Small Molecules for Islet Expansion in the Treatment of Diabetes

University of California System: University of California, Los Angeles - UCLA
posted on 04/18/2012

UCLA researchers have identified a number of small molecules that act through a common molecular pathway to expand islets for diabetes therapy.

Suggested Uses

  • Ex vivo expansion of the number of donor pancreatic islets available for transplantation.
  • Increasing the lifespan and function of these donor islets in the diabetic recipient following transplantation.
  • Directly increasing the number and function of pancreatic beta cells in patients with either type 1 or type 2 diabetes using oral delivery of the drug (i.e. in vivo administration).


  • A small molecule therapeutic: No genetic manipulation required.
  • Approval of a small molecule for ex vivo expansion of donor pancreatic islets may have a faster path to regulatory approval than a traditional therapeutics.

Innovation Details

Detailed Description

Dr. Anil Bushan and colleagues at UCLA have identified a number of small molecules capable of expanding pancreatic islet numbers. These compounds act through inhibition of a common molecular pathway. Currently, no small molecule based methods are available to expand islets in vitro or in vivo.

File Number: 22432 

Disease: Metabolic/Endocrinology

Other Information:


Diabetes is associated with the loss and dysfunction of pancreatic beta cells. Beta cells are responsible for the production of insulin, a hormone which helps the body maintain healthy blood glucose levels. Among the estimated 25.8 million Americans with diabetes, approximately 95% have peripheral tissue insulin insensitivity (Type II) that results in elevated blood glucose, a major risk factor for vascular disease. The remaining patients are insulin dependent (Type 1), meaning they lack functional beta cells and require daily insulin shots to survive. In the United States alone, the societal cost for medical treatment and lost productivity due to diabetes is estimated to exceed $175 billion/year. Therefore, a significant financial incentive exists for improving the treatment of diabetes.

Pancreatic islet transplantation is a relatively recent treatment for controlling blood glucose that allows patients to produce their own insulin. In essence, replacement or expansion of a patient’s pancreatic islets would have major advances over the conventional therapies of daily insulin shots or insulin-sensitizing drugs. Foremost, transplanted islets like their native counterparts would be physiologically regulated, circumventing the need for frequent blood glucose testing and eliminating the adverse consequences of self-administered insulin or drug therapies. Still, there are several challenges associated with islet replacement. The principal road block is a lack of islets available for transplant. Human islets for this procedure are usually only available from cadavers, leading to limited availability. Thus, a novel mechanism that increases pancreatic islets expansion would dramatically improve availability of islet replacement therapies to patients.

IP Protection

Patent Number(s): WO2013119518
Copyright: ©2012-2014, The Regents of the University of California

License Online

This innovation currently is not available for online licensing. Please contact UCLA Office of Intellectual Property & Industry Sponsored Research at University of California System: University of California, Los Angeles - UCLA for more information.

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