Denise Faustman, MD, PHd
The Faustman Lab at work

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T1D Research Overview

The Faustman Lab’s type 1 diabetes (T1D) research is currently focused on testing Bacillus Calmette-Guérin (BCG), an inexpensive generic drug, as a treatment for advanced type 1 diabetes. Other type 1 diabetes studies and basic research projects are also being conducted in support of this specific program and the Lab’s overall goal of type 1 diabetes reversal.

BCG Human Clinical Trial Program
This human clinical trial program is testing BCG, an inexpensive vaccine, as a treatment for advanced type 1 diabetes.

In the double-blind, placebo-controlled Phase I study, BCG was administered to adults who had been living with type 1 diabetes for an average of 15 years. This treatment not only helped eliminate the defective T cells that mistakenly attack and destroy the insulin-producing cells of the pancreas, it also temporarily restored the ability of the pancreas to produce small amounts of insulin. The results of the Phase I study were published in 2012. Learn more about the BCG Human Clinical Trial Program.

A Phase II study is currently underway. The goal of the Phase II trial will be to identify the dose and schedule of BCG vaccination that will put advanced type 1 diabetes into remission and prevent long-term diabetic complications. To learn more about participating in this research, please contact diabetestrial@faustmanlab.org

Early Type 1 Diabetes Research
The BCG Human Clinical Trial Program is the culmination of over two decades of research at the Faustman Lab. In their early research, Dr. Faustman and her team identified a specific defect in the white blood cells that cause type 1 diabetes and other autoimmune diseases in the mouse and human. This cellular defect makes the disease-causing cells sensitive to death in the presence of higher-than-normal levels of tumor necrosis factor (TNF), an immune system protein made naturally by the body.

In their early research, Dr. Faustman and colleagues used a brief, non-toxic treatment—part of which has been translated into the current human clinical trials—to induce TNF in mice with advanced diabetes. This permanently eliminated their disease by halting the autoimmune attack on their pancreases, restoring normal blood sugars and precipitating the regeneration of their insulin-producing cells. The results of these animal studies were published in 2001 in the Journal of Clinical Investigation and in 2003 in Science.

The 2003 Science paper also identified the spleen as a potential new source of adult stem cells that could form new islets in formerly diabetic animals and speed disease reversal and regeneration. However, disease reversal was also seen in animals that did not have live spleen cells introduced. In the BCG Human Clinical Trial Program, there is no intent of using spleen cell transplants for human patients.

Other Recent Findings
For over 20 years, scientists have thought that the ability of the pancreas to produce insulin declines very abruptly (within 1-2 years of diagnosis) in people who develop type 1 diabetes. These findings were based on standard C-peptide tests (an indicator that the pancreas is secreting insulin), and have meant that most type 1 diabetes trials have focused on newly diagnosed patients, who were thought to have the best chances of recovering insulin secretion. Recent research from the Faustman Lab, published in Diabetes Care in 2012, shows that the decline of insulin-production occurs much more slowly than scientists have traditionally thought.

Using an ultrasensitive blood test, the Faustman Lab found that C-peptide production can persist for decades after type 1 diabetes onset and remain responsive to blood sugar levels. Although C-peptide levels were lower among those who had lived longer with the disease, they did not abruptly decline as previously thought, but rather decreased gradually over time. Moreover, C-peptide was detected in 10% of patients in the Faustman Lab’s study who had been living with the disease for 31-40 years. These findings suggest that there may be a longer period in which we may be able to intervene in type 1 diabetes, even in people diagnosed decades ago.

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