Dr. Michael Haller on Clinical Research and Future Treatments for T1D
Dr. Michael Haller is a pediatric endocrinologist at the University of Florida, where he serves as both Division Chief and Professor, as well an investigator on the PROTECT Study. He recently spoke with Beyond Type 1 about his career in endocrinology, the importance of clinical research, and some of the more exciting Type 1 Diabetes Prevention Research currently underway.
Beyond Type 1: Thanks for speaking with us today Dr. Haller. Can you tell us a bit about how you got your start in endocrinology?
Dr. Mike Haller: Sure, thanks for having me. I came to the diabetes space through my grandfather who was diagnosed with Type 1 diabetes as a young man.
I had the great fortune of working with some of the pioneers in Type 1 diabetes growing up in Gainesville, Florida. When I was a teenager, Desmond Schatz, Mark Atkinson, and Janet Silverstein let me come and work with them. I went to Duke for undergrad and solidified my desire to be a doctor and came back here [to Gainesville] for medical school and continued on to a pediatric residency and fellowship. I’ve been here ever since doing mostly clinical and research work with Type 1.
I understand a lot of your work is in research. Do you still see patients as well?
I definitely see patients — I wouldn’t have it any other way. In fact, I’ll often struggle with the division of time between purely clinical care and doing research work, but almost all of my work is what we call translational clinical research. Even when I’m doing research it’s with patients, I spend about 15-20% of my time doing traditional clinical care, seeing patients in my clinics. I spend another 20% of my time doing administrative stuff as the division chief and helping with some departmental programs. The rest of my time is spent on funded research projects, so the clinical trials that are currently funded through NIH (National Institutes of Health) or other philanthropy.
Are there any clinical trials that you’ve worked on in the past that that you’d like to highlight?
I largely got into what I do now because of my experiences working on the diabetes prevention trial, which was really the first effort in the Type 1 prevention space. We were giving oral insulin to kids and adults who had family members with Type 1 who were found to be at risk because of having autoantibodies. Unfortunately, that trial, like many others in the prevention space, was not able to demonstrate an ability to change the natural history of the disease, but it largely was responsible for demonstrating the feasibility of doing prevention studies at all. Because of that, TrialNet was born and funded by the NIH.
Through TrialNet I’ve had the great honor of being involved in pretty much every other immunotherapy trial that’s been funded through that network, since the mid to late 1990s. We’ve researched Rituximab, which has demonstrated the ability to prevent or to preserve beta cell function. We’ve done a trial with low-dose ATG (Anti-Thymocyte Globulin) that demonstrated the ability to preserve beta cell function.
I always tell patients, we’re no longer striking out like we did in the 1980s and 90s in terms of immunotherapies… we’re certainly not hitting home runs either. We’re just starting to get on base. But it’s exciting that we’re in the game now.
Can you talk to me a little bit about the clinical trial process? What are some of the obstacles and some of the things that you’ve seen work well?
So much of it is just about recruiting — explaining to the families why they should be involved and what it means to be involved in a study. That’s really the hardest part. For the longest time we were doing these studies and people were taking drugs or placebos and not seeing any particular benefit of it. Fortunately, now we can point to a number of examples where being in the study has actually been meaningful to the patients who received the drug. And for the larger population of folks living with Type 1, we’re able to give them hope that there’s stuff coming down the pipeline.
My style is just to be as transparent as I possibly can about what’s involved in being in the particular trial, what the risks and benefits are, and what I think the likelihood of a particular therapy being beneficial to the patient might be. I always find it very interesting that I can present the same trial to 2 or 3 different families and get 3 or 4 times that many responses from them about, whether it’s a good idea or not for them.
What can you tell me about Teplizumab?
The Prevention Trial with Teplizumab was run through TrialNet and enrolled folks who had a very high risk for progressing and needing insulin, but who still had no insulin need when they were enrolled. They received either med or placebo, and then we had to follow them.
The challenge with prevention trials is you don’t get a firm endpoint. Once you’ve enrolled everybody, you have to wait until enough patients either develop diabetes or don’t, to have statistical power to say that the therapeutic either worked or did not. That’s why it took almost nine years to get the final result from that trial. Fortunately, in this case, what we saw was the patients who received Teplizumab, experienced about a 2-3 year on average delay in progression to Type 1 diabetes.
The next step is to determine where we can go with the drugs. The company is working to see if they can get it fast-tracked and approved and possibly on the market as soon as the summer. It’ll really be paradigm shifting for the field because then you can start talking about having an FDA-approved immunotherapeutic for treating Type 1. You could argue this is the first meaningfully different therapy for Type 1 diabetes since the discovery of insulin. Everything else is just management really.
To learn more about clinical research into Teplizumab underway, CLICK HERE.
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