A Faustman Bargain? — Part 1

Dr. Denise Faustman As one of the best-known names in Type 1 diabetes research, Dr. Denise Faustman often finds herself embroiled in controversy — for her methods, for her approach to funding, and even for “cruelly rais[ing]” the hopes of people with diabetes. With so much discussion surrounding her work, we thought it would be good to hear from Dr. Faustman herself. In this part of the interview, Diabetes Self-Management‘s Quinn Phillips and Dr. Faustman discuss the basics of her research.


Quinn Phillips: How did you get started down this research path?

Denise Faustman: I did an MD and a PhD, and in the middle of my PhD, I met a guy who had discovered the first way to get islet cells from a rat pancreas — his name was Paul Lacy [a prominent pathologist who is considered the father of islet cell transplants]. And I thought, oh my gosh, there’s the cure for diabetes! If you can get islets from a rat pancreas, now you can just transplant them like a heart, kidney, whatever. So it seemed like it was a translatable concept, and it seemed so close to human trials. Of course, I did PhD work for six years trying to get islets out of a human pancreas and realized the translation was not so easy. After I got recruited to Harvard, the lightbulb went off that putting islets in people with diabetes is a problem because the disease recurs. So I realized early in my career that there were big challenges out there, and I wanted to do something that related to people but that also used really fundamental science.

QP: What exactly did you do in your mouse experiment?

DF: Well, first of all, people need to know that the autoimmune attack in Type 1 diabetes involves good and bad cells, so we want to go after the bad ones. It’s a concept that people understand: For cancer, you want the magic bullet that kills the cancer cell, not the good cell. And for autoimmunity, like Type 1 diabetes, you want the magic bullet that kills the bad T cell — an immune system cell — and not the good T cell, which helps regulate the bad ones. After about 18 years of work, we found out that we could identify, in mice, the bad T cells and kill them. So we decided to set up the first islet transplants in end-stage diabetic mice. Most of the data that was coming out then — and in news reports that you hear even today — used mice that scientists had artificially made diabetic, not ones with diabetes naturally. So we decided to pick really tough mice, ones with a gene that gives them diabetes, and put in these drugs to kill bad T cells, then do an islet transplant. We said, “Let’s finally get islets to work in end-stage diabetic mice.”

So this is what happened — this is when the science gets good! We saw, in the first series of animals, about 85% of the mice restored to normal blood sugar levels. And then we did something that led to the major discovery in this work: We took the islets out after they had been in for hundreds of days to prove that blood sugar would go up. And what happened was, the blood sugar stayed flat. That was the discovery of this massive islet regeneration that had not been documented before, because the concept was, “you need spare body parts,” so in diabetes you need spare islets. But what these animals showed us is that if you remove the bad T cells in a very targeted way, there’s spontaneous healing of the pancreas. That was in 2001, and in the paper we published, we weren’t allowed to use the word “regeneration.” Nobody believed the pancreas regenerated. But it was not a subtle finding. I mean, there were animals with very high blood sugar and animals with a normal blood sugar level, and you had pancreases harvested from them with big, huge islets in them. So it was not subtle. But it was unreal that this could happen! We set out for 18 years trying to get islets to work in diabetic animals, and instead discovered pancreas regeneration.

QP: Do you think the editors who didn’t let you use the word “regeneration” are emblematic of the medical establishment you’ve been dealing with?

DF: Yeah. I think when data comes out that is what some people call “disruptive data” — in other words, data that doesn’t fit the paradigms everybody is comfortable with — the first response is the normal human response: It can’t be. It can’t be that the pancreas regenerates. It can’t be that you can reverse diabetes if you already have it — if you’re a mouse. But now you can go to seven different centers worldwide, if you’re a mouse, and this therapy works really well. It took about seven years for all the duplication to occur, but if you’re a mouse, everybody’s got you covered!

QP: What drug did you use in the mice?

DF: We used two compounds, two drugs. And the mice never needed to be retreated again in their lives. In the human trials that we’re moving forward with, we have one of those two compounds, the drug BCG, being used. It may not seem like this to the public, but that’s a very rapid move from mice to people. And the reason it’s so rapid is that BCG is a generic drug that’s already approved for other indications. So without the normal delays in manufacturing and huge costs of manufacturing, and primate studies for four or five years, we shaved off probably $20 million and 10 years, by taking this generic-drug approach.

The other drug is a protein complex called class 1 and self-peptide that the good T cells should be using to kill the bad ones. Everybody — every mouse, too — makes T cells all their life. And most of them are rogue, and they should die by a process called negative selection in the thymus or bone marrow, and other locations. But what happens in autoimmunity is a few of these guys escape. They should have died in the bone marrow with class 1 and self-peptide, but it’s not there in diabetics — there’s a deficiency of it, so the bad cells get out. Once they become highly activated and in the pancreatic tissue, just adding back the class 1 and self-peptide doesn’t kill them anymore. That’s what we need the BCG for. In the mice, when we added both class 1 and self-peptide and BCG, it was a one-time treatment. In the human trials we’re just killing the most vicious cells that are attacking the pancreas, using BCG.

QP: How is BCG administered in the human trials? Is it administered just once?

DF: We’re using it in the skin injection form, and we’re actually doing two tiny doses 4 weeks apart. This is still very, very low dosing. It’s kind of like the beginning of insulin, if it were 1920: You’re just hoping you can monitor blood parameters that tell you something’s happening.

QP: So you’re not expecting remission of diabetes in this phase?

DF: Oh no, that’s not going to happen yet. Right now we’re trying to monitor the T cells — we’re looking at good T cells, and we’re looking at the elimination of bad T cells that are autoreactive.

QP: Why are you starting with just BCG?

