Diabetes is known as a disease of uncontrolled blood-sugar levels because the body does not produce enough insulin, or the insulin that is produced is not used effectively by the body. Most people are not aware that type-2 diabetes is also the most prevalent amyloidosis. In type-2 diabetes, a small protein called Islet Amyloid PolyPeptide (IAPP) forms toxic aggregates that kill the insulin-producing beta cells in the pancreas.
A new study now shows that the molecular tweezer, CLR01, prevents the aggregation of IAPP and its toxicity towards pancreatic cells. The study, which is published in the American Chemical Society (ACS) journal ACS Chemical Biology, shows how CLR01 binds to IAPP, changes its structure, and prevents its aggregation. The study also shows that the blood levels of CLR01 needed for therapeutic effect can be achieved without causing safety concerns.
The new study was led by Gal Bitan‘s group at UCLA in collaboration with Thomas Schrader and Frank Klärner, University of Duisburg-Essen, Elsa Sanches-Garcia, Max-Planck-Institut für Kohlenforschung in Mülheim, and Chunyu Wang, Rensselaer Polytechnic Institute.