Frank Martin, PhD, director of research at JDRF, shares feedback from patients on JDRF's T1Detect screening program.
The 2020 study by Zeigler et al mentions the screening program in the study took place before there was a treatment available, but the study did mention teplizumab might soon be available to delay T1D onset. Can you discuss these results and what this could mean for patients who have phase 1 T1D?
The screening programs to date have really run on the notion that reducing the rates of diabetic ketoacidosis (DKA) at diagnosis has a very profound public health benefit. They've also built in the notion that, well, when there is a therapy approved to prevent T1D, this entirely changes the cost effectiveness studies and the variables there. But the biggest problem is we don't know who to give those drugs to. You have to be able to identify the people who are going to be able to best benefit from those drugs.
What we've seen over the last 20 years or so is that clinical trials in this at-risk population, the stage 1 and stage 2, population, run very slowly, because we've not yet established a system where we're identifying those people who can participate in those trials, outside of something like Fr1da, outside of something like TrialNet, outside of something like ASK.
This was a lot of the impetus for JDRF to launch our screening program. We want to identify those people who are going to best benefit from a drug when it's approved. But right now there's no therapy that will prevent the disease. In fact, there's no therapy that will delay the honeymoon period either.
JDRF is very, very hopeful that any disease modifying therapy will be approved in the future for people with T1D. We want there to be a day when people—the endocrinologist, the primary care physicians, the patients—have a toolbox of choices at hand, which they can use to help their T1D independent of what stage they're at, whether they're stage 1 with autoantibodies and zero symptoms, whether they're stage 2, and they have autoantibodies, but symptoms only a doctor could see, or they have overt T1D, stage 3, where they're symptomatic, and they're on insulin. All those stages need therapies.
What kind of feedback are you getting from the T1D community about T1Detect?
[Feedback] more generally, for the T1D community is people living with T1D or people who have family members that are at risk for T1D, desperately want therapies that can help either slow, halt, or reverse their T1D. They desperately want cures. They've been calling for this for years and JDRF funds our research as dictated by this. We're trying to fund research that will develop curative therapies.
Now as far as people's responses to T1Detect, something that surprised me, was people are clamoring for more information. They want to be educated more about T1D. They want to be educated more about what to do if they're in this at-risk category. They're asking to learn more about clinical trials.
This is a great opportunity for JDRF to work with partner organizations like TrialNet, who has an established monitoring program called the Pathway to Prevention, where they monitor people in the at-risk category. Then we'll enroll them in clinical trials as they become available. This is just one example of what people can do. We’re making sure through T1Detect that this sort of information is flowing out to the people who are participating.
You can imagine that the first series of education materials we developed were good. And then we started getting feedback from people participating. So now we have to modify those. We actually have to bring them up a level and add more scientific and medical information there, because people are asking for it. We are, I think, really, really proud of what we've done so far. It's a huge, huge, huge amount of work and it's something that we know we need to do in collaboration with the other experts in the field, the ones at TrialNet, the ones in the ASK program. We're trying to make sure everyone's working together on this same good cause.
There's more than one way to skin a cat, right? You can get to screened in a couple different ways. Ours is just one. But I think it's great. It'll be good for the community to come together on how do we best educate the general population about a disease and about our risk for the disease? And how do we best educate the payers about that disease, or the regulatory or government bodies that are going to decide whether or not to include that risk screening as standard preventative services?
These are all things that we're doing right now, with the eventual goal of inclusion of T1D risk screening into standard preventative services, both here in the United States and worldwide.