Updates
Fall 2018from the Faustman Laboratory at Massachusetts General Hospital

A Note from Dr. Faustman
This was a major year for
the Immunobiology Lab
here at MGH and our quest
to reverse type 1 diabetes.
In June, we published dataon the patients in the BCG
program followed for at least
five years. The results showed
restoration of near-normal
blood sugar levels three years
after receiving two dosesof BCG four weeks apart—
improvements that persisted.
All participants in the study
were adults with longstanding
type 1 diabetes. In addition
to the clinical success, webelieve we have uncovered anadditional effect BCG has onlong term diabetic subjects, anovel metabolic mechanismthat increases cellularconsumption of glucose. The
news spread quickly aroundthe world through dozens
of media stories including
coverage by CNN, Time, The

Boston Globe, The Telegraphand The Washington Post.
Our work is not done. The
Phase II clinical trial is still
underway. All 150 patients
have received at least two
doses of BCG or placebo.
We are also actively planninga pediatric trial, which,depending on funding, wehope to start as early as nextyear. BCG is a generic vaccine,which means the profit willbe the people it helps.

This is great for patients, but
it also means we need to fundthe research with donations
from individuals and private
philanthropies.
We hope you will help us
move this important work
forward. Please contact us if
you have any questions at
diabetestrial@partners.org.
Sincerely,

Denise L. Faustman, MD, PhD

UpdateFall 2018

Long-term Follow Up of Phase IIOn June 21st, the Faustman Lab publishedin Nature Vaccine results of participants in thebacillus Calmette-Guérin (BCG) clinical trial
program who had been followed for at least
five years.
The patients had received two administrationsof the BCG vaccine four weeks apart. All wereadults with longstanding type 1 diabetes. All of
the patients showed an improvement in HbA1c
to near normal levels – improvement thatpersisted for the following five years.

What is the significance of HbA1c?
HbA1c refers to glycated hemoglobin andis a measure of overall average blood sugar
levels over a period of months. Higher HbA1c
is directly correlated with greater risks ofdeveloping diabetes-related complications such as
blindness, heart attacks, strokes and renal failure.
A significant lowering in HbA1c is a primary
endpoint for diabetes clinical trials.

HbA1c dropped in the treated participants (red line).

Nature Vaccines

Regular monitoring of clinical trial participants
found that HbA1c levels of those receiving
BCG had dropped by more than 10 percent at
three years after treatment and by more than
18 percent at four years. That reduction wasmaintained over the next four years, with treatedparticipants having an average HbA1c of 6.65
(close to the 6.5 considered the threshold fordiabetes diagnosis) and with no reports of severe
hypoglycemia. Participants in the placebo groupand in a comparison group of patients receiving
no treatment experienced consistent HbA1celevations over the same eight-year time period.
In investigating how BCG administration
produces its beneficial effects, the researchteam identified a mechanism never previously
seen in humans in response to treatment witha drug – a shifting of the process of glucose
metabolism from oxidative phosphorylation,
the most common pathway by which cells
convert glucose into energy, to aerobic
glycolysis, a process that involves significantly
greater glucose consumption by cells. Theresearchers also found that BCG could reduce
blood sugar elevations in mice that were causedby means other than autoimmune attack,raising the possibility that BCG vaccines could
also be beneficial against type 2 diabetes.

www.faustmanlab.org

Updates on How BCG Changes
the Immune System
Over the course of the last year, Dr. Faustman presented updates on the BCG clinical trialprogram at three international diabetes conferences and was invited to give a key note
speech at a special conference organized in Paris at the Pasteur Institute in honor of the110th anniversary of the discovery of the BCG vaccine.

Trial Recruitment
The Phase II trial is fully enrolled, but we are still looking for patients for future study groups.
We are actively planning and designing new cohort and study groups including a pediatrictrial and an expanded trial in adults that we hope to start next year. If you are interested pleasecontact the lab. We recommend you email the lab at DiabetesTrial@partners.org to see if
you might be eligible for a type 1 diabetes trial or FibromyalgiaStudy@partners.org if you areinterested in the fibromyalgia trials.

New Edition of Book
In September, Dr. Faustman, in
collaboration with BCG researchers
around the globe, published a newedition of The Value of BCG and
TNF in Autoimmunity. The bookis based on the third International
BCG conference held in October
2017. The new book contains
several new chapters including
updates on the large pediatricprevention trials taking place inDenmark and Australia.

“The clinical effects andthe proposed mechanism
demonstrated are exciting andadd to the emerging consensus
that the BCG vaccine can have alasting and valuable impact on
the immune system. The MGH
trials and other, larger prevention
and intervention trials underway
around the globe may lead toa major shift in the prevention
and treatment of infections andautoimmunity.”
Mihai G. Netea., PhD, professor
in the Department of Internal
Medicine at Radboud University
Medical Center in the Netherlands

UpdateFall 2018

FUNDRAISING EVENTSDrive To Cure DiabetesTurns Ten

How You Can Help
Please consider making atax-deductible donation to thistype 1 diabetes research program.
Every gift makes a difference.

Kathy Kearney and herteam put together anotherincredible golf tournament inNew York.

Old Friends in Connecticut
On September 12th, Kim
Morse hosted a meeting withdonors organized by Sue
Root. Sue is one of our longest
standing advocates going backto a bike ride she organizedwith Jackie Fusco in 2004.
Stephen Schwartz opened
the evening.

Strike Out Diabetes

1. To make a secure online donation,
visit www.faustmanlab.org andclick on “Donate.”
2. You may make a gift by check(payable to “Massachusetts
General Hospital”) and mail it to:
Diabetes Clinical Trial
c/o Dr. Denise Faustman
Immunobiology Laboratory
MGH - East
Building 149, 13th Street, CNY-3601
Charlestown, MA 02129

On the memo line, please write:“Faustman T1D research.”

Amy Sampen and herhusband, MLB veteran Bill
Sampen, launched a neworganization dedicated tostriking out type 1 diabetes.
Proceeds are going tosupport the lab.

Thank you for joining us in thefight against diabetes!

The NFL Intercepts
Diabetes
Mr. Sid held a specialevening for the Faustman Lab
hosted by former New England
Patriots Scott Zolak, Jerod
Mayo and Rob Ninkovich tokick off Mr. Sid’s season long
campaign to raise funds fortype 1 diabetes research.

For more information, visit
www.faustmanlab.org or email
DiabetesTrial@partners.org.
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