"This is the first personalized treatment for type 1 diabetes prevention," said Aaron Michels, MD, a researcher at the Barbara Davis Center for Childhood Diabetes and associate professor of medicine at CU Anschutz. "We made this discovery using a supercomputer, on the lab bench, in mice and in humans."
The drug, methyldopa, has been used for over 50 years to treat high blood pressure in pregnant women and children. It is on the World Health Organization's list of essential drugs.
But like many drugs used for one condition, Michels and his colleagues found it useful for something totally unrelated.
Some 60 percent of people at risk of getting type 1 diabetes possess the DQ8 molecule which significantly increases the chance of getting the disease. The researchers believed that if they could block specifically the DQ8 molecule they could also block the onset of the disease.
"All drugs have off-target effects. If you take too much acetaminophen you can hurt your liver," Michels said. "We took every FDA approved small molecule drug and analyzed HLA-DQ8 binding through a supercomputer. We searched a thousand orientations for each drug to identify those that would fit within the DQ8 molecule binding groove."