Parkinson's disease (PD) is the second most common neurodegenerative disorder and is estimated to occur in about 1% of the population over the age of 55, costing up to $23 billion annually to the health care community. It is characterized by progressive motor and non-motor decline that substantially contributes to disability and loss of quality of life. There is no treatment that blocks the neurodegeneration and progression of the disease.
A large body of evidence indicates that α-synuclein (aSYN) is involved in the pathogenesis of PD and is a valid target for developing drugs to treat this disease. aSYN mutations and gene multiplications are known to cause familial PD and Lewy bodies, which are a neuropathological hallmark of PD, consist of aggregated aSYN in all PD patients, including the sporadic forms.
Our goal is to develop the drug Posiphen, which targets aSYN, to block neurodegeneration in PD as a novel disease modifying treatment of this disorder. Posiphen reduces expression of aSYN by binding to the 5’UTR region of aSYN mRNA to inhibit translation thereby reducing aSYN expression.
Posiphen lowers translation of aSYN (Rogers et al. 2011) as well as APP (Lahiri et al., 2007).
Posiphen decreases SNCA and APP levels dose-dependently in human brain cells. Cell viability was unaffected.