Update on Islet Regeneration Research – SPIRIT

Team Pushing Forward With Positive Results From Phase 2 Trials

With positive results from last years Phase 2 trials and exciting new findings in its basic science laboratories, the Strelitz Diabetes Institutes’ SPIRIT team continues to set an innovative course for development of the INGAP gene toward a cure for diabetes.

Dr. Aaron I. Vinik’s research team is collaboratively working with international scientists and the biotech industry to swiftly develop regeneration research on all levels.

In June 2005, Dr. Robert Ratner of Medstar in Washington DC reported on the findings of the INGAP Phase 2 clinical trials. INGAP was found to be absolutely safe given in large amounts for a period of three months, and in terms of efficacy, changes were noted in both Type 1 and Type 2 diabetes.

In people with Type 2 diabetes, there was a decrease in A1cs by 0.6%. The Food and Drug Administration considers this sufficient for its approval of a product for lowering blood glucose in the treatment of diabetes. There was also an increase in postprandial (after eating) blood glucose.

Both people with Type 1 and Type 2 diabetes had an increase in C-peptide levels indicating that new islets were being formed.

Clinical trial participants received INGAP treatment for 90 days and were followed for 120 days. Recent re-analysis of the Phase 2 findings indicates that results persisted for 30 days after the trials were completed. This is very important because it shows that INGAP treatment changed the biology of diabetes in trial participants.

Dr. Ratner commented in his report, “In these Phase 2 trials for safety of INGAP, clear trends for efficacy have been found and suggest that with appropriate dosage, site of administration and duration of therapy, this approach holds clear promise to evolve into a new therapeutic approach to Type 1 and Type 2 diabetes.”

In light of these findings, SPIRIT (Stimulation of Pancreatic Islets in Type 1 and Type 2 Diabetes) scientists at the SDI continue their investigations to find out the optimal route for INGAP administration, the optimal dosage and way to administer the gene, how to protect newly formed islets (Type 1), and how to sensitize the body to newly created insulin (Type 2).

Last summer, the SPIRIT team achieved a significant milestone when they used transgenic animals to show that the production of pancreatic islet mass is dependent on INGAP expression in the acinar tissues of the pancreas.

As basic scientists continue their research, Kinexum Metabolics, Inc. has sublicensed INGAP Peptide from GMP Companies for further clinical trials.

Dr. G. Alexander Fleming, President and CEO of Kinexum, was formerly the Food and Drug Administration’s senior endocrinologist and clinical group leader for the evaluation and regulation of diabetes and metabolic therapies.

Dr. Fleming believes that INGAP Peptide “offers excellent prospects for a breakthrough therapy and the achievement of a major scientific milestone.”

For people struggling with diabetes “INGAP Peptide holds great promise for improving the health and quality of life of people with insulin-requiring diabetes – particularly those with Type 1. (It) could provide substantial or major value for all people with Type 1 and some insulin dependent people with Type 2 by restoring the ability to secrete insulin.”

He goes on to say, INGAP Peptide probably does not affect the underlying immunologic cause of Type 1, but it may by itself overcome the ongoing immune destruction of insulin secreting cells. As safe and effective immunotherapies become available, they are likely to work synergistically with INGAP Peptide to return perhaps close to normal insulin secretion.”

Clinical trials are to continue in the near future.

In anticipation of the issues that may be encountered, SDI scientists are working in animal models to investigate the underlying physiological mechanism by which INGAP exerts its biological effects. They are looking at the robustness and durability of the response to INGAP, reproducibility of the response, long-term safety in high doses, alternate routes and frequency of delivery and the possibility of delivering INGAP with a pump.