Type 1 cure trials start
This is the most exciting news I've had since my diagnosis with diabetes 25 years ago.Today ClinicalTrial.gov posted a trial announcement looking for people to participate in the first trial for a possible cure for type 1 diabetes. This is based on the research led by Dr. Denise Faustman over the last decade.
To quote the site "This is the first step in trying to cure established type 1 diabetes". Tell me that's not a big thrill.
When I spoke with Dr. Faustman in January, she was hoping that they could trial this approach with relatively small numbers of people. It seems she was successful, they are looking for 25 subjects to participate in this trial. Dr. Faustman mentioned that the early trial would be used to try and establish basic dosing information and determine whether the approach actually works in people.
As usual for this type of trial there will be two groups of participants, some taking BCG and others taking saline as a placebo. You would receive an inoculation at the start and at 4 weeks into the trial. It's a double blind trial. Neither your nor the investigators will know who's receiving which substance.
BCG has been used for many years as a vaccination for tuberculosis. It's also used in immunotherapy for cancer and other diseases. It's low cost, approximately $3 for a tuberculosis vaccination. It's also not 'owned' by a specific pharma company, so it should be readily available.
I would sign up for this trial today...but I don't qualify. My age and previous inoculation with BCG exclude me. I do hope that's not always the case.
If you're thinking about it, please take a close look at both the inclusion and exclusion criteria. There are a lot of these.
We're definitely living in exciting times.
Labels: bcg, cure, diabetes, faustman
posted by Bernard @ 8:51 PM
27 Comments:
OMG. This is incredibly exciting. I wasn't expecting something like this for a very long time.
I'm excited....did I mention that????
I completely lost it when I read your post.
Thanks, Bernard.
For the update and the email making me aware of it.
This is just... wonderful.
Awesome news!!! Thanks Bernard.
Wow!
That is very exciting!
Kinda made me wish I lived near Boston (and knew what my GAD antibodies and c-peptide looked like now).
Thanks Bernard -
Very cool!
k2
I commented on your other blog. But, I'm so excited about this news that I'm going to comment here also.
Woo hoo!!
Don't get too exciting about this. I was diagnosed in 1993 when I was 12 and went through a study with the BCG. I did get the BCG and they concluded after a certain amount of time that nobody in the study with recently diagnosed T1 had their diabetes reversed. I've been there, done that, was lucky to have a shot at it, and wouldn't get any hope up. I hate to be the killer of excitement but I'm the voice who has done it.
This is incredibly exciting and hopefully a step in the right direction.
It's also comforting to know that work is being made, even if this doesn't pan out, that there are scientists who do want to see diabetes eliminated.
Two days in a row of good news. First the Navigator announcement, now this. Very exciting!
The scope of the trial seems limited, though. It doesn't look like they're going to actually try to cure anybody in this study, just see if BCG can reduce the number of bad T-Cells.
Is that how you read it too?
Thanks!
To anyone who's relatively new to diabetes, we veterans rarely get excited about mouse studies. This one's different--even if it's not "the cure", they're definitely on to something. Let's hope for success :)
I know I live too far away from MA to participate, but I emailed the investigators anyway...got a nice note back saying they will keep me on file if the trials expand out here. Thanks for posting, Bernard!
Bernard: Great to hear the news!
Hi Bernard,
Sorry, Bernard..I have to say this, even if you don't want it advertised: You deserve a lot of credit for your tireless fundraising for Denise Faustman's work.
Imagine being able to tell your grandkids about the old days before diabetes was cured. I'd love to see it go away! THANKS BERNARD!
Very exciting - thanks for the heads up Bernard!
Like Kate, I was diagnosed in 1993 and participated in a study with the BCG vaccine. With that particular study they checked to see if it had any affect on glucose tolerance (it didn't), but it will be interesting to see if this study suggests that BCG might make it possible to transplant islet cells without the immune system going after them again. Perhaps I'm partially cured and don't know it yet.
That said, mice are very different from humans in terms of immune function. I'm hoping for the best but to be honest I'm not very optimistic.
What I'm really wondering is how this can possibly be a double-blind trial with the oozing, smallpox-esque scab that pops up at the vaccination site and sticks around for weeks... or am I remembering a different study? Eew.
I am saying an especially fervent prayer tonight. I hope this is the beginning of the end...
I am always hopeful, so this is great news. Too bad Daniel isn't 18 yet - he's not eligible to participate.
I am so excited, I can hardly wait, I really need the treatment now. How old is too old to be treated? Pardon my ignorance please, where may I find the exclusion and inclusion criteria for treatment?
I don't think the Faustman group is even testing a cure right now. They are making sure their new equipment works. The BCG dose they are using is exactly the same as has been given to millions in TB vaccines and extensively tested in the past and found not to effect type-1.
If you want to follow some researchers who are actually testing cures right now, then look at Diamyd (phase-3), or MicroGenetix/TolerRx (both phase-2) or LCT (phase-1). These guys, and many more, are actually testing cures.
