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.
- 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.
File Number: 22432
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.
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