A drug to stop diabetes’ onset?

From Anaheim, Calif., at a meeting of the American Chemical Society

In an effort to stem the increasing prevalence of type 1 diabetes, researchers are developing a drug that could protect susceptible individuals from the disease.

People with type 1 diabetes need frequent insulin injections to control their blood sugar because their immune system has destroyed the insulin-producing cells in their pancreas. One of the proteins in the cascade of reactions responsible for killing pancreatic cells is called macrophage migration inhibitory factor (MIF).

Medicinal chemist Yousef Al-Abed of the North Shore–Long Island Jewish Research Institute in Manhasset, N.Y., led recent effort in which he and his colleagues identified a compound, called ISO-1, that blocks the activity of MIF. When the researchers treated mice genetically altered to develop diabetes, a 10-day course of the drug prevented the disease’s onset in 90 percent of the mice. What’s more, the drug’s protective effects lasted up to 2 months after the treatment ended.

Although several research groups are developing drugs for prevention, most have concentrated on antibody-based vaccines. Any successful antibody vaccine will require trained health care workers to administer multiple injections, Al-Abed says. Because ISO-1 is a small molecule, it could be taken in a pill and have long-lasting effects.

Candidates for the treatment would include people with certain protein or gene markers in their blood that predict increased risk of diabetes, says Al-Abed, but he cautions that it could be 10 years before ISO-1 reaches patients.