Ben-Gurion University of the Negev researchers have discovered that proteins called BMP5/7 are new promising potential therapeutics for Parkinson’s disease that could change the trajectory of the disorder. Their findings were published recently in the highly prestigious clinical neurology journal, .
Parkinson's disease, which affects over 10 million people worldwide, is characterized by the progressive degeneration of dopamine-secreting brain cells causing severe movement impairments. Today, it is thought that the protein alpha-synuclein, which is present in all human brains, plays a central role in the development of Parkinson’s disease by misfolding and forming toxic clumps in dopamine-secreting brain cells of affected individuals. Currently, all therapies used for Parkinson’s disease improve symptoms, however, their efficacy is unacceptably low in advanced stages of the illness and unfortunately, they do not stop or slow the disease progression.
Dr. Claude Brodski?, MD and his team discovered that in dopamine-producing brain cells of Parkinson’s disease patients BMP5/7 signaling was significantly reduced - a change that could possibly contribute to the development of the disease. To study this possibility, the scientists genetically engineered mouse brains to reduce BMP5/7 signaling in dopamine-producing brain cells. Confirming their suspicion, reduced BMP5/7 signaling indeed leads to an accumulation of toxic alpha-synuclein clumps and loss of dopamine-producing brain cells.
The researchers then hypothesized that if a lack of BMP5/7 signaling causes the loss of dopamine-producing brain cells, then administering BMP5/7 could protect these cells in a Parkinson’s disease model.
“Indeed, we found that BMP5/7 treatment can, in a Parkinson’s disease mouse model, efficiently prevent the accumulation of alpha-synuclein, loss of dopamine-producing brain cells and associated movement impairments. These findings are very exciting since they suggest that BMP5/7 could slow or stop the progression of Parkinson’s disease. Currently, we are focusing all our efforts on bringing our discovery closer to clinical application,” says Dr. Brodski of the Department of Physiology and Cell Biology, Faculty of Health Sciences and the Zlotowski Center for Neuroscience.
BGN Technologies, the technology transfer office of Ben-Gurion University, has filed several patent applications covering this breakthrough discovery.
Dr. Galit Mazooz Perlmuter, Senior?VP Business Development Bio-Pharma at BGN Technologies, said, "There is a huge unmet need for new therapies to treat Parkinson’s disease, especially in the advanced stages of the disease. Dr. Brodski's findings, although still in the early stages, offer a
disease-modified drug target that will address the mechanism of this devastating condition. We are now seeking an industry partner for further development of this patent-pending invention."
Source: Vitic et al., BMP5/7 protect dopaminergic neurons in an α -synuclein mouse model of Parkinson’s disease. Brain. 2020 Nov 30: awaa368. doi: 10.1093/brain/awaa368