Methods and Compounds for Treating Mitochondrial Diseases

Abstract

Researchers at the University of California, Davis have developed a treatment for mitochondrial disease through a repurposing approach whereby a library of FDA-approved drugs was screened for previously unknown therapeutic effectiveness in these diseases.

Full Description

Mitochondrial diseases are a clinically heterogeneous group of disorders of mitochondrial metabolism that can arise from a genetic mutation in nuclear or mitochondrial DNA. There are several inherited mitochondrial diseases, including Leber's hereditary optic neuropathy (LHON), MERRF, MELAS, NARP, and Leigh's syndrome, which affect about 50,000 people in the US. Additionally, Parkinson's disease, which affects about 1 million people in the US, is thought to result from mitochondrial defects. In many of these diseases there is visual function loss, which is particularly severe in patients with LHON. Inherited forms of mitochondrial diseases have high rates of mortality and morbidity. Currently, there are no FDA-approved therapies for mitochondrial disease, and there remains a need for pharmaceutically effective drugs to treat these conditions.

Researchers at the University of California, Davis have developed a treatment for these mitochondrial diseases through a repurposing approach to screen existing libraries of FDA-approved drugs for previously unrecognized therapeutic effectiveness. Using a mouse model, identified drugs have been shown to protect mice from mitochondrial blindness, and have the potential of slowing down or arresting the progression of mitochondrial disease. These molecules represent drugs that potentially go beyond treating symptoms to be the first curative therapy for mitochondrial disease.

Applications

Features/Benefits

• Potential curative therapy as opposed to treating disease symptoms

Patent Status