Brazil's risky diabetes experiment has patients living without insulin

CHICAGO - Could their own stem cells allow people with Type 1 diabetes to live without daily insulin shots? A controversial but promising experiment in Brazil suggests the answer someday might be yes.

In a medical first, 15 young people with newly diagnosed diabetes had stem-cell transplants from their own blood. Thirteen were able to give up insulin for periods ranging from six months to three years. They are being followed to see if the results are long-lasting.

While the procedure is risky and potentially life-threatening, none of the patients died or suffered lasting side effects.

"It's the first time in the history of Type 1 diabetes where people have gone with no treatment whatsoever . . . no medications at all, with normal blood sugars," said study coauthor Richard Burt, of Northwestern University Medical School in Chicago.

Larger, more rigorous studies are needed to determine whether stem-cell transplants could become standard treatment for people with the disease, once called juvenile diabetes. It is less common than Type 2 diabetes, which is associated with obesity.

The hazards of stem-cell transplantation also raise questions about whether the study should have included children. One patient was 14.

Lainie Ross, a medical ethicist at the University of Chicago, said the researchers should have studied adults first before exposing young teens to the potential harms of stem-cell transplant, which include infertility and late-onset cancers.

In addition, Ross said that the study should have had a comparison group to make sure the treatment was indeed better than standard diabetes care.

Burt, who wrote the study protocol, said the research was done in Brazil because U.S. doctors were not interested in the approach. The study was approved by ethics committees in Brazil, he said, adding that he personally believed it was appropriate to do the research in children as well as in adults, as long as the Brazilian ethics panels approved.

Burt and others called the results an important step forward.

"It's the threshold of a very promising time for the field," said Dr. Jay Skyler of the Diabetes Research Institute at the University of Miami.

Skyler wrote an editorial in the Journal of the American Medical Association, which published the study, saying the results were likely to stimulate research that might lead to methods of preventing or reversing Type 1 diabetes.

The patients involved were ages 14 to 31 and newly diagnosed with Type 1 diabetes. An estimated 12 million to 24 million people worldwide - including 1 million to 2 million in the United States - have this form of diabetes, which is typically diagnosed in children or young adults. An autoimmune disease, it occurs when the body attacks insulin-producing cells in the pancreas.

Insulin is needed to regulate blood-sugar levels, which when too high can lead to heart disease, blindness, nerve problems and kidney damage.

Burt said the stem-cell transplant was designed to stop the body's immune attack on the pancreas.

The 15 diabetics were treated at a bone-marrow center at the University of Sao Paulo. All were newly diagnosed, before their insulin-producing cells had been destroyed.

That timing is key, Burt said. "If you wait too long," he said, "you've exceeded the body's ability to repair itself."

See more from the Juvenile Diabetes Foundation via http://go.philly.com/diabetes2 EndText