Potential Type 1 Diabetes Treatment Meets Hurdles
Daily injections of insulin are the life-saving treatment for type 1 diabetes since the body cannot produce the hormone itself. But what if researchers could come up with a treatment that allowed the body to use its own genetically engineered cells to stop the process that kills the cells that make insulin?
A number of scientists are exploring this idea, and at least one team has been working on a method based on adoptive cellular therapy which shows promise. This approach involves modifying how the body’s immune system affects specific cells.
The team in question includes Massimo Trucco, MD, a research pediatrician at Children’s Hospital of the University of Pittsburgh School of Medicine, and Nick Giannoukakis, MD, associate professor of pathology and immunology at the same institution. Both Trucco and Giannoukakis were part of a team that published a report recently in Diabetes in which they explained why the use of modified cells could be advantageous in fighting type 1 diabetes.
More specifically, the scientists are exploring the use of a patient’s genetically engineered dendritic cells to help stop the signature of type 1 diabetes: the autoimmune process that destroys the beta cells that produce insulin. To accomplish this, patients would need to undergo a process called leukapheresis, in which their blood would be collected from one arm, processed through a machine to gather dendritic cells, and then returned to the patient in the other arm.
Leukapheresis is a procedure that is used to obtain blood cells for research or for returning them later to patients for transplant. It also is used as part of an immunotherapy treatment for prostate cancer called sipuleucel-T.
Dendritic cells are immune system residents also known as antigen-presenting cells. That means they process antigen (a substance that is foreign to the body) material and present it to other immune system workers (e.g., B and T cells) and facilitate or help with the immune response. Dendritic cells have been chosen for this immunotherapy process because they are easier to gather from the blood and genetically engineer than are other cells involved in the disease.
According to a recent article, Trucco is in the process of trying to get a grant to pursue a Phase II clinical trial to test this immunotherapy approach in young people who have type 1 diabetes. The $7 to $10 million needed for the research will be difficult to secure given the severe federal budget cuts for the National Institutes of Health.
If Trucco and his team do get the grant monies, he noted in the article that “It would be the first trial in which live cells are used to treat a disease that is not cancer, and we would be dealing with children.”
It is critical to perform this therapy within a few months of diagnosing someone with type 1 diabetes so the researchers can save the most beta cells. Symptoms of type 1 diabetes appear when about 80 percent of beta cells have been destroyed, and the remaining cells continue to die until all or nearly all of the are gone.
Because individuals who have 20 percent of their beta cells can maintain normal blood glucose levels if they follow dietary restrictions, the researchers need to initiate the immunotherapy as quickly as possible.
Right now, Trucco is preparing the funding proposal for the human clinical trial necessary to test this immunotherapy approach. If the monies are granted (and the decision should come in September 2014), then he and his team can move ahead with this potential type 1 diabetes treatment breakthrough.
Creusot RJ et al. It’s time to bring dendritic cell therapy to type 1 diabetes. Diabetes 2014 Jan; 63:20-30
Type 1 diabetes: a treatment’s in sight but where’s the funding? Pittsburgh Post Gazette