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2 Comments
This is a phase 2 trial of the GLP1 agonist lixisenatide, NEJM article [here](https://www.nejm.org/doi/full/10.1056/NEJMoa2312323).
It’s a promising finding, but there remains a way to go.
The study suggests there could be a small effect of the drug in delaying the progression of Parkinson’s symptoms at 12 months. The difference for the primary endpoint was 3 points on a 0 to 132 point scale, which was less than the pre-specified clinically meaningful difference of 4 points used for the sample size calculation.
There were no significant effects on other tests, although the study wasn’t designed to detect these.
There were also prominent GI side effects that led to dose reductions, which are common with this class of drugs, so blinding was unlikely to be too robust, and there might be tolerability or safety issues with longer term use given the age of the recipients (eg, there was weight loss, which is generally not desirable).
Results like this (clinically marginal effect, non-negligible risk for patients) need confirming in a longer-term, larger, phase 3 trial, and as yet there seems to be no follow-up trial fro this drug registered on clinicaltrials.gov. However, at the end of this year there is a [phase 3 trial](https://clinicaltrials.gov/study/NCT04232969?term=exenatide&cond=parkinson&rank=5) of exenatide in Parkinsons, which showed a very similar effect (3 point ‘benefit’) in a [smaller phase 2 trial published 6 years ago](https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(17)31585-4/abstract).
~~From the study~~
~~”Lixisenatide, a glucagon-like peptide-1 receptor agonist used for the treatment of diabetes, has shown neuroprotective properties in a mouse model of Parkinson’s disease.”~~
~~It seems it wasn’t done on humans.~~