a global killer
Aggregation of the toxic peptide Amyloid beta (Aβ) is one of the most frequent afflictions of the elderly brain, a pathological process that becomes increasingly evident as the population ages. Hemorrhagic stroke due to the accumulation of Aβ around the brain’s vasculature, known as Cerebral Amyloid Angiopathy (CAA), is the leading cause of stroke in the elderly. The toxic peptide aggregates within the walls of the arteries, causing the arteries to rupture and bleed into the brain.
CAA is a sporadic disorder that affects millions of patients worldwide, but it also has a genetic subtype known as HCHWA-D. In this rare population, a specific mutation known as the Dutch mutation causes the process of vascular Aβ aggregation to accelerate – leading to the early onset of stroke. HCHWA-D is an ultra-genetic disorder with an extremely well-defined patient population in a single
The predictability of early disease onset in HCHWA-D patients offers a clear clinical path that will allow us to study a potential therapy in a well defined orphan patient population for which no medicines currently exist. Moreover, the fact that CAA and HCHWA-D are highly related means that we intend to use what we learn in HCHWA-D as a gateway to CAA. Simply put – we have the opportunity to develop a therapy for an incredibly rare disease, and it will tell us if it could work in a global one.