Congenital hyperinsulinism models for novel drug discovery

Source: The Orphan Disease Center

Awarded Grants

Congenital hyperinsulinism models for novel drug discovery

Awardee: Michael Kalwat

Institution: Indiana Biosciences Research Institute

Grant Amount: $70,200

Funding Period: February 1, 2024, to January 31, 2025

Summary:

Patients with congenital hyperinsulinism (HI) are in a continual battle to regulate their blood glucose levels. HI is caused by genetic mutations that lead to inappropriately high insulin levels in the blood. Insulin is normally released from beta cells within the pancreas only after meals when blood glucose is elevated. However, in HI these cells are dysfunctional and release too much insulin even when glucose levels are low. The only FDA-approved drug for HI, diazoxide, has side-effects and some patients are unresponsive. Therefore, new treatments need to be developed.

To accomplish this requires the creation of new methods that allow us to test drugs on cells which mimic the human disease. In our project, we will create a human beta cell model that mimics HI and we will test new drugs to determine their ability to suppress insulin release and their mechanisms of action.

To read more about the announcement, go to The Orphan Disease Center.