Friday, September 16, 2022

Research Dropped As A Practical Cure

Every year I have the sad job of removing some projects from my list of active research aimed at curing T1D.  In the perfect world, I would remove research when it was unsuccessful: when clinical trials testing it failed or the researchers working on it stopped.  However, in the real world, I often don't know that a clinical trial was unsuccessful because those results are not published.  Researchers sometimes continue working on a project long after it is clear that it has failed, or simply move on to something else without anyone noticing that their previous research has been dropped.

My policy is to stop following research 2 years after the end of the last active clinical trial aimed at curing T1D, even if there is no specific publication of failure.

These are the research projects which I have stopped covering recently:

AAT (Alpha-1 Antitrypsin) by Kamada 

AAT is an anti-inflammatory/immunomodulatory drug, which the body makes naturally, and which is already FDA approved for people who have a rare condition where they don't make enough of it on their own. Using AAT to treat type-1 diabetes is based on the idea that one of AAT's effects (lowering inflammation, immune modulation, or wound healing) can cure/prevent/treat the disease. 

The last results from an AAT study were two unsuccessful results published in 2018, and there are no ongoing studies now.  During the 2010s, there were a total of 7 studies done on AAT, and not one of them was a success.

The company producing the drug (Kamada) no longer even lists T1D as a "Therapeutic Focus Area" nor is it listed in their research pipeline.

Microvesicles (MVs) and Exosomes by Nassar at Sahel Teaching Hospital

The idea behind this research is that umbilical cord-blood derived stem cells microvesicles may reduce inflammation and hence improve natural insulin production (more β-cell mass) in people with T1D.

This referred to a Phase-I clinical trial started in 2014, which was expected to finish later in 2014.  As of 2020 a summary article reported no results had been published.  I also did a literature search and was not able to find any suggestion that results had been published, or that follow on research had been started.


Alefacept / Amevive® by TrialNet

Alefacept is a drug that has been used to treat the skin condition, psoriasis. Psoriasis is generally considered to be an autoimmune disease, similar to type-1 diabetes, but with the body attacking its own skin cells, rather than its own beta cells. So trying a drug already approved for Psoriasis on type-1 diabetes seemed like a reasonable thing to do, and these researchers did it.  Back in 2016 I reported on the results of the phase-2 trials, which were OK, but not great:

Unfortunately, in a certain sense, none of this matters.  The company that made Alefacept stopped production in 2011, so it has not been available for years.  I don't know of any planned clinical trials with Alefacept, nor any similar fusion proteins. 

Joshua Levy
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.