UPDATES
from Dr. Faustman’s Laboratory at Massachusetts General Hospital

Issue One: Fall 2005

Updates on the Clinical Trial

In order to begin the ﬁrst part of the clinical trial,
we had to develop a new blood test (also known
as a blood assay) to evaluate how well the therapies
to be tested are working. This assay must be highly
reliable and accurate. To develop it, we have beenbuilding a machine that separates different typesof blood cells. We will use the cells isolated by themachine to develop the assay that counts the numberof defective white blood cells that are causing
disease. We have installed the equipment needed
for the cell separation and blood assay machineryat Massachusetts General Hospital. This machinery
includes the robots and robotic arms that will
perform steps on an assembly line. In addition, we
continue to build a computer program and engineer
the many sequential steps to separate white blood
cells from red blood cells. There will likely be over

800 separate robotic steps to this process and boththe movement of the robotic arms and engineer
platforms will be designed and tested. Our goal
is to have this equipment ready to ramp up thehuman clinical trial for 2007.
In addition, we’re collecting blood samples fromvolunteers as far away as California and Manitoba,Canada, as they visit us here in Boston. The
laboratory is using these samples to ﬁne-tune thecell separation procedure and clinical trial assay.
The steps we are taking are critical for theupcoming trial. If we were to proceed without theblood test, it would be like giving insulin without
ever being able to check blood sugar levels. We
need to have a rapid blood test to know if we arecorrectly dosing the drugs we will be testing.

Corroborating Research: Good News from Other LabsEvery month it seems we learn of more research that points to the potential of regeneration of the insulinsecreting cells in the pancreas. From the American Diabetes Association’s 65th Annual Scientiﬁc Sessions, heldJune 10–14, 2005, three abstracts further conﬁrm and support some of the key points of our research:• In Evidence for Sustained Islet Turnover in Humans with Long-Standing Type 1 Diabetes (Abstract # 1612-P),
researchers found that even those with long standing type 1 diabetes have beta cells that continue tobe destroyed, suggesting that concomitant new beta cell formation must be occurring. They concluded
that type 1 diabetes may be reversible by targeted inhibition of beta cell destruction, a key concept inDr. Faustman’s work that will be tested in a clinical trial to be led by Dr. David Nathan.
• Comparative Analysis of Therapies Tested in Animal Models of Type 1 Diabetes and Human Clinical Trials(Abstract # 102-OR) identiﬁed several reasons that may account for past difﬁculties in translatingtype 1 diabetes research from mice to humans and showed that identifying the impact of dose and timeon the efﬁcacy of potential therapeutic agents such as bacillus Calmette-Guerin (BCG) may be critical to
future clinical success. Dr. Faustman’s major research efforts are to develop a human blood assay that willallow researchers to monitor the efﬁcacy of BCG in terms of dose and timing of administration during
clinical trials.
• Finally, In Vitro Generation of Insulin-Producing Cells from Adult Spleen (Abstract # 1642-P) provided supportfor Dr. Faustman’s past research that identiﬁed the splenic stem cell that can form islets. Multiple labs have
now conﬁrmed the existence of this stem cell. This newest data shows that these cells can be grown in thelab, transplanted into diabetic mice, and reduce hyperglycemia in these mice.

page 2UPDATESfrom Dr. Faustman’s Laboratory at Massachusetts General Hospital

Events in NYC and London

I was invited to give two lectures this fall focusing
on the regeneration of islets in type 1 diabetes.
The ﬁrst invited lecture happened in September at
the New York Academy of Sciences, where I spoke

to the Metropolitan Diabetes Society. The second
lecture will take place in London in October at theInstitute of Cell and Molecular Sciences at Queen
Mary’s School of Medicine.

Fundraising Update

The Iacocca Foundation’s JoinLeeNow (www.joinleenow.org) got a big boost this summer. Most of you
have seen by now that Mr. Iacocca is back on television doing commercials for Chrysler. The bigger news
is that Chrysler has agreed to become the ofﬁcial sponsor of JoinLeeNow and will work with the Iacocca
Foundation to raise the money for the clinical trials. JoinLeeNow has over $6 million already committed.
Mr. Iacocca was also on TV during the July 31st broadcast of This Week with George Stephanopoulus.
Here’s what he had to say:“People ask me how we’re doing…and now I’ve hit 80 and I’m running out of time. I made acommitment to my wife to try…to ﬁnd a cure before I die. We have about 100 researchers in her
name all over the world. Now, one of those researchers at Mass General had a 10-strike. Dr. Denise
Faustman cured diabetes in mice about a year and a half ago, and once she cured it, she said, “Wow!
Not only did I stop the disease, but once I stopped the disease in the mice, they started to regenerate
new cells.” So now it’s simple: If you’re a mouse, I got you covered. But how you will translate thatto humans, that is tough. I think we’re on the brink of a breakthrough, and that’s why I’m pouring
the coals on it. So now I’m about one third of the way there on the project. Chrysler and its dealers,
thank God, will probably put me over the top. I’m in the neighborhood. I’m getting close.”
Mr. Iacocca isn’t the only one who is busy. Sue Root and Jackie Fusco held another bike ride
September 24-25, “100 Miles for Human Trials.” They started in Mystic, CT and ﬁnished here at the MGH.
The numbers aren’t in yet for this year, but last year they raised almost $145,000. On September 17th,
two separate motorcycle rides raised funds for the Nathan/Faustman clinical trial program. In Hendricks
County, Indiana, Dallas & Reid’s Ride, which raised over $45,000, included an afternoon of music, ﬁeldevents, and food. In Redmond, Washington, the DOCS Foundation’s sixth annual Ride & Cure raisedover $12,000 and included a concert featuring performances from Styx, Foreigner, and REO Speedwagon
on the cliffs of the Columbia River.
I greatly appreciate the help of Mr. Iacocca and all of the JoinLeeNow volunteers. This clinical trial couldn’t
happen without them.
Keep in touch,

Denise Faustman, MD., Ph.D.
Director, Immunobiology Laborator
Laboratory
Associate Professor of Medicine
Harvard Medical School

Immunobiology Laboratory
Massachusetts General Hospital East
Building 149, Thirteenth Street, Room 3601Charlestown, Massachusetts 02129
Tel: 617-726-4084
Fax: 617-726-4095
Faustman@helix.mgh.harvard.edu