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.
I see this news is being reported in severalplaces. It's interesting because there's been so much talk about using stem cells to treat diabetes. Now Novocell says that they were able to use human stem cells to control diabetes in mice.
This approach is not yet perfect because some of the mice developed tumors. I'm guessing that we won't see anything come out of these labs for several years.
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!
Researchers have been able to get liver and pancreatic cells in diabetic mice to produce insulin by using a naturally occurring protein. According to the article I read, by injecting a protein called Pdx1 into the abdomens of mice, insulin production is restarted in the mice. Pdx1 has a structure that allows it to pass into the pancreatic cells, enter their nucleus and cause insulin production to start.
According to Dr. Li-Jun Yang, founder of Transgeneron Therapeutics, "What is remarkable is that the protein also promotes regeneration of insulin-producing cells in the pancreas, allowing the diabetic mice to become normal."
It all sounds like it has possibility. I'd just caution readers not to get too excited.
This is research and I'd guess it's many years away from any kind of application for people with type 1 diabetes.
I stumbled across this interesting article that outlines what it might take to actually turn a stem cell into a beta cell. Beta cells are the pancreatic cells that produce insulin.
The author, Paul Myers, is an associate professor of biology at the University of Minnesota, so I'd hope he's fairly accurate in his description of the process.
Let me summarize the article by saying, making beta cells from stem cells isn't going to be easy. Reading the steps involved reminds me of the song Dry Bones, 'With the leg bone connected to the knee bone, and the knee bone connected to the thigh bone...'.
To make a beta cell, you have to first convert ES cells into mesendoderm, then into endoderm, then into Anterior Definitive Endoderm (ADE), then into midgut endoderm, then into general pancreatic tissue, then into pancreatic endocrine cells, and finally, you can apply a signal to switch them into the beta cell state.
And even if this could all be done, you'd still need to deal with the
chronic difficulty of removing whatever destroyed the patient's original set of beta cells.
So I won't be holding my breath on this line of research. I think it'll bring benefits, but probably not anytime soon. My money is still on the research being done by Dr. Denise Faustman.
If you've read this blog for a while you may know that I've done some fundraising to support the research by Dr. Denise Faustman into a possible cure for Type 1 diabetes.
I'm subscribed to the Yahoo! newsgroup about this work and today they posted a pointer to a brief USA Daily article with a small amount of news.
This is slow work and Dr. Faustman is conducting it in a careful and rigorous way. Which makes some of us impatient for results. But I'm eternally optimistic about it all.
In early January I'll be at the lab to get some blood drawn that will be used in the research. A good friend from work will also be there as a healthy volunteer! What a guy. While I'm there I hope to meet with Dr. Faustman and ask her some questions about the work. I'll post what I learn online (mid-January).
Updates: I did meeting with Dr. Faustman in January. In March there was an announcement about the start of clinical trials for a possible cure for type 1 diabetes, based on her work.
Today's Wall Street Journal has a front page article on Pfizer's decision to drop Exubera. It covers some of the history of Exubera development and also points out some of it's shortcomings
The Exubera device, which some compared unflatteringly to a bong for smoking marijuana, could also be embarrassing to use in public.
"I can teach someone how to use an insulin pen in five minutes, but it would take nearly an hour to teach a patient to use inhaled insulin," says Anastassios Pittas, an endocrinologist at Tufts-New England Medical Center.
The article also has a pointer to a (PDF) transcript of the Pfizer conference call about its 3rd quarter earnings. The Exubera part starts on page 4 of the transcript. I'm glad to see Jeff Kindler, the Pfizer CEO say:
We remain committed to finding and developing new treatments for diabetes, which is an area of enormous unmet medical need. Our R&D portfolio in this field spans the width of the diabetes disease continuum, from genetic susceptibility at birth to the onset of devastating complications. This is also an important focus for business development. In addition, we will continue to closely monitor developments in medical practice and technology as they relate to inhalation therapies and other innovative delivery systems both for insulin and for other medicines.
Despite the problems with Exubera it was a novel treatment for diabetes that might have really helped people who fail to treat their diabetes because of their fear of injections.
And while most of us hope for and work towards a cure for diabetes, it would be nice to have new treatment options while we're waiting.
Diabetes365 day 5 Oct. 12th 2007 - Diabetes Detritus
Diabetes is a high maintenance disease - it takes a lot to replace a working pancreas. For today's picture I took most of the stuff out that I either carry with me, or have as backup in my office.
As you can see, there's a lot of stuff there.
When I was a child I was into all sorts of gadgets. Now I get to live with these on a daily basis.
