NIH Announces Grants for Frontotemporal Degeneration Research
The National Institutes of Health will award three large, five-year projects on a specific form of dementia, known as frontotemporal because of the areas of the brain that are affected. The projects, funded by the NIH’s National Institute of Neurological Disorders and Stroke (NINDS), National Institute on Aging (NIA) and the National Center for Advancing Translational Sciences (NCATS), announced total more than $5.9 million for 2014.
Approximately 50,000 Americans live with frontotemporal degeneration, or FTD, which causes severe behavioral changes and problems with language and cognition. As the disease progresses, individuals have difficulty planning activities, interacting with others and caring for themselves.
“The grants cover a wide spectrum of FTD research, from fundamental discoveries of the genetics behind this disorder to testing potential therapies in patients. We hope that these projects will provide answers and new avenues of treatment for this devastating condition,” said Walter Koroshetz, M.D., acting director of NINDS.
“The projects aim to advance our understanding of frontotemporal degeneration by improving diagnosis, identifying preventive strategies and providing new insights into the genetics underlying this complex disorder,” said Margaret Sutherland, Ph.D., program director at NINDS.
In 2013, NIH sponsored a workshop on Alzheimer's Disease-Related Dementias: Research Challenges and Opportunities. The workshop helped to identify gaps in dementia research and establish a set of priorities and goals. The new FTD grants address a number of the recommendations established at the workshop.
“This opportunity to identify the biomarkers that may signal the onset and progression of FTD in symptom-free volunteers with the familial form of the disease may one day lead to effective interventions,” said John Hsiao, M.D., a program director at NIA.
“These multicenter, multi-disciplinary projects will enable scientists to combine their areas of expertise to design novel approaches for FTD research, with the ultimate goal of providing treatments to more patients more efficiently,” said Pamela McInnes, D.D.S., M.Sc.(Dent.), deputy director of NCATS.
The grants are:
• Longitudinal Evaluation of Familial Frontotemporal Dementia Subjects
Principal Investigator: Bradley F. Boeve, M.D.; Mayo Clinic, Rochester, Minnesota; AG045390
Dr. Boeve and his colleagues will enroll 300 members of families with familial frontotemporal lobar degeneration with changes in genes associated with the disorder. Study participants will undergo annual brain scans, blood and cerebrospinal fluid analysis as well as behavioral and cognitive tests. Dr. Boeve’s team will use that data to identify biomarkers, which will help determine the effectiveness of potential therapies.
• The Frontotemporal Lobar Degeneration Clinical Research Consortium
Principal Investigator: Adam L. Boxer, M.D., Ph.D.; University of California, San Francisco; NS092089
Dr. Boxer and his colleagues will work with advocacy groups to establish a clinical research consortium to support development of FTD therapies. The groups involved in this project include: the Association for Frontotemporal Degeneration, Alzheimer’s Drug Discovery, Bluefield Project to cure FTD, CBD Solutions, Cure PSP and the Tau Consortium. The goals of the research network will be to improve clinical trial design and bring together a variety of techniques and methods to generate new types of treatments for FTD. The Frontotemporal Lobar Degeneration Clinical Research Consortium is part of the NIH NCATS Rare Diseases Clinical Research Network.
• Pathobiology of Neurodegeneration in C9ORF72 Repeat Expansion
Principal Investigator: Leonard Petrucelli, Ph.D.; Mayo Clinic, Jacksonville, Florida; NS084974
A specific change, or mutation, in the C9ORF72 gene is the most common cause of FTD and amyotrophic lateral sclerosis (ALS), a neurodegenerative disease that affects the muscles. Using cells in a dish, animal models and human tissue, Dr. Petrucelli and his team will comprehensively investigate the mechanisms underlying C9ORF72-related neurodegeneration and develop therapies to overcome the effects of the mutation.