steroids and glucocorticoids.
3. Reduces steroidal side effects of current muscular dystrophy therapy.

Applications

• Therapy for diseases with inflammation: Inflammatory bowel disease, arthritis, asthma and multiple sclerosis
• Therapy for other muscle diseases, such as DMD, BMD, Myositis, Limb Girdle MD, Myoshi Myopathy, Cancer Cachexia and Sarcopenia
• Co-therapy for the above muscle and inflammatory disorders to reduce drug side effects
• Addresses both pediatric and adult populations

Stage of Development

• Preclinical studies ongoing. Conserved miRNA behavior confirmed between animal models and human patients.

Intellectual Property Status

• Provisional Patent Application filed

This technology is available for exclusive or non-exclusive licensing.