The standard cry when we're leaving the house to go anywhere is "do you have your kit" or "Dad can't find his kit". And everyone knows it's the Eagle Creek black bag that has all my stuff. We don't leave home without it.
All of this technology is needed to replace my non-working pancreas. I'm thrilled that it exists and that I have the health insurance coverage to afford it. But I'd trade it in for a real pancreas any day of the week.
Unfortunately currently there isn't a cure for diabetes. But I do my best to support the research for a cure.
For now, I'm trying to take diabetes365 photos at slightly later times in my diabetes day. At some stage, I'll switch to a different approach to documenting what it's like to live with diabetes 24 by 7 by 365.
I have a small scales at home that I use mostly for breakfast. Most packaged food now has nutrition labels which lets me see the carbohydrate content for a specific weight. By adding the carbs for all the foods together and using an insulin to carbohydrate ratio (I:C) I can figure out how much insulin I need to take. The I:C value is different for everyone with diabetes, it can even vary for the same person at different times of the day.
I've been using Symlin for about a year. It's a drug that's I inject shortly before meals. It slows down the release of food from my stomach and makes me feel full. Both contribute to better blood sugar control, though it can cause terrible low blood sugar reactions.
Insulin (and Symlin) are not cures for diabetes. They just allow people with diabetes to control their blood sugars.
I know this story has been on the wires for the last few days, how a company called SemBiosys has been able to produce (human recombinant) insulin from safflowers.
Then I see tonight that even Business Week is excited about the development. Look at what it might do to their stock price.
Think about the increased demand for insulin, because if you believe the numbers quoted by SemBiosys that demand will more than double in the next five years.
I think what everyone in marketplace may be overlooking is that Insulinisnotacure.
I think that as long as the 'market' continues to get excited about these developments, it's not thinking about how to get rid of this terrible disease.
I don't want more insulin, or cheaper insulin, or purer insulin. I don't want insulin.
Today is D-Blog day, an opportunity for the diabetic bloggers to try and get the word out about diabetes and trying to live with it (or perhaps to live despite it).
If you've heard about diabetes, you've certainly heard about insulin. That wonderful hormone that's produced by your pancreas and that does an excellent job of regulating your blood sugar - that is if you don't have diabetes.
If you do have Type 1 diabetes, then your pancreas is dead, it's a stiff, it has passed on! OK, I'm going overboard a bit but your pancreas doesn't produce insulin anymore.
No problem, you say, you can just get it and inject it. If only it was that simple.
Insulin is not a cure.
In the 1920's Banting and Best first 'discovered' insulin. Shortly afterwards companies starting making insulin using chopped up cow and pig pancreases. Sounds kind of gross, huh?
Guess what... Things haven't really changed all that much since then.
Insulin manufacturing is a bit better. And insulin delivery devices are much better. We've gone from testing urine to testing blood to determine how much insulin we need. Neither of these options was available in the 1920's.
But for the most part, not a lot has changed since then.
For most people going on insulin is really a long-term sentence. Sentenced to a life of:
Daily blood sugar testing.
Daily worrying about long-term complications.
Daily calculation of the amount of food you're eating.
Daily concerns about exercise, sickness, emotional swings.
Despite all of this, most folks that I know who have diabetes are remarkably upbeat, and have accomplished all the 'normal' things that others do with their lives.
My question is: why does it have to be so hard?
This is not a trivial disease. According to estimates at least 1,000,000 people in America alone have Type 1 diabetes.
If each of these are testing their blood sugar 4 times daily, then this amounts to 4 million test strips a day at an approximate cost of $2 million every day, or three quarters of a Billion dollars every year spent in America just to test our blood sugars.
Now add the cost of syringes, insulin, alcohol swabs, pump supplies, glucagon, ambulance rides, eye treatment, kidney treatment, amputations, etc., etc.
Have I got your attention yet?
If we got serious about working on a real cure for diabetes, not an improved form of treatment, imagine the cost savings. Imagine what all those wonderful people would do when released from the burden of caring for their diabetes.
What are we waiting for?
So here's my call to action.
Why not write to your legislator and ask them if they support increased National Institutes for Health spending on research towards a diabetes cure? That might get the ball rolling.
Diabetes: technology, devices, software, and other stuff.
About Me
Name: Bernard Farrell
Location: Massachusetts, United States
I was born in Ireland and now live in the US.
I have had Type 1 diabetes for over 35 years. I struggle with my blood sugar, the same as most people with diabetes.
I wear a Cozmo 1800 insulin pump and a Dexcom SEVEN CGM to track my blood glucose levels. I also take Symlin to help control my post-meal blood sugars.
I'm blessed by God, and every day brings the possibility of a cure.
