Updates
Fall 2017from the Faustman Laboratory at Massachusetts General Hospital

A Note from Dr. Faustman
Another year has passed
too quickly, but we are happy
to share a number of major
updates. We continue tomove forward with the clinical
trial “for the people, by thepeople.” Our goal is to prove
the hypothesis that the BCG
vaccine can reverse type 1
diabetes and provide a safe andaffordable treatment option for

it will not be sold by a

We hope you will help us

this disease.

pharmaceutical company, butcontinue to move thisrather it will be made availableimportant work forward.

based simply on the price toPlease contact us if you haveproduce. We have gone to great

any questions:lengths to establish labeling

diabetestrial@partners.org.

and manufacturing to ensure

Thank you again!

Our work is part of growing
global consortium ofresearchers who are testing BCG
in the treatment and preventionof a variety of autoimmune
diseases, including type 1
diabetes and multiple sclerosis.

that BCG will remain available
and affordable. It also means we

Sincerely,need to fund this research withIf BCG is approved by the U.S.

donations from individuals andFood and Drug Administration,private philanthropies.

Denise L. Faustman, MD, PhD

UpdateFall 2017

Phase II Trial Updates
We now have over $22 million raised for thePhase II clinical trial and are working toward
our $25 million goal.
We are also pleased to announce that the Phase II
trial is fully enrolled as of the summer of 2017, with 150$20 m
raisedvolunteers with type 1 diabetes taking part in the trial (100who will receive BCG and 50 who will receive a placebo).
All of the BCG patients have received at least one dose ofBCG. Over the next five years, the participants will receivea total of six vaccinations: two vaccinations in the firstyear and one vaccination a year for the remaining fouryears. The primary endpoint will be to see if BCG can lower
HbA1c. HbA1c is the measure that clinicians use to get
an overall picture of what your average blood sugar levels
have been over a period of weeks/months.

New Data Coming from
Phase I Trial PlaceboGroupAs part of moving from
Phase I to Phase II clinicaltesting, we were able to give
BCG to the three patients
who received placebo in thePhase I trial under a special“open label” study. Data
from these patients, alongwith long-term follow up
data from the BCG-treatedpatients who participatedin the Phase I trial, willcontinue to inform us of theefficacy of BCG.

We Are Seeking Patientsfor Future Study

BCG Working Group andWeb Site

Our Phase II trial is fully
enrolled, but we are still looking forpatients for future study groups.
For the Phase II trial, the FDA
recommended we test in a narrowly
defined population of patients withtype 1 diabetes, one that has similar
characteristics to the patients weenrolled in the Phase I trial (i.e.,similar ages, years since diagnosis,etc.). That does not mean we think
BCG will only work in those patientgroups. We are actively planningand designing new cohorts andstudy groups. If you are interested,
please contact the lab at

The global consortium of BCG
researchers we helped organize
has met again, during our thirdbiennial “BCG and TNF Signallingin the Treatment and Preventionof Autoimmune Diseases”
conference, held on October
7th in Athens, Greece. At theconference, we shared updates
on BCG trials and on theeffects that BCG may haveon the immune system that
could prevent and reverse
autoimmunity. We also
launched a new web site
for the working group,diabetestrial@partners.org.

www.bcgandautoimmunity.org.

www.faustmanlab.org

Updates on How BCG
Changes the Immune
SystemReview Article Published
Recently, Dr. Faustman
published a new review article inDiabetes/Metabolism Research andReviews on the role of BCG and
TNF in type 1 diabetes. The article
is called “TNF, TNF Inducers and
TNFR2 Agonists: A New Path toType 1 Diabetes Treatment.” Itprovides a great overview of ourwork and the potential of BCG:Over the past year,
Dr. Faustman has presented
updates on the BCG clinical
trial at three international
diabetes conferences:• Immunology of Diabetes
Society• The American Diabetes
Association• European Association for
the Study of Diabetes
One of the key updates was
new data on the mechanism
by which the BCG vaccine may
restore the proper immune
response to the insulinsecreting islet cells of thepancreas. In their research,
Dr. Faustman and teamshow that BCG may inducea permanent increase in
the expression of genesthat restore the beneficial
regulatory T cells (Tregs) thatprevent the immune systemfrom attacking the body’s owntissues.

http://onlinelibrary.wiley.com/“We and other global efforts
have known for some time that
restoring beneficial Treg cells
might halt the abnormal selfreactivity in type 1 diabetes
and other autoimmune
diseases, but therapies torestore this immune balance
have not achieved long-lastingresults,” Dr. Faustman statedat the American Diabetes
Association conference. “Thediscovery that BCG restores
Tregs through epigenetics– a process that modulates
whether or not genes areexpressed – is exciting. Thisnow provides a better ideaof how BCG vaccination
appears to work by powerfully
modulating Treg induction andresetting the immune system
to halt the underlying cause of
the disease.”doi/10.1002/dmrr.2941/fullMore Data on Long-Term
Pancreatic Function
One of the key learnings fromthe Phase I clinical trial has been
the ability of type 1 diabetics tokeep producing insulin years
after diagnosis. This was first
discovered and published by theFaustman Lab in 2012. Multiple
labs have now confirmed theseresults and expanded on what
we know about the importanceof blood sugar control, insulin
production and outcomes in type1 diabetes. This information has
been vital as we begin to design
new treatment groups for futurestudies. So, remember, yourpancreas is still working long after
you are diagnosed.

UpdateFall 2017

Prince of Egypt Event
On October 12th, Stephen Schwartz hosted a special
fundraising event in California as part of the world premiereof his new musical, Prince of Egypt, at the Mountain View
Center for the Performing Arts in Mountain View, CA. Thiswas our third event with Mr. Schwartz following our fantastic
Pippin event in 2013 and Schikaneder event in 2016.

How You Can Help
Please consider making a taxdeductible donation to sustain themomentum of this type 1 diabetes
research program. Every gift makesa difference.
1. To make a secure online donation,
visit www.faustmanlab.org andclick on “Support.”

Drive to Cure Diabetes
Kathy Kerney and herteam put together anotherincredible golf tournament.
This year was the ninth
tournament they haveorganized, and the date isalready set for next year.
Mark your calendar forJuly 30, 2018.
facebook.com/

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 Laboratory
Massachusetts General
Hospital-East
Building 149, 13th Street, CNY-3601
Charlestown, MA 02129

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

drivetocurediabetes/.
Thank you for joining us in thefight against diabetes!

Overcoming Obstacles for Diabetes
Some of our more adventurous supporters convened in
Hingham, MA on October 22nd for the annual RenegadeRun. Participants overcame a four mile obstacle course tohelp raise funds for our research.
http://typeonerenegaderun.com/Seeking more information about this type 1 diabetes research?
Visit www.faustmanlab.org or Email: DiabetesTrial@partners.org.

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