In the quest for a cure for Type 1 diabetes, McGowan Institute for Regenerative Medicine
faculty member Massimo Trucco, MD (pictured), Chief of the Division of Immunogenetics at Children’s Hospital of Pittsburgh of the University of Pittsburgh Medical Center, and his colleagues are currently conducting a randomized clinical trial to evaluate the safety of a new diabetes-suppressive cell vaccine. Dr. Tucco’s leukapheresis-based dendritic cell approach, approved by the U.S. Food and Drug Administration (FDA), is being tested in humans at Children’s Hospital.
If the leukapheresis-based approach continues to show exceptional safety, the researchers hope to launch a national clinical trial that will assess the effectiveness of the dendritic cells in pediatric patients to prevent diabetes or reverse the disease right after it is clinically confirmed. The study, “Autologous Dendritic Cell Therapy for Type 1 Diabetes Suppression: A Safety Study,” is currently open for enrollment.
Scientists increasingly hope to control Type 1 diabetes by curbing the rogue immune cells that cause it, before patients become completely dependent on daily insulin injections to survive. "Treating at onset in children is the best chance we have," said Dr. Trucco, whose novel vaccine is a possible immune therapy to control Type 1 diabetes. The research suggests if the therapy preserves enough precious insulin-producing cells before irreversible damage is done, maybe patients would need far less insulin.
Leukapheresis is a process that allows for the collection of dendritic cell precursors from the patients in the study, which takes 2 to 4 hours. After the precursors are collected, they are treated in the lab with specific growth factors that turn them into dendritic cells. The growth factors are also combined with short DNA sequences that specifically block the expression of molecules that are found at the surface of dendritic cells known as CD40, CD80, and CD86. Once these reprogrammed dendritic cells are tested in the lab, they are injected back into the patient. They then orchestrate an anti-diabetic effect by suppressing the activity of T-cells which are responsible for the impairment and destruction of the pancreatic insulin-producing cells.
Type 1 diabetes is regarded as an autoimmune disease because a person’s immune system’s T cells attack and destroy the beta cells in the pancreas that produce insulin. Symptoms of Type 1 diabetes usually develop over a short period of time and include increased thirst, frequent urination, constant hunger, weight loss, blurred vision, and extreme fatigue. People with Type 1 diabetes require daily injections of insulin to survive. Type 1 diabetes also is known as insulin-dependent diabetes mellitus or juvenile-onset diabetes. The National Institutes of Health (NIH) reports that more than 1 million children and teenagers (age 19 and younger) have Type 1 diabetes. According to the NIH, 5 percent to 10 percent of diagnosed diabetes cases in the United States are Type 1 diabetes.
Read a 4-part Spotlight Series on Dr. Trucco here…
McGowan Institute for Regenerative Medicine In the News, Part 1 of 4
McGowan Institute for Regenerative Medicine In the News, Part 2 of 4
McGowan Institute for Regenerative Medicine In the News, Part 3 of 4
McGowan Institute for Regenerative Medicine In the News, Part 4 of 4
Illustration: McGowan Institute for Regenerative Medicine.
Bio: Dr. Massimo Trucco