The results of a promising clinical trial were released earlier this month during the American Diabetes Association’s annual “Scientific Sessions” conference. Teplizumab, an immunosuppressant, was found to successfully delay the onset of Type 1 diabetes. The details are published in the New England Journal of Medicine.
Teplizumab was given in a single 14-day course to a group of 44 participants that had been identified as at high risk for development of T1D. A placebo group of 32 high risk participants was also monitored. Patients were followed for seven years, during which 72% of the control group was diagnosed with T1D, compared to only 43% of the teplizumab recipients. Moreover, among those that did develop full blown T1D, the drug appeared to delay onset by an entire two years. The trial was a resounding success.
Teplizumab works not only by suppressing the autoimmune activity of T cells, but by somehow activating regulatory T cells, a subpopulation of T cells that modulate the immune system. The regulatory T cells seem to maintain stability even after the teplizumab treatment has ended. This may account for how researchers found that a mere 14-day course continued to improve outcomes even over seven years.
While the trial was fairly small and prompts many questions, it represents a potential landmark in approaches to diabetes. The prevailing wisdom is that T1D cannot be delayed or prevented, and as such the mainstream medical industry pays very little attention to identifying those with a high risk of developing the disease. But if teplizumab (or alternatives) can reliably slow the progression of disease, it is easy to envision a future in which a diagnosis of T1D quickly prompts antibody testing of immediate family members so as to delay or prevent their own progression as much as possible. Provention Bio, the owners of the patent on teplizumab, saw its stock price triple overnight.
Antibody screening should become more popular on the heels of this news. Free T1D antibody screening, which can detect the presence of the antibodies that precipitate Type 1 diabetes, has been available from TrialNet for years. But undoubtedly many have opted against the testing, suspecting that there would be no recourse should they learn that any family members are categorized at high risk.
Inevitably, as an increasing number of people have been identified as high risk for T1D – and as a means to a true cure for T1D has remained elusive – many have focused more keenly on the issue of prevention. Recently we profiled efforts to prevent the onset of Type 1 diabetes with a homemade cocktail of commonly available anti-inflammatory supplements and vitamins. The analysis underpinning the recipe seems to be based on good science, and some may find its anecdotal success encouraging. Advocates of the prevention cocktail use “#WeAreNotWaiting” as a battle cry, in reference to agonizingly long wait for clinical study and federal approval of novel pharmaceutical treatments.
The teplizumab study was conducted by Dr. Kevan Herold, who has Type 1 diabetes himself. Dr. Herold has been working on this approach for a long time: we interviewed him a decade ago on this very subject. At that time, he referred to the upcoming prevention trial as “the most exciting” of his career.
The same drug has been extensively studied as to its effects in those newly diagnosed with Type 1 diabetes, with mixed results. A 2010 study found that teplizumab did not reduce daily insulin use or A1C; but a 2013 study showed that it had some potential to slow the progression of the disease. Not all patients responded, however, and as the response duration was limited (the treatment group eventually “caught up” to the control group), it wasn’t clearly worth the known side effects of the pharmaceutical. This, however, is the first time that teplizumab has been tested among those with a high risk of developing T1D.