Type 1 Diabetes Cure?  MGH Researchers Say New Drug Could be the Answer

Type I diabetes cure? MGH researchers say new drug could be the answer

Jun 7, 2015, 4:30pm EDT Updated Jun 7, 2015, 7:54pm EDT
Scientists at Massachusetts General Hospital are hoping to prove that type 1 diabetes can be reversed with the help of a generic drug.

The five-year long, phase II clinical trial, which received approval from the U.S. Food and Drug Administration, was announced Sunday during the 75th Scientific Sessions of the American Diabetes Association, one of the largest diabetes gathering in the country.

Scientists have already tested the bacillus Calmette-Guerin vaccine in mice, showing the drug’s ability to eliminate abnormal white blood cells responsible for the autoimmune disease.

Phase 1 trials in humans, results of which were published in August 2012 in the journal PLOS Medicine, showed that two injections spaced four weeks apart led to temporarily elimination of the T cells and a moderate return of insulin secretion.

“We have learned a lot since the early studies in mice – not just about how BCG works but also about its potential therapeutic benefits, similar to what are being seen in trials against other autoimmune diseases,” said Dr. Denise Faustman, director of the Massachusetts General Hospital Immunobiology Laboratory, an associate professor of Medicine at Harvard Medical School and principal investigator of the study, in a release.

With promising results in phase I trials, scientists will conduct phase II trials over a five-year period of time with more frequent dosing. The hope is to see if researchers can completely reverse the disease and see bettered clinical outcomes, such as average blood sugar control.

The double blind trial will include 150 adults with long-term type I diabetes. Patients will receive two injections four weeks apart and then a single injection annually over the next four years.

The vaccine is currently a generic drug with over 90 years of clinical use and proven safety, as it is used as a vaccination against tuberculosis and for the treatment of bladder cancer.

“Our goal in phase II is to create a lasting therapeutic response,” Faustman said. “We will be working again with people who have had type 1 diabetes for many years. This is not a prevention trial; instead, we are trying to create a regimen that will treat even advanced disease.”

While the FDA has approved the trial, additional funding is still needed. Despite help from long-time funding partner The Iacocca Foundation, researchers need another $6 million to complete the $25 million study.

But Faustman hopes to raise the remaining amount by the end of the year, as well as enroll the 150 patients needed for the study to begin.

Article Written by: Jessica Bartlett, Boston Business Journal