Frank Martin, PhD, director of research at JDRF, outlines some of the past and current efforts of the organization to support type 1 diabetes prevention therapies.
JDRF researchers and clinicians have all worked to identify different drugs or pathways that are involved in type 1 diabetes (T1D) progression, said Frank Martin, PhD, director of research at JDRF.
Can you discuss the highlights of some of the prevention efforts that JDRF has been involved in, from where the research began to the efforts the organization is funding today?
What JDRF and clinicians, researchers in the field have done is through research, identified different drugs or pathways that we think are really involved in T1D progression. Once that preclinical data, that laboratory-based data, is good enough the clinical testing starts typically in people who are recently diagnosed with the disease. Then we get an insight into how safe the drug is, how effective the drug is at protecting those beta cells in people with T1D. Once that safety has been determined, then we'll start moving that drug into earlier and earlier stages of T1D, when people might not be symptomatic.
Right now, there's at least 10—that I can think off the top my head—trials going on in the recent onset population for drugs that might improve the survival of the beta cells themselves, or drugs that might correct the problem with the immune system that’s leading to autoimmunity. One great example of success there is probably teplizumab. Teplizumab is right now awaiting feedback from the FDA on a potential approval as a preventative for T1D. JDRF has been part of that process almost the whole way, where we funded the basic research, we helped fund some of the early clinical trials. Now Prevention Bio is trying to take that drug into and towards approval.
Some other great therapies with big potential look more at re-educating your immune system. Novo Nordisk just started off a trial called TOPPLE T1D, which will look to re-educate people's immune system and, in that way, stop autoimmunity. Those are just 2 examples of research that we've sort of been supporting the whole way. There are other trials like Ver-A-T1D, which uses verapamil, a commonly used migraine drug, to improve the survival of beta cells. That just kicked off a large clinical study in Europe through a consortium called Innodia. So, we're looking forward to a lot of great data in the years to come and hopefully we'll see successes from these multiple clinical trials that will move them closer to the hands of people living with T1D right now and move them back into the hands of people who are at risk for the disease, as will be done with TOPPLE T1D, is being done with hydroxychloroquine through TrialNet, and we hope will be approved for teplizumab with Prevention Bio.