Updates
Fall 2015

from the Faustman Laboratory at Massachusetts General Hospital

A Note from Dr. Faustman
his has been an incredible

T

Many have been asking why weyear. In June, our Phase II trial

will be studying this specificprotocol was approved by the U.S.

population of people with type

Food and Drug Administration

1 diabetes. Why not patients(FDA), allowing us to fully launchwith 40 years of diabetes? Whythe Phase II trial testing BCG in

not children? One of the biggest

type 1 diabetes. The five-year trial

reasons is that these are thewill investigate whether repeatsame characteristics as theBCG vaccinations can improve

patients in our Phase I study. By

type 1 diabetes in patients withstudying patients with the same

longstanding disease.

characteristics, we will be able

Since that time, we’ve received
literally thousands of emails andphone calls from individuals
wanting to participate in the study.
What an amazing response! We arestill working to respond to each
person that has contacted us, andto submit combined data fromthe Phase I and Phase II trials tothe FDA to seek drug approval.
We believe that this approach
will make this program advance
more quickly towards the goal ofbringing BCG forward for type 1

letting us know how much thisprogram means to you. Everykind word and shared story has
been an inspiration to us here at
the lab.

diabetes. As we progress in theIf you have questions about thePhase II trial, we will work closely

Phase II trial or other aspects

with the FDA to discuss includingof the BCG research program,

In total, we anticipate enrollingother groups of patients inplease don’t hesitate to email us

approximately 150 people inadditional BCG studies.

at diabetestrial@partners.org.

Thank you to everyone who has

Thank you again!

we have already enrolled many
patients into the study.

the Phase II study. These will be
adults who still have detectablelevels of insulin secretion from thepancreas. Those who fulfill such
criteria usually have had type 1
diabetes for 25 years or less.

helped us launch this trial—
whether by spreading the word

Sincerely,

about this research, by offering
financial support or simply byDenise L. Faustman, MD, PhD

UpdateFall 2015

Update on the BCG andAutoimmunity Working Group

Protecting Patients from Type 1
Diabetes Complications

The BCG and Autoimmunity Working
Group is a global coalition of researchersinvestigating the use of BCG and similar
immune boosting strategies to prevent orreverse autoimmune diseases. As part of thisgroup, Dr. Faustman and other experts met
this October to discuss research and progressin type 1 diabetes, multiple sclerosis, Sjögren’s
syndrome and other forms of autoimmunity.

Our new paper, “Low levels of C-peptide
have clinical significance for established type 1
diabetes,” was published in the journal Diabetic
Medicine this summer. In it, we report on ourfindings that even low levels of insulin production
can be protective against the development ofdiabetic complications and hypoglycemia. Thisfinding is significant in two important ways.
First, it suggests that using new tests that canbetter measure low levels of C-peptide may help
doctors identify patients who are at higher riskof complications. It also suggests that helping
patients maintain even low levels of insulin
production—such as through interventions
like the BCG vaccine—can help patients avoid
complications and improve blood sugar control.
As we progress through the Phase II trial, we hope
to expand on these findings.

The group last met in 2013 and collaborated
on a scientific book, “The Value of BCG andTNF in Autoimmunity,” which was publishedin 2014. With the growing public and researchinterest in BCG and similar approaches,
this year’s meeting includes even moreinvestigators and experts who are active andinterested in moving this field forward.

Help Us Reach Our Funding Goal
We now have over $19 million raised for thePhase II clinical trial.
To find out how you can help us reach our$25.2 million goal, please see “How You Can Help”
on the last page of this newsletter.

www.faustmanlab.org

Conquering Diabetes One Mountainat a Time
Rick Noble has been supporting the Faustman
Lab’s research for over a decade. His inspiration ishis daughter, Kate, who was diagnosed with type 1
diabetes in 1992, when she was only 1 year old.

send the team off, and then hiked to Rainbow Falls,”
said Rick. “We brought prayer flags with quotes fromother KTS climbs, including one of my favorites fromT.S. Eliot—‘Only those who will risk going too far canpossibly find out how far one can go.’” This latest effort
raised more than $50,000 to help the Faustman Labconquer diabetes.

