Yasoumi Ouchi, Ph.D., M.D., Hamamatsu University; Toshiharu Suzuki, Ph.D., Hokkaido University
Competition Sponsor: Japan Agency for Medical Research and Development
While many medical trials such as antibody therapies for amyloid to combat Alzheimer’s disease (AD) are still in vain due to a difficulty in curing the disease, a new drug with a novel mechanism acting on unique targets has been expected to develop. This project entails a novel peptide drug for treating AD. The drug candidate is based on a newly discovered molecule Alcadein β which is intrinsic in the brain. Alcadein β is known to be cleaved to p3-Alcβ, a brain-derived peptide that functions as a suppressor of AD progression. Experiments also show that a decrease in the p3-Alcβ level in the brain relates to generation of dementia and that a peripheral administration of this peptide drug compensates its reduction, allowing to prevent from the development and progression of the disease. Removing a chance of AD onset has a great impact on the welfare in our recent longevity society. We will show substantial evidence on biochemical and pathophysiological changes in the brain before the onset of dementia and the effectiveness of our peptide drug acting on them in vitro and in vivo experimental settings using rodents and monkeys. Currently, we are performing animal experiments to verify the drug safety and a good transdermal delivery of the drug to the brain as a necessary step before first administration to the human (FIH) in a clinical trial. It is expected that the use of a newly-developed device enabling controlled transdermal absorption of the peptide drug allows the optimal drug-delivery to the brain. With a preclinical proof of concept in the efficacy of this drug in vitro and in vivo, we plan to start the clinical trial not only at an early stage of the disease but also during a preclinical period to prove its effectiveness in humans in our already-established cohorts.
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