GK
GK
Both the press release and the clinical trials say they are testing a cure. So I believe that's what is happening.
I think they're already done a great deal of testing on the assay equipment they have been working on. Dr. Faustman told me that the blood sample I gave would be tested by hand and by machine.
From what I can tell Diamyd is working on a vaccine. Not a cure. Tolerx's TRX4 seems to be similar to the approach that Dr. Faustman is taking with BCG.
Which would you prefer to use? A compound that has been used by millions of people or a brand new (and probably expensive) immunotherapy that hasn't been tested for long-term use.
Right now, I'll continue to put my money on the approach that Dr. Faustman is taking.
That is good news... regardless of the outcome, a step toward a cure is, after all, a step. Oftentimes answers are not found in a linear fashion. I'm obviously not ready to throw out my Humalog and pump yet, but am thrilled that we continue to move forward.
Forgot to add: ...and at 22 years of living with Type 1, I reckon I'm a veteran. Hope is hope, and hope is always exciting.
Well, Dr Faustman can say anything she wants in a press release.
But think about this: what would you measure, if you were testing a cure? BG numbers? Insulin used? I would do both. Faustman is doing neither! Take a look at the official description of her experiment (not the press release), it says:
"Primary Outcome Measures:
concentration of autoreactive t-cells
Secondary Outcome Measures:
Concentration of TNF, TNF-receptors, other cytokines, and c-peptide levels"
Notice: no measures of blood glucose, and no measures of insulin used. That makes perfect sense if they are testing their equipment or testing part of a cure (or both). It makes no sense if they are trying to cure type-1 in this trial. I'm not saying that this is a bad thing: you must test your equipment and your parts, if you are going to use equipment and combine parts. But this experiment is not itself a test of a cure for type-1. Hopefully they will learn enough to run such an experiment in the future.
As for your question, as to which cure would I want: I want one which is safe, effective, and available soonest. I think it is clear that LCT is a year (or a little more) ahead of Faustman in a head to head comparison of cures for non-honeymoon type-1s. Diamyd, TolerRx, and MacroGenics are all several years ahead of Faustman, if they can convert their honeymoon cures into non-honeymoon cures. That's a big "if" to be sure. But Faustman has many big "if"s herself:
1. Will BCG, a treatment that has been tested and found NOT to work in the past, be made to work for her?
2. Will she find a second drug to solve the second part of her problem (that BCG doesn't).
3. Given both drugs: will they result in a cure?
She's got to get lucky three times. The MacroGenics guys just need to get lucky once, and they have at least a three year head start.
GK
In my previous post, I forgot to add, that another measure of a cure would be A1C numbers. This is another thing that Faustman is NOT measuring in her research -- again showing that this is not even trying to be a cure.
GK
GK
I could go into a lot of detail about my opinion on these trials. About how I think Dr. Faustman is a smart and caring doctor. But those are all just my opinion. Those are no more valuable than yours.
Let me just say that I have a great deal of trust in God. If this trial leads to a cure then I will be dropping to my knees and thanking him profusely.
If it doesn't, I hope it will lead to a better understanding of the disease process that leads to diabetes.
That's my (optimistic) opinion.
I've had diabetes for 35 years. Yes, I'd like a cure. But I can wait for another 5 or 10 years. So I'm being patient and seeing what happens.
Hopefully we'll all be pleasantly surprised. And wouldn't that be great.
Drop me an e-mail if you'd like to chat more.
bernard dot farrell (at) gmail.com
Bernard,
You're the best!
GK,
FYI, measuring c-peptide levels gives an indirect measurement of insulin levels in the blood. Measuring insulin levels presents both endogenous and exogenous insulin availability, whereas c-peptide will measure endogenous insulin production.
Hi Bernard,
There is a chance the honey moon vaccine Diamyd is testing out will work for patients who have had diabetes a long time.
Note, that it may work, not saying it will (or even that the vaccine is working at all, since they have to finish phase 3 first)
check: http://diamyd.blogspot.com
The BCG vaccine is a substitute for Freund's Adjuvant (http://en.wikipedia.org/wiki/Freund%27s_adjuvant). Freund's Adjuvant is given regularly to experimental animals as a control or to "reset" their immune systems I believe. It has adverse effects when given to people though the wikipedia article has a link to approved alternatives that have the same effect on humans' immune systems. The trial may hinge upon the exact way the BCG vaccine is administered (the protocol used) since BCG vaccinations have not been shown to prevent the onset of Type I diabetes in children if the epidemiology studies are to be believed. Unfortunately, people are not mice and many protocols and procedures that have cured cancers and other diseases in mice via immune system manipulation have generally failed because the human immune system is different in some significant respect (likely, it's too aggressive). So, there may be a similar way for you to be cured besides BCG if the Faustman trial works for people and mice. You will likely see a cure within the next 10-20 years via either stem cell, gene therapy, or immune system "reboot", so hang in there.
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