Parkinson’s disease (PD) is a chronic neurodegenerative disease characterized by deposition of α-synuclein-containing Lewy Bodies and a progressive degeneration of dopaminergic neurons in substantia nigra pars compacta, manifesting as motor symptoms such as tremor, rigidity, and bradykinesia. In addition to motor symptoms, non-motor symptoms e.g., cognitive dysfunction and depression often occur.
PD is the second most common neurodegenerative disorder and affects 1-2% of the population over 65 years of age, with estimated 10 million patients globally. This number is expected to grow rapidly in the next decades due to increased life expectancy of the general population. There is thus a growing need for medical treatments to treat PD. Current approved drugs focus entirely on treating initial symptoms of PD, and there are no drugs on the market that cure or modify the disease.
The cause of PD is unknown, however impaired glucose metabolism is indicated early in the disease and an upregulation of β-oxidation is observed in PD patients. Here at 2N Pharma, we believe that mitochondrial dysfunction has a central role in the progression of PD. We are currently testing this hypothesis by using Mitometin to treat PD-like symptoms in animal models of PD.
Most cases of PD are sporadic, meaning that there is no clear explanation or genetic component that is responsible for the development of the disease. There are, however, genetic mutations such as PARK2 and leucine-rich repeat kinase 2 (LRRK2) that are implicated in development of PD, which is why we are using both genetic and environmental toxin models to test the efficacy of Mitometin.