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Biofluid Biomarkers Hold Promise for the Future of Huntington Disease

By Erica Slaughter

Huntington disease (HD), a genetically dominant trinucleotide repeat disorder resulting from cytosine-adenine-guanine (CAG) repeats within the huntingtin (HTT) gene, has a near-full pene- trance and onset of symptomatology and neuronal loss typically by the third decade of life.

A neuropathological hallmark of HD is cortical atrophy, as indicated in postmortem assessments. These studies have been limited, however, because the assessments were performed on tissue of neuropathological grades 2-4. Cortical atrophy at early stages of HD, before symptoms develop, remains unclear.

There have been few reliable and quantifiable indicators of disease severity, progression, or phenotype, which has limited therapy development. Currently, there are no disease-modifying medications and few interventions available to effectively alleviate symptoms. The lack of indicators of pathobiological processes at the molecular level has limited the ability to prove efficacy and safety in clinical trials.

About Mitochon Pharmaceuticals
Mitochon was founded in 2014 with the mission to develop treatments for insidious diseases through the modulation of mitochondrial physiology, with applications to neurodegeneration, neuromuscular, and developmental diseases. Mitochon’s lead programs, MP101 and MP201, specifically harnesses the power of the mitochondria to provide broad neural protection. These compounds elicit mild increases in energy expenditure that result in strengthening cellular survival—similar to the positive effects seen with fasting and exercise. These compounds also induce an important neurotrophin, Brain Derived Neurotrophic Factor (BDNF), involved in cognition and neural growth. Mitochon is supported by Ben Franklin Technology Partners Southeastern PA, an initiative of the Pennsylvania Department of Community and Economic Development funded by the Ben Franklin Technology Development Authority.