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The Michael J. Fox Foundation Launches LRRK2 Cohort Consortium

The Michael J. Fox Foundation (MJFF) launched the LRRK2 Cohort Consortium, a group of eight separate groups funded by MJFF to assemble and study groups of people with and without Parkinson’s disease (PD) who carry mutations in the LRRK2 gene. LRRK2 is more frequently found in cases of PD than any other gene discovered to date, particularly in certain populations including Ashkenazi Jews, North African Berbers, and Asians of Chinese descent.

Comprising more than 3,000 people across 20 clinical sites worldwide, the Cohort Consortium is one element of The Michael J. Fox Foundation’s multi-pronged approach to speeding development of LRRK2-based treatments for Parkinson’s disease. Diverse populations are being assembled and studied over time to help scientists gain a better understanding of the clinical features of LRRK2 Parkinson’s. By building a network of patients and their families, and compiling significant clinical data on LRRK2 parkinsonism over time, MJFF hopes to facilitate the design and execution of conclusive clinical trials once promising drug candidates are identified.

Scientists in the Cohort Consortium will share clinical data, which will be housed at a central repository at the University of Rochester in Rochester, New York. MJFF will coordinate the standardized collection of various biosamples from individuals with LRRK2 mutations, and make them available to the research community.

“One of our top priorities at MJFF is to find ways to alter the progression of PD. By studying individuals with the LRRK2 mutation, we hope to learn more about why certain populations are susceptible to PD,” said Brian Fiske, PhD, director of research programs at MJFF. “By investigating how this mutation works in people with the disease, we also can find out more about Parkinson’s on the whole, accelerating development of therapies that will benefit everyone with PD, not just those with mutations in LRRK2.”

The Cohort Consortium was piloted in 2009 to assemble cohorts of Ashkenazi Jews in New York and Tel Aviv and North African Berbers in Tunisia. Today the Consortium has expanded to include additional sites across the United States, Canada, China, Germany, Norway, and Spain.

Initial findings from cohort studies to date already have led MJFF to establish working groups to validate results related to issues including posture and gait disturbances, a LRRK2-cancer link, and impaired sense of smell as a pre-diagnostic biomarker for LRRK2-related PD.

LRRK2 is a high-priority research area for MJFF, which, with leadership funding from the Brin Wojcicki Foundation, has invested more than $33 million in research projects devoted to LRRK2 to date. In addition to the Cohort Consortium, the Foundation is funding and coordinating the LRRK2 Consortium, a collaborative network of more than 30 investigator teams studying LRRK2's structure and function in order to identify drug candidates that might modify its activity in the body and the brain. MJFF is also developing LRRK2 tools to speed the research process. The goal is to attack LRRK2 on multiple fronts simultaneously, rather than serially, in order to efficiently lay the groundwork for effective LRRK2 clinical trials once appropriate drug candidates have been found.

Pharmaceutical companies are already showing an interest in developing drugs to target the mutation. LRRK2 is a type of protein called a kinase, which in LRRK2 parkinsonism, is slightly overactive. Industry already has extensive experience developing kinase inhibitors to treat cancer. As of yet, there are no specific drugs in clinical testing to treat LRRK2 parkinsonism, but drug companies are actively pursuing research that could lead to potential therapies.

In conjunction with the clinical cohorts, the Foundation has established a LRRK2 Industry Advisory Group, including representatives from Pfizer, Elan, Sanofi-aventis, Eli Lilly, MerckSerono, and others. The group’s goal is to ensure that LRRK2 therapeutic development is carried out in a way as useful as possible to the pharmaceutical companies who will ultimately bring LRRK2 treatments through clinical testing and regulatory approval. Members of the group come together in a precompetitive space to discuss how to best create and share resources that will most effectively push LRRK2-based drugs closer to the clinic.

Researchers on the pilot study of Ashkenazi Jews include: Susan Bressman, MD, chairman of the department of neurology at Beth Israel Medical Center; Nir Giladi, MD, chairman of the Tel Aviv Sourasky Medical Center; Karen Marder MD, MPH, Sally Kerlin Professor of Neurology at New York Presbyterian Hospital, Columbia University Medical Center; Avi Orr-Urtreger, MD, PhD, director, the Genetic Institute, Tel Aviv Sourasky Medical Center; and Rachel Saunders-Pullman MD, MPH, MS, associate professor of neurology at the Albert Einstein College of Medicine.

Researchers on the pilot study of North African Berbers include: Matt Farrer, PhD, Canada Excellence Research Chair in Neuroscience at the University of British Columbia; Fayçal Hentati, MD, director, the department of neurology and neurobiology laboratory at the National Institute of Neurology in Tunis, Tunisia; and John Duda, MD, co-director of the Parkinson's Disease Research, Education and Clinical Center of the Philadelphia VA Medical Center.

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