Meeting Dr. Faustman
In early January I visited the Mass General Hospital research lab where Dr. Denise Faustman is leading research into a possible cure for type 1 diabetes. I was there to give a blood sample for use in this research.
If you've ever met someone you've admired for a while, or maybe a rock or movie star, then you'll know how I felt as I spent time with her.Over the last two years I've taken part in two bike rides to raise funds for this research. But now I had a chance to actually help support the research in a more direct way.
The lab is drawing samples of blood from people with type 1 diabetes, and 'healthy' volunteers. Because they're having trouble getting healthy volunteers, they ask everyone with diabetes to bring a volunteer with them. These folks must not be directly related to you, and not have any auto-immune diseases. I was fortunate that a friend of mine from work kindly offered to come with me. Paul is one of the founders of the yard sale search site GoYarding.com.
I met Dr. Faustman early in the morning. They do all the sample collection early in the morning so it doesn't interfere with work and school schedules. While we talked, she collected four test tubes of blood from me. She explained that some of this blood was tested using machinery they're developing and some was tested manually by one of the researchers. I believe that manual testing takes about one day to complete.
They're trying to accurately measure the amount of T cells that I have. Her theory is that these T cells are responsible for destroying my insulin producing beta cells. These cells also produce amylin. This is now available as an injectable drug called Symlin that I've written about several times.
You can see a short video of Dr. Faustman and an animation of how they believe the T cell process works on the Iacocca Foundation website.
Some of the blood is also sent to another research lab. They have a method for measuring autoantibodies in blood. She explained that autoantibodies are produced when beta cells are destroyed. So if these were found in my blood, that would indicate that my body had recently lost some beta cells. Which would mean that my body is still making beta cells, 35 years after I first got diabetes.
She hopes to start trials before too long where they will be administering low doses of BCG to see if this can destroy these T cells. BCG is used in Europe as an inoculation against tuberculosis, and is also used as an immunotherapy treatment for bladder cancer.
BCG has been around for a long time. As it's already approved for use as a medical treatment, it is likely that getting approval to use this for other purposes will be quicker and easier than for a new drug. It will probably also be less expensive.
Dr. Faustman's hope was that the FDA would permit trials with low number of subjects. This makes it easier to administer the trials because getting enough people is always a challenge and testing the outcomes is easier with smaller numbers.
She explained that there will likely be several trials with increasing doses, so they can determine if it works, and at what dosage levels. Clearly having automated measurement machinery will make this process a lot faster. They've been working on developing this machinery over the last few years.
I told her that it would be wonderful if they started a blog, even a low-volume one to keep everyone informed about their progress. But she's concerned that will take away from research time and also that the blog may draw a lot of comments that would need to be read and handled.
I imagine the entire lab must feel a little like Banting and Best after insulin was first discovered. They had hundreds of parents from around the world contacting them to try and get their children treated. And that was in the days before the internet and e-mail.
It was a real pleasure spending some time with Dr. Faustman and getting an update on their progress. I have another appointment in September to get another blood draw. As Dr. Faustman pointed out, this is an easy way to get the latest news. And by then I hope to have completed another bike ride to support her exciting research. I can't wait!
Labels: bikeride, cure, diabetes, faustman, research
posted by Bernard @ 12:15 AM
20 Comments:
Exciting stuff to be involved in! I hope you'll keep us up to date as much as you can.
bernard,
what an awesome visit that must have been! her research sounds fascinating!
landileigh
Is it a specific type of T-cell? We have quite a few, right? And don't we need most of them? I'm sorry if these are stupid questions, I'm just curious! Sounds awesome to me, and how cool that you got to meet her and participate in this good work.
Beth
I believe it is a specific T cell. I think that's why this measuring work is difficult. They have to distinguish between the 'bad' T cells and the good ones. There's an animation in the Iacocca video that may help.
