Local diabetes study earns spotlight
Safety trials of a medical technique that could reverse Type 1 diabetes might be completed by year's end at Children's Hospital of Pittsburgh, the lead researcher told diabetes experts gathered Wednesday in Miami.
"We've had very positive results so far," Dr. Massimo Trucco, director of immunogenetics at Children's Hospital, said about the first phase of the trials that are testing the technique's safety. "We have to first prove that this is safe. When we pass that goal, the second step will be to prove its efficacy."
The trials, which began in July, involve injecting patients with their own immune cells -- after they have been genetically altered so they can block the destruction of insulin-producing cells in the pancreas.
Such a technique has proven effective in mice. Trucco and his colleagues are eager to test the technique in children -- a population at high risk of developing Type 1 diabetes.
The trials do not involve children. Instead, researchers are using the technique on 18-year-olds who are able to give consent to the experiments. Five people have been enrolled so far, and four of them have started to receive the injections. Trucco's goal is to enroll 10 more people in the trial.
None of the participants has shown any sign of side effects, such as rash or fever, said Trucco, who also is a professor of pediatrics at the University of Pittsburgh.
If successful, the technique could be a weapon against Type 1 diabetes -- a disease that robs people of their ability to produce insulin and forces them to rely on daily doses of insulin to survive.
The research at Children's was one of several approaches discussed over the past four days at a meeting of the Type 1 Diabetes TrialNet Study Group -- which includes researchers from more than 150 medical centers in the United States, Canada, Europe and Australia.
Trucco said more extensive studies of the technique's efficacy could start in the spring.
The injections are made with so-called dendritic cells isolated from the patient's own blood. Study participants receive three injections a week for six weeks. Half of the participants are in a control group, which means they are getting injections of cells that have not been altered.