NEW YORK--(BUSINESS WIRE)--A study by Columbia University Medical Center partially funded by CurePSP, the leading nonprofit advocacy organization focused on prime of life neurodegenerative diseases, has identified a class of drugs that leads to the removal of abnormal proteins from the brain in mouse models, thereby improving memory. With further study and clinical trials, this treatment could potentially lead to developments that help improve health and memory in neurodegeneration patients.
The study was conducted by Dr. Natura Myeku and her team of investigators in the laboratory of Dr. Karen E. Duff, professor of pathology and cell biology at Columbia. It is being published this month by Nature Medicine, a leading biomedical research journal. CurePSP provided $100,000 to support the study.
“This study is groundbreaking because is boosts activity in the brain’s ‘garbage disposal’ system and can decrease levels of toxic proteins associated with neurodegenerative diseases, including Alzheimer’s disease,” said Dr. Alexander Klein, Vice President-Scientific Affairs at CurePSP. “CurePSP funded this research because we saw promise in the study that Dr. Myeku and Dr. Duff proposed and that promise was realized.”
Dr. Myeku’s study involved tau, a critical brain protein that can assume a misshapen form and aggregate in the brain, leading to neurodegeneration. Dr. Myeku examined the mechanism involved in the accumulation of the tau protein in the brains of patients who suffer from progressive supranuclear palsy (PSP), frontotemporal dementia (FTD) and related neurodegenerative diseases. The study showed that aggregated tau protein profoundly impairs proteasomes -- protein complexes that dispose of old and damaged proteins in the brain -- that are essential to healthy brain function.
The study also found that in mice, FDA-approved Rolipram increased activity of brain proteasomes, promoting the removal of abnormal tau and improving memory. Dr. Duff’s lab is now investigating drugs in the same class as Rolipram that could have similar effects on humans without Rolipram’s side effects. Their goal is to identify and develop new therapies for neurodegenerative diseases, especially tau-based diseases such as PSP, FTD and others, including Alzheimer’s disease, by “repurposing” drugs that are already approved by the FDA. These could be employed in clinical trials quickly because of their preexisting approval.
Over the last 20 years, Dr. Duff’s lab has genetically engineered various mouse models for the study of Alzheimer’s disease, tau-related FTD and Parkinson’s disease. Dr. Duff has been a leader in studying the underlying causes of neurodegenerative diseases and in developing therapeutic approaches to their treatment, including the identification and development of novel drugs.
CurePSP is the leading nonprofit advocacy organization focused on prime of life neurodegenerative diseases – a spectrum of fatal brain disorders that often strike during a person’s most productive and rewarding years. Currently there is no treatment or cure for these disorders, which affect more than 150,000 people in the U.S. alone. Since it was founded in 1990, CurePSP has funded more than 160 research studies primarily in progressive supranuclear palsy (PSP) and the related disease corticobasal degeneration (CBD) and is the leading source of information and support for patients and their families, other caregivers, researchers, and doctors and allied healthcare professionals. CurePSP is based in Timonium, MD, with an office in New York City. Please visit http://www.psp.org/ for more information.