Rare form of diabetes needs different treatment

Treating patients with a rare genetic form of diabetes with therapies designed to treat other more common forms of the disease may do more harm than good, warn researches.

Patients with this rare form of diabetes called maturity-onset diabetes of the young (MODY-1) are at increased risk getting misdiagnosed as having Type-2 diabetes because the two share symptoms.

But the underlying problems in patients with the MODY-1 are very different from those in Type-2 diabetes, and treating these patients with drugs for Type-2 diabetes appears to lead to destruction of insulin-secreting beta cells that regulate blood sugar, the scientists found.

MODY-1 runs in families because of a change in a single gene which is passed on by affected parents to their children and it affects an estimated three to five percent of all patients with diabetes.

"People diagnosed with Type-2 diabetes are treated with oral medications that make insulin-secreting beta cells very active," said study first author Benjamin Moore from Massachusetts General Hospital in the US.

"But the MODY-1 pathway we've uncovered shows that stimulating those cells with those drugs can lead to beta cell death. That means these patients can become dependent on insulin injections much sooner," Moore noted.

The findings were published in The Journal of Biological Chemistry.

Instead, patients with MODY-1 might benefit from therapies that target a specific pathway that appears to be essential to the function of insulin-secreting cells, the researchers suggested.

The findings suggest that doctors treating patients with diabetes need to determine whether they have the MODY-1 form of the disorder before moving ahead with treatment.

"It's important to diagnose patients as accurately as possible and to attempt to target the correct pathway," Moore said.