They used the drug because they realized that both disorders result, in part, from an insufficient supply of a brain neurotransmitter called acetylcholine.
Neurotransmitters are substances that one neuron uses to trigger another in the brain.
Donepezil, trade name is Aricept, blocks a brain enzyme cholinesterase that typically breaks down acetylcholine, thus leaving more of the neurotransmitter available in the brain. While these are preliminary data, if it continues to prove beneficial to these patients, this may be a therapy we could start early in their lives and use on a continuing basis. All four patients experienced improved cognitive, adaptive and behavioral functioning during the 26 weeks they took donepezil. Based on the initial study's success, Duke researchers are now testing donepezil in a randomized and double-blind group of 20 adults with Down syndrome. In this study, each patient will receive the drug and the placebo at different times, and their performance during these periods will be compared.