G E T T H E F A C T S A B O U T T Y P E 1 D I A B E T E S
Type 1 diabetes (T1D) is an autoimmune disease in which a person’s pancreas stops producing insulin, a hormone that enables people to get energy from food. It occurs when the body’s immune system attacks and destroys the insulin-producing cells in the pancreas, called beta cells. While its causes are not yet entirely understood, scientists believe that both genetic factors and environmental triggers are involved. Its onset has nothing to do with diet or lifestyle. There is nothing you can do to prevent T1D, and—at present—nothing you can do to get rid of it.
Who T1D Affects
Type 1 diabetes strikes both children and adults at any age. It comes on suddenly, causes dependence on injected or pumped insulin for life, and carries the constant threat of devastating complications.
How T1D Is Managed
Living with T1D is a constant challenge. People with the disease must carefully balance insulin doses (either by injections multiple times a day or continuous infusion through a pump) with eating and other activities throughout the day and night. They must also measure their blood-glucose level by pricking their fingers for blood six or more times a day. Despite this constant attention, people with T1D still run the risk of dangerous high or low blood-glucose levels, both of which can be life threatening. People with T1D overcome these challenges on a daily basis.
Insulin Is Not a Cure
While insulin injections or infusion allow a person with T1D to stay alive, they do not cure the disease, nor do they necessarily prevent the possibility of the disease’s serious effects, which may include: kidney failure, blindness, nerve damage, heart attack, stroke, and pregnancy complications.
The Outlook for Treatments and a Cure
Although T1D is a serious and difficult disease, treatment options are improving all the time, and people with T1D can lead full and active lives. JDRF is driving research to progressively remove the impact of the disease from people’s lives until we ultimately achieve a world without T1D.
• 1.25M Americans are living with T1D including about 200,000 youth (less than 20 years old) and
over a million adults (20 years old and older)1, 2, 5
• 40,000 people are diagnosed each year in the U.S.1, 2
• 5 million people in the U.S. are expected to have T1D by 2050, including nearly
600,000 youth.2, 3
• Between 2001 and 2009 there was a 21% increase in the prevalence of T1D in people
under age 20.3
• $14B T1D-associated annual healthcare costs in the U.S.4
• Less than one-third of people with T1D in the U.S. are achieving target blood glucose
• T1D is associated with an estimated loss of life-expectancy of up to 13 years7
Warning signs of T1D may occur suddenly and can include:
• Extreme thirst
• Frequent urination
• Drowsiness or lethargy
• Increased appetite
• Sudden weight loss
• Sudden vision changes
• Sugar in the urine
• Fruity odor on the breath
• Heavy or labored breathing
• Stupor or unconsciousness
1. CDC National Diabetes Statistics Report, 2014
2. Impreatore, et al. 2012. Diab Care 35: 2515-2520
3. Dabelea, et al. 2014. JAMA 311: 1778-1786
4. Boyle, et al. 2010. Pop Health Metrics 8:29
5. JDRF Estimations
6. T1D exchange data
7. Jama 2015; 313(1):1-9)
T Y P E O N E S U P P O R T S T H E W O R K O F
T H E F A U S T M A N L A B A T M A S S A C H US E T T S G E N E R A L H O S P I T A L
A phase II clinical trial testing the ability of the generic vaccine bacillus Calmette-Guérin (BCG) to reverse advanced type 1 diabetes has received approval from the U.S. Food and Drug Administration (FDA). The approval of this trial, which has already begun enrolling qualified patients, was announced at the 75th Scientific Sessions of the American Diabetes Association (ADA) by Denise Faustman, MD, PhD, director of the Massachusetts General Hospital (MGH) Immunobiology Laboratory and principal investigator of the study.
The five-year trial will investigate whether repeat BCG vaccination can clinically improve type 1 diabetes in adults between 18 and 60 years of age who have small but still detectable levels of insulin secretion from the pancreas. Faustman’s research team was the first group to document reversal of advanced type 1 diabetes in mice and subsequently completed a successful phase I human clinical trial of BCG vaccination. She announced the FDA approval to launch the phase II trial during her ADA presentation, “Low Levels of C-Peptide Have Clinical Significance for Established Type 1 Diabetes.”
“We have learned a lot since the early studies in mice – not just about how BCG works but also about its potential therapeutic benefits, similar to what are being seen in trials against other autoimmune diseases,” says Faustman. “We are so grateful to all of the donors, large and small, who have made this trial possible – especially the Iacocca Foundation, which has believed in us and has been a supporter since our early days.
A generic drug with over 90 years of clinical use and safety data, BCG is currently approved by the FDA for vaccination against tuberculosis and for the treatment of bladder cancer. The vaccine is known to elevate levels of the immune modulator tumor necrosis factor (TNF), which Faustman’s team previously showed can temporarily eliminate in both humans and mice the abnormal white blood cells responsible for autoimmune type 1 diabetes. Increased TNF levels also stimulated production of protective regulatory T cells.
In the phase I clinical trial, which was published in the August 8, 2012, issue of PLOS Medicine, two injections of BCG spaced four weeks apart led to temporary elimination of diabetes-causing T cells and provided evidence of a small, transient return of insulin secretion. The phase II clinical study will include more frequent dosing over a longer time period to determine the potential of repeat BCG vaccination to ameliorate the autoimmune state and improve clinical parameters such as HbA1c, a marker of average blood sugar control.
In the new trial, which will be double blinded and conducted at MGH, 150 adults with long-term type 1 diabetes will be randomized to receive two injections four weeks apart of either BCG or placebo and then a single injection annually for the next four years. Patients will be closely monitored over the five-year trial period. The primary outcome measure will be improved results on the HbA1c blood test, which have been shown to prevent complications.
“In the phase I clinical trial we demonstrated a statistically significant response to BCG, but our goal in phase II is to create a lasting therapeutic response,” says Faustman, an associate professor of Medicine at Harvard Medical School. “We will be working again with people who have had type 1 diabetes for many years. This is not a prevention trial; instead, we are trying to create a regimen that will treat even advanced disease. In addition to our phase I trial, we took guidance from the BCG clinical trials that are underway globally for other autoimmune diseases such as multiple sclerosis.”
Lee Iacocca, founder of the Iacocca Foundation, says, “My family and I have been fortunate to be part of this research for many years. It is incredibly exciting to be talking about curing people, not mice. I made a promise to my late wife to find a cure for type 1 diabetes. Now my family and I look forward to the continued progress and are proud to support this effort to get closer to that goal.” The Iacocca Foundation provided major funding for the phase I trial and has taken a leadership role in funding the phase II trial.
Visit the Faustman Lab’s Facebook Page and stay up to date on their progress.
For more information on the clinical trials and the research study please click on faustman.org
On behalf of the 3 million people with type 1 diabetes, their family and friends we thank you for your commitment to our cause and our promise to find a cure!