“Our doctors at the time said that we would behearing about a potential cure in about a decade,”said Rick. “To mark the end of that decade, welaunched Kiss the Sky (KTS) to Conquer Diabetes
in 2002, climbed Mount Whitney in California
and pledged to keep climbing until we conquereddiabetes. We joined the effort to help finance theFaustman Lab’s human clinical trials in 2004, andhave been part of it ever since.”
Since that first climb, KTS teams have climbed
mountains from Washington state to Bolivia.
They have also held numerous other fundraising
events—from dinners featuring mountaineers asguest speakers to a hike around the monuments inWashington, DC. This summer, the KTS team headed
back to California to climb 14,042-foot Mount
Langley.
“Our team included my daughter Sarah, friends
Lindsay, Mackenzie and Taylor Kingston, and theirmother Heather Pierce, all making their first attempt
at a fourteener (a mountain 14,000 feet or more inelevation). My wife Karen and daughter Kate helped

The team reached the summit under clear blue skies.

Spotlight on Some of Our DedicatedSupporters
There are so many people who care about thisresearch and who work hard to make sure wecontinue to move forward. (You may have readsome of their stories in our “Spotlight on aSupporter” series on Facebook.) We want to takethis opportunity to acknowledge a few groups thathave fundraising activities every year on behalf ofour Lab, each donating over $20,000:• Brendan’s Brigade - Annual bike ride put together
by the Buono family in honor of their son,Brendan• Drive to Cure Diabetes - Annual golf tournamentand auction produced by the Kearney family, inhonor of their daughter, Casey• Mr. & Mrs. Lijewski - In honor of their son, BrianWhite

Prayer flags with inspirational sayings brought by theKTS team.

Thank you to these individuals and all who havegiven their time and support to help further a curefor type 1 diabetes!

UpdateFall 2015Phase II BCG Clinical Trial Program
Led by Dr. Denise Faustman, the Phase II clinical trial isa randomized, double-blinded, study being conducted at
Massachusetts General Hospital. It will include approximately
150 people with type 1 diabetes, ages 18-65. The purpose of thisstudy is to see if repeat injections of bacillus Calmette-Guérin(BCG) are beneficial in longstanding type 1 diabetes. Published
Phase I data on repeat BCG vaccinations in long-term diabetics
show that BCG can specifically eliminate disease-causing “bad”
white blood cells (misprogrammed cells that attack and kill theinsulin-producing cells of the pancreas) while also temporarily
restoring a small degree of insulin secretion. In the Phase II
study, participants will receive more frequent injections to see ifthese desirable effects can be sustained.
Eligible volunteers will receive 2 injections of either BCG
or placebo spaced 4 weeks apart during the first year, then 1
injection per year for the next 4 years. Over the course of thestudy, the investigators will see whether BCG vaccination isassociated with improvements in HbA1c and look for any
beneficial changes in autoimmune status (such as continued
elimination of bad white blood cells).

How You Can Help
Please consider making a taxdeductible donation to sustain themomentum of this type 1 diabetes
research program. Every gift makes adifference.
1. To make a secure online donation,
visit www.faustmanlab.org andclick on “Support.”
2. You may make a gift by check(**payable to “Massachusetts
General Hospital”**) and mailyour check to:
Diabetes Clinical Trial
c/o Dr. Denise Faustman
Immunobiology LaboratoryMassachusetts General
Hospital-East

A Unique Type 1 Diabetes Research Program• Our goal is to reverse advanced diabetes, not just temporarily
halt new-onset disease or treat symptoms• The trials do not require lifelong immunosuppressive drugs orcell transplants• The trials are designed to kill only disease-causing cells, sparing
healthy cells

Building 149, 13th Street, CNY-3601
Charlestown, MA 02129

On the memo line of your check,please write: “Type 1 diabetes
research.”Thank you for joining us in the fightagainst diabetes!

• The trials employ BCG, a safe and inexpensive generic drug

Seeking more information about this type 1 diabetes research?
Visit www.faustmanlab.org.
Have questions about participating in the research?
Email: DiabetesTrial@partners.org.

Find us on Facebook:www.facebook.com/FaustmanLab
Follow us on Twitter:
www.twitter.com/FaustmanLab