August 20, 2012

Clinical Paper on Phase I Trial with BCG Is Now Published

We are excited to share today's long-awaited news - the full published results of the Phase I clinical trial using BCG in advanced type 1 diabetes. You can find our paper, "Proof-of-Concept, Randomized, Controlled Clinical Trial of Bacillus-Calmette-Guerin for Treatment of Long-Term Type 1 Diabetes" on our website http://www.faustmanlab.org.

The findings from this double-blinded clinical trial, even at this early testing stage in humans, show that the generic BCG vaccine can raise the levels of an immune system modulator (called tumor necrosis factor, or TNF) to kill the disease-causing T cells that attack the pancreas and help to temporarily restore insulin secretion in patients with long-term type 1 diabetes.

We are trying to create a regimen that will reverse type 1 diabetes, even for people in the most advanced stages of the disease. With the Phase I clinical trial data, we believe we have validated in humans the treatment pathway we originally reported in mice. We expected to verify the safety of the BCG vaccine in type 1 diabetes in this Phase I study, as well as now show early signs of BCG efficacy. Our findings show that a simple, inexpensive vaccine can modify the autoimmunity underlying type 1 diabetes by selectively killing only the disease-causing T cells, leading to at least some restoration of pancreatic beta-cell insulin function. In Phase II human clinical testing of BCG, we hope that more frequent or higher BCG dosing will help the body eliminate the disease-causing T cells for a longer time, which we hope will restore insulin production to a greater degree and for a more sustained period of time than in the Phase I study.

Dr. Paul Burn, PhD, chair and director emeritus of the Sanford Project and professor of Pediatrics at the Sanford School of Medicine at the University of South Dakota, had this to say about our results: "Dr. Faustman and her team's clinical research data indicate that modifying the autoimmunity underlying type 1 diabetes allows for a safe and temporary restoration of insulin-secreting beta-cell function in patients with established type 1 diabetes. Restoring beta-cell function is a promising first step towards a cure. During my tenure in industry, at Sanford Health and at the Juvenile Diabetes Research Foundation, I have seen how hard it is to get a project from mice into humans, and these are very impressive results."

These human clinical trials are unique in testing an immune intervention in advanced type 1 diabetes (not just new-onset disease), using an inexpensive and safe generic vaccine, understanding the mechanism by which the treatment works to selectively eliminate the unwanted, disease-causing T cells, and being able to successfully track efficacy with careful blood monitoring. These factors will contribute to speeding our progress as we move forward in clinical testing.  

Currently, $11 million of the total $25.2 million needed has been raised for a Phase II study. Please consider making a donation to support this groundbreaking work by visiting www.faustmanlab.org/support/support.html.

Denise Faustman, MD, Ph.D.