Stem Cells Help People with Diabetes become Insulin Free
Author: JAMA and Archives Journals
Published: 2009/04/14
Category Topic: Regenerative Medicine - Academic Publications
Page Content: Synopsis - Introduction - Main
Synopsis: People with type 1 diabetes who underwent a certain type of stem cell transplantation became insulin free.
Introduction
The majority of patients with type 1 diabetes who underwent a certain type of stem cell transplantation became insulin free, several for more than three years, with good glycemic control, and also increased C-peptide levels, an indirect measure of beta-cell function.Main Content
The majority of patients with type 1 diabetes who underwent a certain type of stem cell transplantation became insulin free, several for more than three years, with good glycemic control, and also increased C-peptide levels, an indirect measure of beta-cell function, according to a study in the April 15 issue of JAMA, a theme issue on diabetes.
Richard K. Burt, M.D., of the Northwestern University Feinberg School of Medicine, Chicago, presented the findings of the study at a JAMA media briefing at the National Press Club in Washington, D.C.
Clinical evidence indicates that there is an inverse association between beta-cell (a type of cell in the pancreas that secretes insulin) preservation and function and chronic complications of type 1 diabetes mellitus (DM), and the higher the C-peptide levels (a byproduct of insulin production, made up of amino acids), the lower the incidence of some types of complications of type 1 DM. A previous study found that autologous non-myeloablative hematopoietic stem cell transplantation (HSCT) in 15 patients with newly diagnosed type 1 DM resulted in the majority of patients becoming insulin free during the follow-up, which averaged about 19 months. "However, it was suggested that subsequent insulin independence was a prolonged honeymoon period due to dietary and exercise changes associated with close post-transplant medical observation," the authors write, and it was not known if this change was because of an improvement in beta-cell preservation.
HSCT, which uses a patient's own blood stem cells, involves the removal and treatment of the stem cells, and their return to the patient by intravenous injection.
Dr. Burt and colleagues conducted a study to determine if post-transplant insulin independence was due to improved beta-cell function by monitoring the C-peptide levels of 23 patients who underwent stem cell transplantation. The patients, with type 1 DM, were ages 13-31 years.
Of the 23 patients, 20 experienced time free from insulin (12 continuously and 8 transiently). Patients remained continuously insulin free for an average time of 31 months (range, 14-52 months). One patient had more than 4 years with no exogenous (produced outside the body) insulin use, 4 patients for at least 3 years, 3 patients for at least 2 years, and 4 patients for at least 1 year. Eight patients relapsed and resumed insulin use at low doses. The majority of patients achieved good glycemic control.
In the continuously insulin-free group, average area under the curve (AUC; a type of measurement) of C-peptide levels before transplantation (225.0 ng/mL per 2 hours) showed a significant increase at 24 months after transplantation (785.4 ng/mL per 2 hours) and at 36 months after transplantation (728.1 ng/mL per 2 hours). In the transient insulin-independent group, average AUC of C-peptide levels also increased from 148.9 ng/mL per 2 hours pre-transplantation to 546.8 ng/mL per 2 hours at 36 months, which was sustained at 48 months. In this group, 2 patients regained insulin independence after treatment with the anti-hyperglycemic drug sitagliptin, which was associated with an increase in C-peptide levels.
Two patients developed pneumonia in the hospital, 3 patients developed late endocrine dysfunction, and 9 patients developed oligospermia (sperm deficiency). There were no deaths.
"In conclusion, autologous non-myeloablative HSCT was able to induce prolonged and significant increases of C-peptide levels associated with absence of or reduction of daily insulin doses in a small group of patients with type 1 DM," the researchers write. "At the present time, autologous non-myeloablative HSCT remains the only treatment capable of reversing type 1 DM in humans. Randomized controlled trials and further biological studies are necessary to confirm the role of this treatment in changing the natural history of type 1 DM."