Researchers from the City of Hope demonstrated that some patients with Type I diabetes can be cured, at least temporarily, with a stem cell transplant. The team’s work was published in Frontiers in Immunology. 

The trial enrolled 21 patients and found that autologous hematopoietic stem cell transplantation increased C-peptide levels and induced insulin independence in patients with Type I diabetes. The team concluded that transplanted stem cells are able to “balance” the immune system.

“This means we can cure Type I diabetes, be it with a risky therapy — although 1 that is also very successful in cancer, and 1 for which City of Hope is a world-renowned expert, with more than 13,000 patients having received similar treatment for blood cancers,” Bart Roep, director of The Wanek Family Project for Type I Diabetes, said in prepared remarks. “We now understand stem cell transplants can succeed in treating diabetes for some, but not in others, and we can predict either outcome before the therapy is administered by ‘reading’ the immune signature of the patient with a novel nanotechnology that I developed.”

Most patients were insulin free for 3.5 years, on average, following transplantation and C-peptide levels remained higher than initial values for at least 4 years.

“1 patient is free of insulin for more than 8 years now, without any major side effects,” Roep said. “However, we discovered the immune signature predicting these outcomes — either favorable or not — which is the first step toward personalized medicine in Type I diabetes. We have a foot in the door.”

The authors pointed out that stem cell transplants, which involve intense immunosuppression prior to transplantation, are risky procedures and are unlikely to become the 1st line of defense for patients Type I diabetes. But the team argued that their findings could enable the development of new strategies for immunotherapies and Type I diabetes.

“This study paves the way for personalized therapy in Type I diabetes,” Roep said. “Understanding why it sometimes fails will allow us to design new treatment strategies for those less fortunate patients. Also, it is the 1st definitive proof that T1D can be cured.”