I previously blogged about this trial when it started:
This included 32 people. The treatment was a Verapamil pill once a day. The study was done on honeymooners and was double blind. The primary end point was C-peptides, which are key for a cure, as they measure the body's ability to create insulin. You can see the results below:
|Graph is from the published paper, and is presented|
for educational purposes only.
Having the C-peptide numbers go up noticeably in the first three months is a good outcome, but it is not -- by itself -- a cure or a prevention. The treated people were still doing much better than the untreated group after a year, but again, that's not a cure by itself. So the real question is how to move these good results forward to a cure, and I think there are four ways to think about that question:
One big question is, why did C-peptide production (and thus insulin production) improve so much for the first three months, but then stop improving? Remember, that the drug was given for 12 months, but the improvement was seen only in the first 3 months. Obviously, if we could make the Verapamil continue to help insulin production for longer, it would be much closer to a cure.
Another question is, what would happen if Verapamil was given to people who had two autoantibodies, but were not showing any symptoms of type-1 diabetes? Would Verapamil result in a delay of onset, or could it prevent type-1 completely? Current research shows that essentially everyone who has two autoantibodies will eventually show symptoms of type-1 diabetes, but this study shows that for the length of time of the study, C-peptide generation was basically the same at the end of the trial as at the beginning. If people without symptoms were treated, would they continue to be symptom free as long as they were treated? That would be a preventative (and possibly the first step to a cure).
A third option would be to try to improve the treatment, either by combining Verapamil with another drug which helps preserve beta cell function, or by finding a better dosing regimen, with results in a better outcome.
Finally, this study was done on recently diagnosed adults, and the researchers mention that running a similar study on recently diagnosed children in the honeymoon phase be worthwhile.
Paper: https://www.nature.com/articles/s41591-018-0089-4 (behind a paywall)
Trial Registry: https://clinicaltrials.gov/ct2/show/NCT02372253
I'd like to thank the authors for sending me a copy of the paper, which is otherwise behind a pay wall.
publicjoshualevy at gmail dot com
All the views expressed here are those of Joshua Levy, and nothing here is official JDRF, JDCA, or Bigfoot Biomedical news, views, policies or opinions. In my day job, I work in software for Bigfoot Biomedical. My daughter has type-1 diabetes and participates in clinical trials, which might be discussed here. My blog contains a more complete non-conflict of interest statement. Thanks to everyone who helps with the blog.