The Alzheimer’s Drug Discovery Foundation has awarded a grant of $210,300 to Amicus Therapeutics to evaluate small-molecule, orally delivered pharmacological chaperone compounds as a novel approach to treating Alzheimer’s disease.
The deal will capitalise on the link discovered by Amicus between various lysosomal enzymes and accumulation of the beta-amyloid and p-tau deposits observed in the brain of Alzheimer’s patients.
The ADDF’s award will fund initial pre-clinical proof-of-concept studies for a specific pharmacological chaperone that targets one of these lysosomal enzymes.
Amicus Therapeutics director of Exploratory Biology Brandon Wustman said the pharmacological chaperones are effective in animal models.
“We believe this research has the potential to lead to the development of new, disease-modifying therapies for Alzheimer’s disease, as well as the rarer lysosomal storage disorders that share a common pharmaceutical protein target,” Wustman said.