If you have lived in the world of diabetes for any length of time, you have heard a million stories about diabetes being cured in mice. We have read about encapsulate cells and stories of islet cell transplants. Most recently we have been listening to the stories of bionic and artificial pancreases.
This summer I was asked if I would be interested in hearing from a woman who was involved in a human clinical trial aimed at preserving beta cell function. A person who was involved in working towards a real organic cure? I was very interested. This is Mary’s story….
I was seated in a conference room at the American Diabetes Association Scientific Meeting in San Francisco watching a presentation of type 1 diabetes (T1D) clinical study. Projected on the screen was a chart with dots and trend lines representing the functioning of each participant’s insulin-producing beta cells over the span of two years. As I squinted to see the detail, it suddenly struck me: one of those dots was me. I was one of those “participants.” In fact, I was Patient Number 1 in the study.
Rewind to March 2011. I had just been diagnosed with T1D at the age of 35. It was a shock for me, as it is for most people, to be diagnosed with a serious chronic illness especially after thankfully not having any medical issues up to that point. After absorbing the shock and starting to understand all the ways my life was about to change, I did what any 30-something living in San Francisco would do…I turned to Google.
Online I found a tremendous amount of information about the disease. “Knowledge is power” tends to be my mantra. But all the knowledge I gained through my searches seemed to lead to more questions, and I found myself looking for answers to what felt like the most pressing question – now that my immune system is attacking my pancreas, how do I make it stop? Nothing I was reading could answer this question. So, I shifted my focus to what researchers are currently trying to learn about T1D. I found a host of studies on sites like clincialtrials.gov and trialnet.org, and many of these studies were in fact seeking to answer the question that was most important to me – how do we stop the autoimmune attack on the insulin producing cells?
One of the studies that interested me the most was taking place right in my backyard. Researchers at the University of California at San Francisco (UCSF) were studying ways to use a patient’s own regulatory T cells (Tregs) to change the activity of the immune system. The study was so new that it had just wrapped up testing in mice. It was just now ready for a Phase I clinical trial in humans, and they were looking for participants! Without much hesitation, I dialed the study phone number. It turned out the study was even newer than I realized; in fact they hadn’t yet enrolled any participants – which explains why they seemed so happy to hear from me! And with this phone call my adventure as a research participant began.
It started with a basic phone screen with the study coordinator, and once it was determined that I met the basic criteria, I met the researchers to learn more about the study. The researchers drew diagrams on the board, talked about the theories behind the Treg study, and expressed a lot of enthusiasm for what they hoped to learn from this Phase I trial. There was also a lot of discussion around the practical elements of the study: the time commitment, the potential side effects, the upcoming process and the details of the procedures. I made the decision to enroll in the study. I was a bit nervous, but onboard.
Once I was enrolled, the real fun began; notably, the many, many blood draws. I found myself thinking, “Seriously – are you going to fill all of those tubes again?” The mixed meal tolerance tests (MMTs), which required me to drink a meal supplement while my blood was drawn every 30 minutes, were the most draining (pun intended) and lasted for up to four hours! And the worst part, the MMTs started first thing in the morning and required fasting, which meant no coffee! I must have complained about the lack of coffee a lot, because the nurses eventually started presenting my meal supplement in a Starbucks-like coffee cup in an effort to make me feel better – or to stop my complaining!
In between these draws, I was told to go home and ‘stay healthy’ (and take iron supplements!). That meant eating right, exercising, managing the stress of work and the stress that comes with adjusting to a new high maintenance chronic illness. Life went on and didn’t slow down for diabetes.
After a few weeks I was scheduled for what turned out to the biggest blood draw yet – enough to extract the Treg cells that would be expanded in the lab and infused 14 days later. It felt like a long 14 days, and the waiting was in some ways the hardest part. It was the anticipation of what was going to happen on the infusion day and wondering if this was really going to work and what might happen if it didn’t.
The day of the infusion arrived. The transportation of the Treg cells across town from the lab to the hospital was a highly choreographed affair, with the timing of the transfer scheduled down to the minute. Since this was the first time this was being done, there was a lot of anticipation on everyone’s part, and my cells arrived with an entourage! Soon there were eight people in my room, all members of the research team there to watch the infusion happen. While the nurses prepped me, we waited for the exact moment when the infusion was scheduled to happen. In my nervousness, I looked at everyone and said, “I still have the right to withdraw, right?” Let’s just say that my audience didn’t find that very funny. After the infusion, my vital signs were monitored every 30 minutes and overnight every hour, looking out for any potential side effects. But other than a metallic taste in my mouth thanks to the saline drip, nothing happened. “Is that it?” And I went home.
Guess what? MORE blood draws after the infusion. They started right away, multiple times a week for the first month, then (mercifully) every few months for the next two years. And in all this time, I didn’t really know how it was going, at least not officially. What I did know is that my “honeymoon” phase seemed to be continuing. My insulin needs were still quite low because my body seemed to be producing a fair amount of its own insulin. During this time I was also learning more and more about how to control my diabetes, and as part of the study I had ongoing access to diabetes educators who reviewed my paper and electronic logs (I started wearing an insulin pump and using a continuous glucose monitor) every two weeks. That might sound a bit much, but actually it was like having my own personal diabetes coach and it ended up really helping me manage my diabetes as well as possible.
Friends and family have often asked me what it was like to participate in a trial. I usually keep it short and say something like, “Oh, it was interesting.” Actually, it’s more than just interesting. It feels like a good thing to do. It feels empowering, and it also feels necessary. Science simply can’t advance without participants. Through this process I’ve become very appreciative all of the people behind the science – others with T1D who have stepped up and participated in research before me and the researchers themselves who have the vision, patience and tenacity to develop an idea and see it all the way through to fruition. So the next time you are reading the latest issue of Diatribe summarizing findings from the latest studies, take a minute to think of all of the people behind the data. They are average, everyday people – real people – helping to make day-to-day life with diabetes easier and bringing us one step closer to finding a cure.