Testing of a new gene therapy for diabetes is under way in humans at three US centers, according to the Diabetes Institutes Foundation. It involves a gene named INGAP (Islet Neogenesis Associated Protein) that appears to help the pancreas regenerate new cells that make insulin.

Dr. Aaron Vinik, one of the original discoverers of the gene and head of the Strelitz Diabetes Institutes at Eastern Virginia Medical School, explained that this is a new approach to treating diabetes. Whereas current methods treat the metabolic consequences of diabetes (such as high blood sugar), this approach, called islet cell regeneration, stimulates growth of insulin-producing cells, thereby correcting the defect that causes the disease. It does this by stimulating precursor cells that are located in the pancreas, in effect turning them into beta cells to produce insulin.

A major advantage of this approach over islet cell transplantation, which is also under development as a potential cure for diabetes, is that it will be widely available. Islet cell transplants rely on pancreas donors, which severely restricts availability of the treatment. The peptide, however, can be synthesized, providing whatever quantities are needed to treat type 1 and type 2 diabetes.

In animal studies, INGAP was found to be safe and effective in producing new beta cells within the islets. The treatment is years away from routine use, however, if it does prove beneficial. The human trials now under way are testing for safety and dosing. If they are successful, then human trials of effectiveness can begin.

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Source:  The Diabetes Institutes Foundation. INGAP research reaches human trials. Dec. 16, 2001.