Dr. Faustman Research Update

Type 1 and LADA
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Woo-hoo! From www.diabetesincontrol.com:

Update on Dr. Faustman’s Research on Reversing Type 1 Diabetes

The Faustman Lab at Massachusetts General Hospital, run by Denise L. Faustman, MD, PhD, is moving rapidly through the clinical trial challenge to test and possibly establish a vaccine using a generic drug, Bacillus Calmette-Guérin (BCG) to reverse Type 1 diabetes.

The experiments have moved from mice to a clinical trial in humans, and the testing has passed every research milestone ahead of schedule – an astonishing feat for any clinical research project. The Phase I safety trial in people with Type 1 diabetes is near completion. The next step is to find, in a Phase II study, the possible dose and frequency of administration of BCG vaccinations that will benefit patients with Type 1 diabetes.

What makes these trials unique? First, unlike other immunosuppressive therapies for autoimmune diseases that harm both healthy and disease-causing T-cells, this treatment appears to provide a way to achieve “targeted removal” of only autoimmune disease-causing cells. BCG works by causing the release of a natural protein in the body called tumor necrosis factor, or TNF. In mice, temporarily elevating TNF levels destroys the autoreactive T-cells, allowing the insulin-producing cells of the pancreas to regenerate and produce insulin. TNF in mice also elevates a good population of T-cells, thus restoring the immune system to near normal. In other words, the treatment literally reverses Type 1 diabetes, at least in a mouse model of diabetes. In laboratory experiments, TNF also destroys the autoreactive cells in human blood samples. TNF does not harm the normal, healthy T-cells that help fight infection, leaving the immune system intact.

The human clinical trials seek to expand these findings in human subjects. The BCG trial is also unique since BCG is a generic drug that has been used widely as a vaccine for more than 80 years in humans. Currently, BCG is used in small doses as a vaccine against tuberculosis, and it is used in larger doses as a bladder cancer therapy. Because it has been used for so long and in so many humans, the safety of this vaccine is well-established. This fact allows the trials to move more quickly through safety studies compared to clinical trials that test new drugs.

With a generic drug, it can move BCG to the public rapidly if the drug is indeed found to be safe and effective in patients with Type 1 diabetes. The phase I safety study is following the enrolled patients over months looking for changes in the T-cells and also testing the reliability of the blood tests for tracking disease-causing T-cells.

The BCG trial is one of the few translational studies in human testing where the animal data showed disease reversal in diabetic mice that had hyperglycemia, not just in mice with normal blood sugars and with a predisposition to diabetes. Therefore, this human trial at Mass General is designed to treat people with established Type 1 diabetes unlike most human testing that focuses only on not yet diabetic patients at risk for the disease or newly diagnosed diabetics with a short time period of high blood sugars.

Click here for more information in a special report.

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" I’ve got high hopes"

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This is greattttt!!! Its coming, I believe:) We all need to keep our heads up!!

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I am very excited about their work and how quickly they are moving forward. It also seems to be the only real solution that fixes the problem…faulty immune system. Everything else requires that I continue to be concerned that my own system will destroy or disable the treatment provided. Even if it did not…I would continue to attack other tissue. The inflammation response can attack inner ear, joints, etc. I cannot wait to see the results of Phase 1. How many of us have provided blood samples? I am thinking of making the trip back east to do so. I used to work at MGH and lived on the south shore:) Interestingly, Faustman was trained right here in my home town…where the first islet transplants were done. She is a neat and committed researcher. If anyone has personal experience w/ participating in the trials in any way…please share:)

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I asked friends & family to send donations to Dr. Faustman instead of birthday & holiday gifts.

So encouraging! Hoping this has implications for other autoimmune diseases as well.

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How long before big Pharma sets fire to Dr. Faustman’s lab?

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How long before Big Pharma sets fire to Dr. Faustman’s lab?

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Amazing that they haven’t given the combo of losing Type 1 customers & that a generic drug is involved. Hmm, maybe her lab is safe since Type 1s are only 10-15% of their customer base.

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I’ve been a fan of Dr. Faustman for a long time. Thanks for posting this.

