Saturday, March 27, 2021

Possible Cures for Type-1 in the News (March)

This posting is a collection of shorter news items. 

Fenofibrate Extends Cure Of T1D In Single Case Study

I originally reported on Fenofibrate in June 2019:
https://cureresearch4type1diabetes.blogspot.com/2020/06/strong-results-from-single-case-use-of.html
This was a single person case study (so not a clinical study), but the results were very strong.  The person was treated during her honeymoon phase, and has not needed to inject insulin since.

The update is that she has now gone about 31 months without needing injected insulin.  This is up from 21 months at the time of my previous report.  She continues to take one pill a day.  I know of nothing unique about this person or her type 1 diabetes, so there is every hope that this success can be tested and the used on others in the future.

Oral Insulin is Unsuccessful For At-Risk Patients (Pre-POINT-Early Study)

Rational: One of the autoantibodies that is associated with type-1 diabetes targets insulin.  Therefore, there is a theory that giving insulin to people with T1D might prevent or delay the onset of type-1 diabetes by training the body not to produce this autoantibody.  The process is vaguely similar to giving small amounts of peanut proteins to people with peanut allergies.  But I want to stress that T1D is not a classic allergy.  The mechanism for T1D is very different than the mechanism of classic allergies. 

Results: There were no statically significant results in the primary outcome.   "Immune responses to insulin were observed in children who received both insulin and placebo, and the trial did not demonstrate an effect on its primary outcome." (Quote from the abstract, but I've removed the numbers to make the English flow better.)  So from an effectiveness point of view, the study was unsuccessful.

Trial Registry: https://clinicaltrials.gov/ct2/show/NCT02547519

Discussion: Oral Insulin has been tested as a possible cure or prevention for T1D for longer than this blog has existed.  In that time, several different clinical trials have been run.  All have been unsuccessful, like this one.   A few have been unsuccessful, but the researchers did extra analysis after the study was over, and found that maybe if they had tested oral insulin in a different subgroup of patients, then it might have worked.  That, in turn, has kept hope of oral insulin alive, and the cycle repeats.

There are three clinical trials of oral insulin which are currently running.  One is a 26 person study by a private company (Oramed).  Clinical Trial Registry: https://clinicaltrials.gov/ct2/show/NCT04150107

The other two are being done at Technische Universität München, and are called Fr1da and GPPAD-POInT.  Both are phase-II studies, of 220 and 1040 people, respectively.  The study reported above is a "proof of concept" version of the GPPAD-POInT. 

 
I'm often asked "If the proof concept Pre-POInT-Early study was unsuccessful, why then continue with the much larger, much more expensive GPPAD-POInT study?"  A detailed answer to that question would not fit here, but two things to remember are:
  1. There is no requirement of a successful small study in order to run a larger study.  Running a clinical trial requires a safety document (called an IND), money, and interest.  You do not need good results from previous studies, if you already have money.  There is never any regulatory review that previous studies were "successful enough" to start another study.  If the researcher is committed, and has the money, and the safety document, bad results from previous studies don't matter.
  2. Researchers are naturally optimistic, and we want them to be.  Most research fails, but we cannot let that stop research in general, or there would be no new breakthroughs.  So we need optimistic (even overly optimistic) researchers to push forward even in the presence of some bad news.  Of course, we don't want to waste money on research which has a history of failures, either, and so it is a tough judgement call.

More Bad News from a BCG Meta Analysis

Years ago, there was a lot of hope that BCG might cure type-1 diabetes, and this lead to several clinical trials.  All of those were unsuccessful.  The only BCG trial still ongoing is aimed  aimed at lowering A1c numbers (so treatment rather than a cure).  Recently a group of researchers, who were not previously involved in BCG research, published a meta analysis which combined data from four different clinical trials of BCG.  Each trial involved giving BCG to people with established T1D and measuring A1c and C-peptides.  The results did not show statistically significant improvement for people treated with BCG:
So even as a treatment, it appears that BCG is unsuccessful.


Joshua Levy
http://cureresearch4type1diabetes.blogspot.com
publicjoshualevy at gmail dot com
All the views expressed here are those of Joshua Levy, and nothing here is official JDRF or JDCA news, views, policies or opinions. 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.

 

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