Neonatal Diabetes Patients Weaned Off Insulin : Patients with certain genetic mutations are being successfully treated with oral sulfonylureas instead.

“These kids [with neonatal diabetes] are thin. All the children that I've seen and that Dr. Hattersley's seen have normal or below-normal [body mass indexes]. They're highly sensitive to insulin, and the insulin secretion we're looking for is modest,” he said.

In addition to Lilly Jaffe, the little girl he treated last summer, Dr. Philipson and his colleague Dr. Graeme Bell have treated six other children from across the country. He estimates about 20 children have been treated in the United States. “And there are at least several hundred others … who could be reached,” he said.

Tests for the mutations are commercially available through at least one company, Athena Diagnostics, he said.

Dr. Philipson has used glyburide, the sulfonylurea used in most of the patients in Dr. Hattersley's study, in an inpatient protocol that allows rapid switching from insulin. His protocol resembled that followed by investigators in Dr. Hattersley's study, which entailed starting glyburide at a dosage of 0.1 mg/kg twice daily and increasing the dosage by 0.2 mg/kg per day. In an outpatient protocol that some of the investigators chose, glyburide was introduced at 0.1 mg/kg per day and increased by that amount once a week.

In Dr. Hattersley's study, the median dosage of glyburide that was required for insulin independence was 0.45 mg/kg per day.

Lilly Jaffe is now receiving much smaller doses of glyburide twice a day, and “remarkably, her morning blood sugars are still quite reasonable,” said Dr. Philipson.

In Washington, Dr. Cogen said she will be “less worried” about using high doses of glyburide in her 18-year-old patient than she would be in a younger child, though she plans to get the word out to all of her patients with neonatal diabetes. “To be able to take a patient off of insulin is so exciting—it's everybody's dream.”