Parkinson's disease is characterized by the selective loss of dopamine neurons, which leads to reduced control of voluntary locomotor activities. The cause of this degeneration is unknown; however, recent data from animal models of PD suggest that a core structure of the neuron, namely microtubules, fall apart.
Microtubules provide structure to the neuron -- the framework within the cell. Additionally, microtubules also function in transporting nutrients and chemical messengers like dopamine within the cell. Therefore, a breakdown of microtubules can lead to a loss of function and ultimately death of the neuron.
Previous research by Professor Illana Gozes and collaborators found that a small protein fragment called NAPVSIPQ or NAP peptide protected neurons from a variety of toxic insults and improved cognitive function in animal models of Alzheimer's, stroke, and traumatic brain injury. The mechanism of action appears to involve stabilization of the microtubular network, thereby promoting cell survival.
This proposed research project will expand these findings and focus on exploring the ability of NAPVSIPQ peptide to protect dopamine neurons to chemical insults and to test whether this compound can prevent degeneration in animal models of Parkinson's disease. NAPVSIPQ peptide is currently in Phase 2 clinical trials, thus the goal of this project is to rapidly provide scientific justification to evaluate the potential of NAPVSIPQ in Parkinson's disease patients.
Final Outcome
Allon researchers presented promising results of their peptide showing neuroprotective capabilities in a traditional model of PD. As a result, MJFF staff has brokered a partnership between Allon and Marie-Françoise Chesselet, a member of MJFF’s Scientific Advisory Board, to continue animal testing of this peptide in the alpha-synuclein transgenic pre-clinical model.