Use of Quercetin and Related Flavonoids for the Treatment of Myotonic Dystrophy Types 1 and 2

Background:

Myotonic dystrophy (DM) is the most common form of adult muscular dystrophy. It is a multisystemic disorder affecting nearly all organs including muscles, brain, heart, eyes, and the GI tract, among others. Symptoms of the disease are numerous and include myotonia (inability to relax muscles), muscle atrophy, cognitive impairment, sleep disturbance, Christmas tree cataracts, cardiac conduction defects, and insulin resistance among others. Recent estimates of disease prevalence for DM suggest that the potentially affected population could be over 150,000 individuals in the US alone. There are currently no approved disease-targeting therapies for the treatment of DM.

Technology Overview:

This technology uses the natural flavonoid compound quercetin and related flavonoids for the treatment of DM types 1 and 2. We have identified quercetin as a novel therapeutic lead that rescues molecular, cellular, and physiological markers of disease in DM1 and DM2 patient cell lines and in a transgenic DM1 mouse model. Quercetin can be administered in a number of ways, including beverage form or ingested supplement. Quercetin and related flavonoids, either alone or in combination with compounds that selectively upregulate muscle-blind-like (MBNL) levels, represent a novel therapeutic strategy for the treatment of DM. In addition, quercetin may offer potential as a treatment in the broader repeat expansion disorder family.

Advantages:

•    Provides a potential first treatment for myotonic dystrophy (DM), a disease that affects many thousands of patients.
•    May also offer potential for the development of therapeutics in the broader repeat expansion disorder family.

Applications:

The primary application for this technology is the treatment of DM and potentially other disorders.

U.S. Provisional Patent 63/421,937, filed 11/4/22.

Stage of Development

TRL 3 - Experimental proof of concept

Licensing Status:

This technology is available for licensing.

Licensing Potential:

This technology would be of interest to anyone involved in the development of therapeutics of DM and other repeat expansion disorders, including:
•    Pharmaceutics companies.
•    Hospitals.
•    Medical research laboratories.
•    Universities.