MJFF initiative targets LRRK2 gene biomarkers, therapies
Aim is to identify promising strategies, foster collaborations
The Michael J. Fox Foundation for Parkinson’s Research (MJFF) announced an initiative to accelerate the identification of biomarkers and the development of new therapies targeting the LRRK2 gene, one of the most common causes of inherited Parkinson’s disease.
The program, LRRK2 Investigative Therapeutics Exchange, or LITE, provides “tens of millions of dollars” in grants over three years to support projects focusing on translating basic science discoveries into drug development, the foundation said. More than 30 academic and clinical researchers and more than 12 companies have expressed interest in the program, according to MJFF.
“We’re building a translational research engine that diversifies ways to target LRRK2, improving confidence and clarity in the most promising approaches to targeting the pathway,” Shalini Padmanabhan, PhD, MJFF‘s head of translational research, said in a foundation press release. “This program will make therapeutic development faster and more informed while ‘de-risking’ industry investment.”
Mutations in the LRRK2 gene are among the most prevalent genetic factors associated with Parkinson’s disease. The gene is responsible for producing a protein that plays a crucial role in cellular communication and maintaining cellular balance through recycling and waste-disposal processes. These mutations lead to an excessive activation of the LRRK2 enzyme, triggering cellular dysfunction and leading to the development of the disease.
Several treatments for LRRK2-Parkinson’s are in clinical trials, and several other candidates have been identified. Research also suggests that LRRK2 targeting therapies may be beneficial for Parkinson’s patients without mutations.
‘A chance to clarify key points of understanding’
The LITE program will be led by University of Dundee professor Dario Alessi, PhD, who runs a lab focused on the study of kinases, a class of proteins that includes LRRK2. Esther Sammler, MD, PhD, a neurologist with LRRK2 expertise at the university, will serve as co-principal investigator.
“LRRK2 presents key opportunities to the field, both for better understanding Parkinson’s and for treating it,” said Alessi, who directs the Dundee Signal Transduction Therapy Unit, a collaboration between the university and pharmaceutical companies. “The LITE initiative gives us a chance to clarify key points of understanding and use that knowledge to inform drug development.”
The program will connect companies developing LRRK2-targeting therapies with researchers and establish best practices for preclinical and clinical studies to advance therapeutics. It will also establish infrastructures to test new clinical biomarkers.
Current initiatives including the Aligning Science Across Parkinson’s Collaborative Research Network (CRN), the Parkinson’s Progression Markers Initiatives , and the Global Parkinson’s Genetics Program, will contribute to and support the new program.
At the end of funding period, the program is expected to have tested several strategies targeting LRRK2 and have identified lead molecules that could be further investigated by the research community or in collaboration with companies; to have identified and tested biomarkers that could be used in future clinical trials; and to have set up an infrastructure and model to be expanded to other emerging targets for Parkinson’s.
“Through the initiative, we see the potential for transformative advances in understanding Parkinson’s, intervening in the disease process and, we hope, stopping Parkinson’s disease in its tracks,” said Todd Sherer, PhD, chief mission officer at MJFF.
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