DF: There are economic reasons to move forward with BCG first. Keep in mind that the average drug in the U.S. costs a billion dollars to develop — so we’re shortcutting this in time and cost by using a generic drug. If Bill Gates came through the door and said, “I want to develop class 1 and self-peptide — hurry up!” we’d be happy to do that. We just can’t raise the money for two programs at once. The generic drug development is totally sponsored by the public, as you may know, since there’s not a huge economic incentive to develop cheap, rapi
d therapies for people wit
h existing diabetes.

Read part 2 of the interview here.

Next week: Dr. Faustman discusses the controversy surrounding funding of her research — including denial of funds from the JDRF — and competing approaches to research by drug companies.

For more information about Dr. Faustman’s research, visit www.faustmanlab.org

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9 thoughts on “A Faustman Bargain? — Part 1

  1. The Medical Profession is over-run by Politics.
    (Egos)Which is why some research becomes an end in itself rather than a useful tool to be applied to those in need and who can benefit from a new way of thinking, a new medication or a modality that has not been used before.

  2. This is a huge step forward. It makes you wonder about any collusion between fund raising organizations and the pharmaceutical companies. This could be a big blow to both. Keep up the good work by reporting these issues.

  3. Thanks for posting this interesting interview. Now that the tests on mice have been duplicated, perhaps a for-profit clinic will sponsor more of the research – when profit is possible, more effort is expended to achieve results.

    We may prefer a more altruistic approach, but the profit motive is tried and true.


  4. Hello,

    I have been a diabetic for 17 years. I acquired this after a surgery. The surgery was a laperotomy to clean my fallopian tubes. My fasting blood sugar at that time was 93. A year after the surgery I was diagnosed with full blown Type I diabetes. The pills didn’t do anything for me. I don’t know what happened in that surgery process but I couldn’t live without my insulin. The surgery left me with much pain for years. 2 of my brothers had surgery after me. One for his neck, the other for his shoulder. They both became Type II diabetics within a year after their surgeries.

    Here we are almost 90 years since insulin was discovered. It is a blessing because many people would absolutely have been dead years ago.

    I have issues with the drug industry. My belief is that if diabetes were cured then the big boys wouldn’t have our flesh to get rich from. To cure us would be would almost shut them down.

    Example of this is the cost of the insulin, test strips, and supplies. I can buy a local store brand test strip at 1/3 the cost of the name brand that my insurance company REQUIRES me to purchase. And then we have the partnership of insurance working with drug companies to rape the people plus manufacturers that have to purchase insurance. They are actually raping the nation.

    My next suggestion is for a nation health plan. If we had health care for all then we wouldn’t need the insurance companies. If we had someone watching over the drug companies ridiculous prices then we would have care for all. I have heard that the drug industry has many layers on patents. After a reasonable time of MUCH PROFIT others still cannot use their patent because of this layering of thousands of restrictions.

    You can see that I have strong feelings about our drug companies and insurance companies. I think GREED drives their decisions. Feeding off our misfortunes.

    I am praying that Dr. Denise Faustman can help develop a CURE. You go girl!!!!!

  5. In the usual process of capitalism and the profit derived from investment in research and development it is understandable that corporations, individuals and etc. should receive rewared for their efforts.

    There is also a time when advances in human health should become the focus of financial outlay for specific research. The bizarre multiplication of(free) BG meters and the accompaning super expensive test strips befits the huge profit motive.

    The onset of diabetes has determined that now is when we need to priortize on any pathological advances in treating and curing this intractable disease.

  6. I would sure love to have this done to me, I have had Diabetes, Type 1 for 59 years, still have all my limbs, monitor my blood sugars at least 8 times daily…….I like to maintain an average blood sugar level of 120 or below. I try really hard all the time to do this. It’s such a pain. I am also on the Insulin Pump since 2000 year. Much better than taking multiple shots daily as I used to do. I’d be willing to volunteer for this treatment!

  7. I’ve known of Dr. Faustman’s work for years now, and I am incredulous how little publicity she has gotten. Every time I hear some celebrity going on about stem cell research, I want to be able to grab them by the shoulders, shake them a few times, point them at Dr. Faustman, and say, “what about this?” Why is it that stem cells get so much publicity, and this research, which is so much farther along to an actual cure, gets ignored?

    I’m very afraid one of the answers (and I am also very sure there are many different answers in addition) is plain old $$. It’s far more lucrative to treat diabetes than to cure it. If you study the history of Dr. Faustman’s attempts to fund this research, it’s hard to draw any other conclusion. It makes me wonder how many other medical breakthroughs might be currently suppressed because the pharmaceutical companies don’t want to lose their cash cows.

  8. This has been the greatest news of the century. To bad it draws sharp critisim by diabetic organizations like the JDRF. I have followed a number of her papers and interviews. We know that we can not expect help from the drug companies. This would cut into their $35 Billion a year industry. As far as teh other in the medical feild who refuse to think that just maybe the beta cell could grow back is hog wash well remember when the world was flat.

    If we want to see a cure it is up to us Diabetic. We can not count on anyone else. Write your congressman, your senator and even President Obama. Remember that he pleaged $5 Billion dalors for research into finding a cure for high cost diseases. Push hard and when they push, we just push harder.

    De Oppresso Liber!

  9. To all Dr faustman supporters , you could help speed up the funding of Dr. Faustman’s most promising research:

    A) to reverse type 1 diabetes
    B) at cheap price (generic BCG)
    c) with the safest approach (long history of safety use)

    How: You can join the lobby group newsletter totally free at http://www.hdiabetes.com
    It’s free to join. We will be stronger in group.

    Spread the word about the lobby group. We will fund the research fast.

    Thank you

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