The National Institute of Neurological Disorders and Stroke and the National Center for Advancing Translational Sciences, part of the National Institutes of Health, have awarded University of Miami Miller School of Medicine neurologist Michael Benatar, M.D., Ph.D., a $6.24 million cooperative agreement award to establish a Rare Diseases Clinical Research Consortium that will study amyotrophic lateral sclerosis (ALS) and a group of related disorders.
This consortium will be part of the Rare Diseases Clinical Research Network (RDCRN), an initiative of the Office of Rare Diseases Research.
The consortium, which will be called CReATe (Clinical Research in ALS and related disorders for Therapeutic development), includes J. Paul Taylor, M.D., Ph.D., at St. Jude Children’s Hospital, as well as neurologists and scientists at the Mayo Clinic in Jacksonville, University of Kansas Medical Center, University of California San Diego, University of California San Francisco, Duke University, the Medical University of South Carolina and the University of Tübingen in Germany.
Key UM partners in this initiative include Stephan Züchner, M.D., Ph.D., professor and interim Chair of the Dr. John T. Macdonald Foundation Department of Human Genetics, Jacob McCauley, Ph.D., associate professor of human genetics and Associate Director of the Center for Genome Technology at the John P. Hussman Institute for Human Genomics, Evadnie Rampersaud, Ph.D., research assistant professor of human genetics and Director of the Division of Genetic Epidemiology at the Hussman Institute, Rebecca Schüle, M.D., Joanne Wuu, Sc.M., research assistant professor of neurology, Zane Zeier, Ph.D., assistant professor of psychiatry and behavioral sciences, and Claes Wahlestedt, M.D., Ph.D., Vice Chair for Research, Leonard M. Miller Professor of Psychiatry and Behavioral Sciences, Associate Dean for Therapeutic Innovation, and Director of the Center for Therapeutic Innovation.
Amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, is a neurodegenerative
disease that attacks the motor nerves, leading to progressive muscle weakness. There is no effective therapy for ALS and the lifespan for those affected is typically only three to five years.
Operating under the five-year cooperative agreement award, the CReATe consortium will carefully evaluate the relationship between phenotype and genotype in this group of motor neuron and related disorders, search for novel genetic causes of disease, identify genetic modifiers, and advance development of biomarkers that may aid therapy development. In addition, the consortium has the goal of training and mentoring fellows and junior faculty in clinical research related to ALS and related disorders.
“Funding for CReATe as part of the National Center for Advancing Translational Sciences’ Rare Diseases Clinical Research Network provides a unique opportunity to advance clinical and translational research in the area of ALS and related disorders,” said Benatar, who is Chief of the Neuromuscular Division and the Walter Bradley Chair in ALS Research. “We are particularly excited about the seamlessly translational nature of this consortium in which neurologists, geneticists and other scientists have joined forces to advance scientific progress toward effective therapies for this devastating group of diseases.”
A special and important aspect of CReATe is its partnership with a number of organizations that represent patients afflicted with this group of rare diseases. These include the ALS Association, the Muscular Dystrophy Association, the ALS Recovery Fund, the Spastic Paraplegia Foundation, the Association for Frontotemporal Degeneration, and the National ALS Registry and PatientsLikeMe.
Benatar said, “Clinical and translational research is motivated by the needs of patients and can only succeed if predicated upon genuine partnerships with patients and patient advocacy groups. Herein lies a unique underpinning of the CReATe consortium.”