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Study Backs Pill Use for Neonatal Diabetes

The small number of diabetics who were diagnosed early in infancy may be spared a lifetime of insulin shots, according to new research. Investigators two years ago pinpointed a genetic cause for about half of all cases of neonatal diabetes, uncovering a flaw that prevents the body from producing insulin. The revelation led researchers to test pills taken for decades by older diabetics. The results show those pills can circumvent the mutation and allow patients to start producing insulin for the first time.

About 1,500 to 2,000 Americans with neonatal diabetes rely on daily insulin injections to survive, said lead researcher Ewan Pearson, a lecturer at the University of Dundee in Scotland. The study found that 44 of 49 patients with the genetic mutation were able to safely switch to pills and get better control of the disease.

“Anybody who was diagnosed before six months of age should go and have a genetic test for the mutation," Pearson said in a telephone interview. “If they have it, there is a 9 in 10 chance they could come off insulin."

The study, along with the report of another genetic mutation that may explain as many as 12 percent of neonatal diabetes cases, appears in today’s New England Journal of Medicine.

While almost 21 million Americans have diabetes, more than 90 percent have type 2, which tends to develop during adulthood. They often take drugs called sulfonylureas to boost insulin production.

Neonatal diabetes is very rare, occurring in just 1 in 100,000 births. Patients traditionally are treated with daily insulin shots like those with type 1 diabetes, which is generally diagnosed in children and accounts for 5 to 10 percent of all diabetes cases.
The researchers used sulfonylureas in the study. The treatment restored insulin secretion and led to greater control of the diabetes, wrote Mark A. Sperling, an endocrinologist at Children’s Hospital of Pittsburgh, in an editorial accompanying the study.

Pearson ER et al. "Switching from Insulin to Oral Sulfonylureas in Patients with Diabetes Due to Kir6.2 Mutations." N Engl J Med 2006;355:467-77.

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