This clinical trial tested whether long-term daily treatment with Remygen®, an oral drug developed by Diamyd Medical, could help restore insulin production in adults with longstanding type 1 diabetes (T1D). The active component of Remygen® is GABA (gamma-aminobutyric acid), a compound that in earlier experimental studies appeared to support the regeneration of insulin-producing beta cells, improve insulin release, and reduce inflammation.
What Was the Clinical Trial Testing?
The trial enrolled 35 adult men with T1D for at least five years and divided them into three treatment groups. One group received a lower daily dose of GABA, a second received a higher dose, and a third group received the higher GABA dose plus a short-term course of alprazolam (an anti-anxiety, benzodiazepine drug). The treatment lasted six months.
Researchers monitored insulin production using fasting and post-meal C-peptide levels, tracked blood glucose control, and recorded any side effects or adverse events. The goal was to assess both the safety of long-term GABA use and whether it had any regenerative effect on the pancreas.
What Were the Results of the Clinical Trial?
The trial found that Remygen® did not restore insulin production or improve any markers of beta-cell function.
C-peptide levels, which reflect the body’s natural insulin production, remained unchanged in all treatment groups throughout the six-month period. This included individuals who had some detectable C-peptide at baseline, as well as those with undetectable levels. No meaningful changes were observed for insulin production either.
Measures of blood sugar control—including continuous glucose monitoring data and HbA1c—also remained stable, with no significant improvements seen in any group.
Additionally, the trial found no change in the body’s hormonal response to low blood sugar. This was in contrast to some earlier short-term studies that had hinted at possible effects of GABA in this area.
In terms of safety, the treatment was generally well tolerated, but side effects were common. One participant had a serious liver reaction, likely related to the drug, though liver function returned to normal after stopping the medication.
Discussion
My memory is that, at its height, there were 4 or so GABA related clinical trials running. But this was the last GABA clinical trial that I knew of, so I think this line of research is dead for now.
Diamyd has also been developing a DNA-based immunotherapy (also called Diamyd®) aimed at slowing or stopping the immune system’s attack on beta cells, a different strategy from GABA. That program remains in clinical development.
Clinical Trial Registry: https://www.clinicaltrials.gov/study/NCT03635437
EU Clinical Trial Registry: 2018-001115-73
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 BreakthroughT1D or JDCA news, views, policies or opinions. I sometimes use generative AI ("chatbots") to generate draft blogs, parts of blogs, or drafter alternate wordings for these blogs. I always review every part of every published blog to ensure that it is saying what I want, in the tone that I want, truthfully, and accurately. My kid has type-1 diabetes and has participated in clinical trials, which might be discussed here. I am obese and right on the boarder of T2D and therefore may be taking drugs for those conditions. My blog contains a more complete non-conflict of interest statement. Thanks to everyone who helps with the blog!
