Aquinnah Pharmaceuticals recently announced the preclinical findings on its novel therapeutic designed to slow or stop the progression of Alzheimer’s Disease (AD) and related disorders. Their drug discovery relies on identifying compounds capable of eliminating persistent stress granules. Stress granules are aggregates of proteins and RNA molecules formed as a cell survival process in response to stress. In neurodegenerative diseases, they co-localize with pathological protein aggregates. In tauopathies such as AD, frontotemporal dementia, progressive supranuclear palsy, corticobasal degeneration, stress granules co-localize with tau aggregates. This lead compound targets the interaction of stress granules with pathological tau, the key contributor to loss of memory and cognition in AD.
The compound removed ~70% of tau pathology, measured by three different markers of pathological tau, in an AD model with advanced stage disease. The compound’s unique mode of action gives it the potential to work in combination with the recently FDA approved anti-amyloid drugs. Additionally, it also has the potential to treat other tauopathies including traumatic brain injury.
If it succeeds in clinical trials and gets FDA approval, this once-a-day oral pill would be a less costly and easier medication option for patients over the currently approved injectable AD immunotherapies. The latter often leads to amyloid related imaging abnormalities in the form of cerebral edema, micro-hemorrhage, seizures and even death. Currently, Aquinnah’s compound has a potential for therapeutic against tauopathies although its side effects are yet unknown.
