The cholinesterase inhibitor drugs have been shown to improve memory in Alzheimer’s disease. They increase the level of a chemical in the brain called acetylcholine which is reduced in Alzheimer’s disease. It has been suggested that this chemical is also reduced in patients with cognitive impairment due to vascular disease, which CADASIL can cause. This study tested whether one of these drugs, called Donepezil, could improve memory and other thinking skills (cognition) in patients with CADASIL. The overall study was negative although there did appear to be a small effect on certain cognitive functions (which we refer to as executive function). However, this was small and did not seem to have a significant effect on an individual’s lifestyle.
This study is also important as it has shown that it is possible to carry out drug trials in CADASIL. Even though it is a rare disease, by collaborating across many countries and continents, it was possible to include a sufficient number of patients to answer this research question. Hopefully, we will be able to carry out further trials in the future when potentially useful drugs are discovered.
Reference: Dichgans M, Markus HS, Salloway S, Verkkoniemi A, Moline M, Wang Q, Posner H, Chabriat HS. Donepezil in patients with subcortical vascular cognitive impairment: a randomised double-blind trial in CADASIL. Lancet Neurol. 2008 Apr; 7(4): 310-8. Click here for PubMed abstract