Wednesday, May 1, 2019

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

This blog posting contains two different new items, with a short note on each:

The One That Got Away

The first article covers some work in Multiple Sclerosis (another autoimmune disease):
The article reports on a promising phase-II study on an incurable disease.

The reason I'm discussing it here, is because this researcher was involved in very similar clinical trials on people with type-1 diabetes, many years ago.  Those trials were the most successful ever done.  People did not use injected insulin for years.  You can read my previous blogging here:
or more generally here:

But years ago, this researcher switched from Type-1 Diabetes to Multiple Sclerosis.  Why?  Because of safety trade offs.  One researcher told me that the T1D version of this treatment would likely have an expected death rate of 1%.  In actual studies (on people!) the death rate was about 1 in 65.  For a disease like Type-1, that is not acceptable.  But for a disease like Multiple Sclerosis (which can lead to death after 10-30 years, and which often has a much bigger quality-of-life impact), maybe it is acceptable.

So, while I'm sad that the type-1 world will not benefit from this research (at least not in the short term), I am happy that our disease is not so dire that a treatment like this is viable.

Phase-II T-Rex Trial Reports Unsuccessful Primary Results

On a completely different note, the T-Rex Trial reported its primary end point.  For background,
you can read my previous blogging about this research here:

A quick summary of this treatment is as follows: remove one specific type of T regulator cell (called "CD4(+)CD25(+)CD127(lo)") from a person with type-1 diabetes.  Grow them out so you have about 500 times more, and then put them back in the same person.  Since regulatory T cells naturally regulate the body's immune system, and the patient now has more of them, the hope is that they will prevent the autoimmune attack which causes type-1 diabetes.

In February, the T-Rex trial reported its primary end point results at 1 year, and they were unsuccessful.  The exact quote was:
no improvement in the primary endpoint of preservation of C-peptide levels vs. placebo at 1 year was observed at the group level (using the standard mixed meal tolerance test)
Of course, the company is hopeful that they can find some good news by reanalyzing the data, looking at subgroups, etc.  but I'm very dubious about this.  In the type-1 diabetes research that I follow, I have yet to see this kind of reanalysis yield a success after the primary analysis was unsuccessful.  The trial is scheduled to collect data for two years, so there should be another update in about a year.

This information all comes from a corporate press release.  There was no mention of a research paper.

Press Release:
News coverage:

Joshua Levy 
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.



Dear Josh, could you please cover the story about Stano lab's clinical trial for Gaba? BR

Oscar said...

Any cure for type 1 diabetes operating through some significant intervention in the immune system is bound to be risky, given how essential the normal functioning of the immune system is to health, and how any tinkering with it can heighten cancer risks. But that said, I wonder at the reasoning that the risks and harms of multiple sclerosis are sufficiently worse than those of type 1 diabetes to make the significant risks in treating the former but not the latter worthwhile. Type 1 diabetics face a high risk of death from hypoglycemia, deteriorate rapidly in terms of neurological and vascular health, suffer a significantly diminished life expectancy, and have a much reduced quality of life, while some multiple sclerosis patients can have a comparatively mild course of disease. I would rate both diseases about equal in terms of their misery and thus of their justification for drastic but potentially curative medical interventions.