I remember reading about this in the past year or two. I asked my doc about it, but his response seemed to indicate this would be used primarily to treat new onset diabetics because it wasn't expected long timers would have any islet cells left. Glad to read there is still hope for us long timers and the research is still ongoing! Will they be sharing the results of your tests with you before the general results are published?
Craig
That's the thing that's always excited me about this research. It's not aimed just at people who were recently diagnosed. As I understand it, Dr. Faustman's theory is that we're constantly producing islet cells and that these T cells are destroying them.
As for sharing the results with me in advance of anyone else...I don't think so. I just happen to be a blogger as well as someone who was giving up some blood. I don't have any special kind of status there.
Great post Bernard - very exciting!
Sounds like it was very cool to meet and talk with Dr. Faustman. Thanks for sharing the experience with us!
I'm intrigued by her research, sp. after reading "Cheating destiny". But there's one aspect that intrigues me. Folks who have organ transplants have their T-Cells suppressed by anti-immune medication (cyclosporine/FK-506) to prevent rejection. Yet, these drugs, when tried on normal folks, don't seem to prevent type-1 diabetes, although they are effective in preventing other auto-immune diseases. Do you have her contact information? I would love to get in touch. I think there's a connection between her research and solid organ transplant recipients, who incidentally develop diabetes if they survive the transplant for more than 10 years.
Bernard,
Less than a month after Joseph's diagnosis, the New York Times published their article announcing Dr. Faustman's breakthrough.
After reading it (and so many others that followed), I was convinced that Joseph would be cured within a few years.
Convinced.
A year later, my focus shifted to helping Joseph live with the disease rather than waiting for that cure.
It just hurt too damn much to do otherwise.
But still.
When I saw the title of this post, I thought, "rock star, it's like meeting a rock star... "
So yeah, I'll be tuning in to read more of Dr. Faustman's progress-- and I'll let myself feel hopeful when I do.
That animation narrative is good stuff!! Thanks for posting that link. Wish I lived closer to the Faustman lab. I'd love to donate blood.
Bernard,
This is exciting stuff! Thanks for sharing.
Wow, Bernard. How cool is that?
This post has been removed by the author.
Olá!
Gostaria de saber se alguém fala a lingua Portuguesa, gostaria muito de trocar idéias sobre a pesquisa da Drª Denise Faustman. OK
msn: gipecar@hotmail.com
talk: gipecar@gmail.com
skype: gipecar
Dear Bernard,
I took love Dr. Faustman like a star. I predict she will win Nobel prize in medicine on day. I met her twice in Boston following "Miles for the Trials" in 2005 and 2006. Love her.
My son,17, has type 1. We cannot wait to hear the results from the BCG trials. Thanks for donating your blood and keeping us informed on this blog. Way to go!
Bernard, thanks for the update on this research.
Unfortunately, BCG has been used in the past to try to halt Type I diabetes with zero success.
It has been tried in Israel and in the States at the Barbara Davis center.
Did Faustman mention what they planned on using for the "second" compound?
Where the hell are companies like Living Cell Technologies and others working on ecapsulated pig islets? They seem to have disappeared, no updates on their sites for a long time...
Bernard, thank you for visiting my blog and for sharing this post with me. More importantly, thank you for contributing to this research. I get a lump in my throat thinking that you are helping in finding a cure for my dear son and all the others who live with this terrible disease. May you be blessed with good numbers today. :-)
Bernard, please check out the video I put together, trying to get more publicity for this research. http://www.youtube.com/watch?v=PhAtlAtnKwY
Bernard - like you, I had the privilege of being a lab rat (and I actually like this term, because it's just as snarky as I am) for Dr. Fautman in March. Coincidentally, I also recommend going to Boston over St. Paddy's Day. I'd never been to Massachusetts before and while my husband (who is disgustingly healthy and sans autoimmune illnesses of any sort) wasn't thrilled to be walking around on the Liberty Trail in what turned out to be a cold snap, I felt the trip back east was worth it on many levels. Bunker Hill was worth it.
