Repurposing Dipyridamole in Niemann Pick Type C Disease: A Proof of Concept Study.

ABSTRACT: Niemann Pick type C disease (NPC) is a rare disorder characterized by lysosomal lipid accumulation that damages peripheral organs and the central nervous system. Currently, only miglustat is authorized for NPC treatment in Europe, and thus the identification of new therapies is necessary. The hypothesis addressed in this study is that increasing adenosine levels may represent a new therapeutic approach for NPC. In fact, a reduced level of adenosine has been shown in the brain of animal models of NPC; moreover, the compound T1-11, which is able to weakly stimulate A_2A receptor and to increase adenosine levels by blocking the equilibrative nucleoside transporter ENT1, significantly ameliorated the pathological phenotype and extended the survival in a mouse model of the disease. To

SUBMITTER: Pepponi R 

PROVIDER: S-EPMC8999038 | biostudies-literature | 2022 Mar

REPOSITORIES: biostudies-literature