Updates
Fall 2016

from the Faustman Laboratory at Massachusetts General Hospital

A Note from Dr. FaustmanGreat things have happened
this year! Most importantly,
we began enrolling participantsin our Phase II trial. This
double-blind, randomized,
placebo-controlled study in
150 participants will continue
to investigate the ability of theBCG vaccine to reverse type 1
diabetes. Supported entirely byphilanthropy, it is a clinical trial
by the people, for the people.
With this trial, I hope that wewill find a way to stop the bodyfrom destroying the insulinproducing cells of the pancreasand help the body restore
blood sugars to normal levels.
A decade ago, that may haveseemed unachievable, but I
believe it is a goal that we arecoming closer to every day.
We are very proud to be doing
this work and thank all who

have helped us get here. We aretouched by all of the individuals
and families we hear from andsee who are affected by type 1
diabetes, from the patients that
donate blood for our research
to those who send in messagesof support. The dedicationand energy we are giving tothis project is matched—if notexceeded—by the dedicationand energy of our supporters.
Thank you all!
I know that we all share thishope, and the Faustman Lab
On a more sober note, we arewill continue to work towards
very deeply saddened each time this goal.
we learn about a life lost to type1 diabetes complications. As
Please contact us withyou read further, you will see
any questions atthat this is something we touch diabetestrial@partners.org.
upon in one of the updates inThank you again!
this newsletter. The loss of life
caused by this disease makes
Sincerely,our research goals very real tous and very clear – we must findDenise L. Faustman, MD, PhD
a way to stop type 1 diabetes.

UpdateFall 2016

Phase II Trial Updates
We now have over $20 million raised for thePhase II clinical trial.
The Phase II trial is a double-blind, randomized, placebo
controlled study that will investigate the ability of the BCG
vaccine to help those living with type 1 diabetes permanently
lower their blood sugars. The primary endpoint of the trial will
be to see if BCG helps to lower HbA1c.
To date, we have enrolled over 130 of the 150 candidates
that we need for the trial, and have given at least one doseof the BCG vaccine or placebo to over 100 participants. We
will follow participants over a five-year period. We will keep
everyone updated with trial updates as they are available.

BCG Trials in Multiple Sclerosis
Moving Forward in Italy

Long-Term Follow Up of thePhase I Trial Continues

Our colleagues in Italy are moving forwardwith a Phase III trial testing BCG in patientswith multiple sclerosis (MS). Led by Dr.
Giovani Ristori at Sapienza University inRome and supported by the Multiple Sclerosis
Society of Europe, the trials will look at theclinical effect of BCG vaccinations in patientswith early signs of MS. This is a follow up totheir Phase II study, which showed that even asingle dose of BCG stopped multiple sclerosis
progression in over half of patients comparedto placebo.

We continue to follow the progress of thepatients who participated in the Phase I BCG
study. All patients who were randomized toreceive placebo during the blinded portionof the Phase I study have now received two
doses of the BCG vaccine, just as the patientsrandomized to BCG did. We expect to publish
data on our long-term follow up of all of thePhase I patients in 2017.

$20 mraised

www.faustmanlab.org

Faustman Immunobiology Lab
Research Identifies & Corrects
Regulatory T Cell (Treg) Defects in
Type 1 Diabetes.
Regulatory T cells or “Tregs” are a type ofwhite blood cell that helps regulate the immune
system, and they normally should prevent
autoimmune conditions like type 1 diabetes.
In our latest type 1 diabetes research paper, we
identified a defect in the Tregs of people withtype 1 diabetes. Using human blood samples(thanks to everyone who donates blood for ourresearch), we showed how tumor necrosis factor(TNF) is one way to correct this Treg defect.
Specifically, TNF treatment of type 1 diabetic Treg
cells “awakens” the Tregs so that they become
potent and functional again, fighting the cells
that cause autoimmunity. Part of what we hope
to do in our trials with the BCG vaccine (whichis a known way to stimulate the body to produceTNF) is to awaken these cells to put up a fight.
Our paper was published online in Nature
Clinical & Translational Immunology in January
this year.

Kiss the Sky to Conquer Diabetes:
A Tribute to Murphy (Murph) Roberts
We featured the amazing efforts of Kiss
the Sky to Conquer Diabetes (KTS) in ournewsletter last year. For those who don’t know,
KTS is a mountain climbing group led by Rick
Noble. They joined the effort to help raiseawareness for and finance our human clinical
trials in 2004, and they have since taken
on some incredible climbing challenges insupport of these goals.
This summer, KTS took on the Six-Pack ofPeaks Challenge, which Rick described as “6peaks, 82 miles, over 27,000’ of vertical gain
and one epic challenge to conquer diabetes!”
Right before setting out on the fifth andbiggest climb of the challenge, the KTS teamsadly learned of the passing of Murphy(Murph) Roberts, the brother of a friend of
Rick’s daughter, Sarah. While hiking in Utah,
Murph experienced a seizure related to his
type 1 diabetes. The seizure caused a fall that
resulted in a series of complications, leading to
his death.
Murph, a member of the class of 2017at Middlebury College in Vermont, isremembered as a great student and person,
and an avid adventurer. He was also
passionate about finding a cure for type 1diabetes. As a tribute to Murph and with theblessing and support of Murph’s family, theKTS team has renewed their pledge to “keepclimbing until we conquer type 1 diabetes.”Dr. Faustman and Charlotte, a young supporter whosevery successful rollerskating fundraiser helped benefit
the Faustman Lab’s research.

UpdateFall 2016

New Musical from Stephen Schwartz
Benefits Our Lab’s Research

How You Can HelpIn September, musical theater lyricist and composer
Stephen Schwartz together with type 1 diabetes advocates
Barbara and Michael Herscovici and their daughter Zoé (anupcoming musical performer) hosted a fundraising eventfor the Faustman Lab at the Raimund Theater (managed by“Vereinigte Buehnen Wien”) in Vienna, Austria.

Please consider making a taxdeductible donation to sustain themomentum of this type 1 diabetes
research program. Every gift makesa difference.

One hundred donors to the lab were greeted by Stephen
Schwartz, Barbara, and Zoé. The guests enjoyed the preview
show of Stephen’s new musical, “Schikaneder,“ whichpremiered September 30th. At the after-show reception,
Stephen gave a special performance for the guests, whoalso had the opportunity to mingle with the cast.

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

Photo: Rolf Bock

Immunobiology Laboratory

The Herscovici family with Stephen Schwartz and cast member at afundraising preview of Stephen’s new musical “Schikaneder.”Interview with Diabetes in Control
Diabetes in Control recently published a three-partinterview with Dr. Faustman. In the interview, Dr. Faustman
discusses why the BCG vaccine may lead to a cure for type1 diabetes, the past challenges of dosing BCG correctly, andhow a cure based on the inexpensive BCG vaccine could
permanently change type 1 treatment. You can find the full
3-part interview at www.faustmanlab.org in our “News”
section, under “Media Coverage.”
Seeking more information about this type 1 diabetes research?
Visit www.faustmanlab.org or Email: DiabetesTrial@partners.org.

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

On the memo line of your check,please write: “Faustman T1Dresearch.”Thank you for joining us in thefight against diabetes!

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