I understand the feeling behind the word 'rock star' when describing someone who could make or break you. Dr. Faustman was the epitome of kindness when we met with her and took the time to not only diagram the two separate parts of what would be the kind of treatment that would evolve from this study, but answered my questions on some of the material that had been presented by the media regarding the upcoming trials. Days before we met with her, her lab announced the beginning of the Phase 1 trials utilizing the BCG generic.
Dr. Faustman was not only humble, but honest about what she was trying to accomplish. It isn't going to happen overnight and any of the media stories that say so are misleading (idiot reporters). The actual cure for Type 1, according to her, consists of two separate treatments. The first treament is the one that's been outlined in the media - using BCG to destroy the defective T-cells that are constantly destroying the new beta cells the body is attempting to create in the pancreas. This would probably take an injection of the treatment at regular intervals, if used on its own. Since the drug she is using is a generic, and therefor cannot be patented or claimed by a big pharmaceutical company, her funding has been limited to donations from outsiders (Lee Iaccoca, for example). Seems like big Pharma is more interested in keeping us sick and dependent on their overpriced insulin. Even the JDRF, instead of giving her a grant, commissioned separate studies to disprove her (WTF? I thought they were trying for a cure!!!). Despite all of this, she managed to get enough funding for Phase 1 trials. Now she's having to canvass for money to conduct Phase 2 trials, which would expand the number of individuals receiving the treatment, and so on. So yes, I understand that she doesn't have time to blog or to keep track of each individual email. She and her staff are VERY busy.
The second half of the treatment, she explained, was to develop a drug that would keep the dysfunctional T-cells from being formed in the marrow in the first place. Since that is where all blood cells originate, it seems like a smart place to start. Unfortunately, there is no drug that will do this right now and the cost to develop something like this would be in the billions of dollars, since the drug would have to meet current FDA standards and pass all of the necessary tests. that takes a great deal of not only money, but time. Even if Big Pharma was to try to come up with something like this, the population it would serve is too small for the drug to make a profit, when all of the developmental costs are factored in. So Dr. Faustman is working on the part of the cure that can be attained right now, namely using the generic drug to get rid of what's killing our beta cells.
Like Bernard, I believe that this woman, and Dr. Nathan (who is administering the Phase 1 trial) are the Banting and Best of our time. They won't make millions off of this, like a Pharma executive will. Dr. Faustman doesn't work in a huge sparkling office with a secretary and all of the perks of a Big Pharma job. When I was there, the blood pressure cuff they had to take my sample and my husband's had definitely seen better days. I'm half tempted to simply purchase one with my FSA dollars and send it to her. These people are working on a shoestring and they are trying to do something that no one else in the community that claims to want to help us seems willing to do: stop the problem at its source and prevent it from happening in the first place. So what can be done to get this potential cure, which doesn't require any stem cells transplants, embryonic research, animal-based transplants etc. out and to the people who might benefit from it faster?
You can donate money directly to her. She has a link set up at the bottom of the page so that those who wish can donate whatever they want to help fund the trials.
Here is the link:
http://www.faustmanlab.org/
No messing with a third party, no wondering where your donation dollar goes. If everyone who reads this blog just sent in $5, I suspect that she might be able to afford another blood pressure cuff to help the donations. I'm not part of her organization, just another lab rat, albiet an enthusiastic one. I wish I could be part of the clinical trials, but unless they expanded them to multiple locations, it will have to be done with the help of the good Type 1 diabetic adults of Massachussetts. Any support you can give this researcher is needed and would probably be deeply appreciated.
In a recent article, Dr. Faustman described the three weeks she spent as a clinician when she was out of college, dealing with diabetics and the complications that arise from this disease. After three weeks, she said that she'd had it - she felt she had to do something to fix the problem at its roots, not just take care of the carnage that this disease causes and that no one was trying anything to help those who had had the disease for a long time. She wanted to be that person. I say we help her.
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