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I have to say I am really excited about this research.

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Great idea:) I thought of having a fundraiser…maybe a Tu meetup here in St. Louis! Share stories, laugh, and put some sheckles in for both Tu and Dr. Faustman:) I really wish I was independently wealthy!! Ok…or JUST working!

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I remember something to the effect…that she is watching her back. I know she got flack from other researchers and colleagues…even at MGH. I so admire her committment and tenacity…a true physician and scientist:)

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I have my fingers and insulin producing pancreas cells crossed! This sounds so encouraging.

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Does anyone know how long these trials are going to last? Is Dr. Faustman going to share some of her research with other facilities like teaching hospitals where they can use the patients as patients for new and upcoming researchers to watch the process begin to work. It’s too bad that when a small breakthrough happens that other clinics and labs refuse to look at the validity of the research they didn’t do. Let’s just all say a prayer that this is something that may work for those of us that have Type 1 related to a viral attack! Pray…Pray…Pray for the cure to be found yesterday!!

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I think other researchers are waiting to see the results of Phase 1…should be out in early 2010. Phase 2 is projected at taking 3 years. Interestingly, I filled out the patient information stuff for Phase 2 and all the questions were exactly the same questions that I was sent from my doctor who is doing a study on related autoimmune disease. I hope, if all goes well, that they open up the Phase 2 and 3 trials to other locations across the US:) Always good to be in the pool of potential research candidates at one’s local teaching hospital. I purposely picked my MD (endo and researcher) for that reason. I am so hopeful that one of the current lines of research will lead to some real changes in care…or a cure.

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Be skeptical of organizations whose livelihood depends on perpetuating the disease. Dr. Faustman’s livelihood centers on finding a cure.

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Here’s an interview with Dr. Faustman.

http://www.diabetesmine.com/2009/04/a-talk-with-denise-faustman-the-hope-is-%E2%80%9Cin-the-mechanism%E2%80%9D.html

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I started giving blood to Dr. Faustman’s Trial back in Feb. 2008. I used to go 6 times a year, and I live about 350 miles away (700 round trip). At the beginning, her lab was abuzz with people. However, in the last year I have seen some decline in her crew, and also the frequency of the blood she wanted from me. I was involved in the Phase 2 part of the Trial, where they introduce the BCG to my blood and see how it responds. I went from 6 donations a year in 2008 down to 4 in 2009.

The last time I went (late Nov. 2009) Dr. Faustman was the only one there. She actually came down and brought me up from the lobby, which has never happened before. After my very first visit (where I actually sat down with her for about half an hour to talk about the trial), if I saw her at all, she was busy at her desk. Now I do not know at all what is going on there, but I was hoping to be included in the Phase 3 Trial (she said it had already started). She told me that she had enough participants and then didn’t ask me to come back after SHE drew my blood. Maybe it was some crazy off week for the trail center (first Tuesday after Thanksgiving), but it was weird. She thanked me very much and said (in so many words) ‘take care’ and I asked her when I should come back. She said she had plenty of blood and really didn’t see my need to come back for awhile. I pressed her for a follow-up appointment and she gave me one in August of this year.

I really don’t know what any of this means, and I have been looking forward to the winter 2009 Newsletter, which hasn’t come out yet. Maybe she has so many participants, that she feels bad asking me to drive 700 miles to donate blood (I try and make a day of it in Boston, since I am only at Trial Center for about 20 minutes).

As far as opening up the testing sites in other areas of the country, I can tell you why this probably will not happen anytime soon. The testing equipment they use in this trial is unique and VERY expensive. It’s also not easy to disassemble and reassemble elsewhere. Dr. Faustman explained this to me because I was curious if people in other areas of the country might be able to participate.

I’m not saying one way or another what is going on here, but that has been my experience in the trial since Feb 2008. If anyone knows anything else, please respond.

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Bill,

Thanks for the info. I hope Dr. Faustman’s research isn’t slowing, or that this isn’t the result of funding problems.

I contacted her research team to donate blood. Someone returned my email promptly, said they weren’t scheduling appts until Sept & they’d be in touch.