JACKSONVILLE, Fla. — For his work contributing to a potential new treatment approach for Parkinson's disease, the Michael J. Fox Foundation (MJFF) for Parkinson's Research has awarded a $500,000 grant to a neuroscientist at the Mayo Clinic campus in Florida.
The researcher, Matthew Farrer, Ph.D., studies how a gene, known as LRRK2, functions normally within brain nerve cells (neurons), and also how it can go awry when mutated. Dr. Farrer was part of the original team who discovered the link between LRRK2 and Parkinson's disease in 2004. A year later, he described LRRK2 G2019S, believed now to be the most common genetic risk factor for Parkinson's disease.
For these reasons, MJFF has identified LRRK2 as a high-priority therapeutic target and has invested nearly $17 million to date for a broad and integrated strategy to drive critical LRRK2-related initiatives at every stage of drug development, according to the foundation. As part of those awards, on Feb. 17, MJFF announced grants of $3.5 million to nine research studies.
Dr. Farrer's work in teasing apart the function of LRRK2 in dictating the structure of neurons earned him the two-year grant. The co-principal investigator on the grant is Justus Daechsel, Ph.D., an assistant professor of Molecular Neuroscience at Mayo Clinic.
"At Mayo, our molecular neuroscience research is now focused on the genetic basis of Parkinson's disease, on the specific proteins and the molecular pathways that are affected," Dr. Farrer says. "Our model and drug development program is designed to halt disease progression, not just to treat the symptoms."
Dr. Farrer and his team have genetically altered mice so that they do not have the LRRK2 gene. When brain cells from these animals are grown in laboratory culture, the researchers have found that neurons without LRKK2 branch out more than do neurons with LRRK2, which shows that the gene normally helps regulate neuronal architecture.
They are using the mice to further understand the function of the protein produced by the LRKK2 gene. "It is a large protein, with many parts. So we are going to replace pieces of the protein, with and without the mutations that predispose people to develop Parkinson's disease, back into cells derived from these animals to see what roles different regions of the protein play," he says. They will also delete other proteins known to interact with the LRRK2 protein to see their effect.
"Answers to these questions are critical for future drug development for patients with Parkinson's disease, and we are pleased the support of the Michael J. Fox Foundation can help us reach our common goal," Dr. Farrer says.
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