Investigational Therapies Targeting New Mutations and Atypical Proteins May Have Potential to Deepen Understanding of Frontotemporal Degeneration
The Alzheimer’s Drug Discovery Foundation (ADDF) and the Association for Frontotemporal Degeneration (AFTD) announced today the recipients of their seventh annual partnership awards program to accelerate drug discovery for frontotemporal degeneration (FTD), a devastating form of dementia characterized by profound changes in behavior, personality, language and movement. Jeffrey Rothstein, MD, of Johns Hopkins University School of Medicine and Donald Lo, PhD, of Duke University Medical Center were each awarded $150,000 to conduct new research focused on developing novel drugs to treat FTD.
Dr. Rothstein is investigating therapies that target a newly discovered mutation in FTD and amyotrophic lateral sclerosis (ALS) patients, a mutation believed to cause brain damage through a mechanism called RNA toxicity. These studies, being investigated in collaboration with ISIS Pharmaceuticals, will build on Dr. Rothstein’s prior work and aims to develop biomarkers, or drug efficacy markers, for clinical trials.
Dr. Lo’s novel investigational therapies aim to protect FTD patients against neurodegeneration caused by defective forms of the protein tau that are found in the brain. Through collaboration with Regulus Therapeutics, Dr. Lo’s proprietary brain tissue screening platform will be combined with custom drug lead libraries for this project.
“We are so excited to provide early stage funding to Drs. Rothstein and Lo to further study their respective therapies to treat FTD,” said Diana Shineman, PhD, director of Scientific Affairs for ADDF. “This new research will not only provide the scientific community with a deeper understanding of this under-studied disease, but also provide patients and caregivers with the hope for potential treatments for FTD.”
“Through continuous support for research on FTD, we can raise awareness, develop effective treatments and ultimately prevent this brain disorder,” said Nadine Tatton, PhD, scientific director for AFTD. “We are so proud to partner with ADDF to continue this award program for the seventh consecutive year and highlight some of the revolutionary studies being done to aid those who cope with this disease.”
Since 2007, the ADDF/AFTD partnership program has provided more than $2.4 million toward 22 research projects around the world. FTD is the most common cause of dementia for people under the age of 60, most frequently affecting people between the ages of 40 and 65. As with Alzheimer’s disease, there are currently no drugs to treat, prevent or cure FTD.
About the Association for Frontotemporal Degeneration (AFTD)
The Association for Frontotemporal Degeneration (AFTD) is a not-for-profit organization created in 2002, originally named The Association for Frontotemporal Dementias, to advocate for more funding into the causes and treatments for frontotemporal degeneration (FTD), as well as to provide caregivers and patients with a central place to find information and support. Mounting evidence that these debilitating disorders are more prevalent than was originally thought and the limited information and support available for caregivers and families, along with the lack of funding for research, inspired a group of dedicated caregivers, clinicians, and researchers to create an organization to address these unmet needs. For more information, please visit www.theAFTD.org.