Parkinson’s disease is a slowly progressive chronic neurological disease, caused by the degeneration of dopaminergic (DA) neurons in the substantia nigra. Modern medicine still cannot cure the disease or slow down its progression. Only symptomatic treatment is currently available. We would like to utilize the potential of Glial cell line-derived neurotrophic factor (GDNF), which was discovered to be a survival factor for DA neurons (Lin, Doherty et al. 1993). GDNF is able to support and regenerate neuronal population within patients suffering from Parkinson’s disease. Since GDNF protein itself has poor pharmacological properties, we decided to develop small molecules that act similarly to GDNF. So far we have already identified several chemical compounds that activate GDNF receptors in cultured cells and promote survival of DA neurons in vitro. We are currently testing these molecules in animal model of Parkinson’s disease. Active ones will be further optimized to improve their potency and efficacy using computational and medicinal chemistry methods mastered by Molcode Ltd. Resulting compounds will be synthesized and tested first in cultured cells and afterwards in animal model of PD. Successful execution of this project will result in development of drug candidates with proven activity against PD in animal models.
