Lady GABA - The Final Chapter

Published 01/16/2023

Previously, we looked at how PD manifests, dopaminergic treatments, and a little on promising previous studies.

Finally, we're peeking under the hood for the final promising treatment: Gene therapy.

Following along a similar idea of inhibiting neuronal firing, it appears that gene therapy that consists of insertion of the glutamic acid decarboxylase gene (GAD) into the subthalamic nucleus, could prove an alternative. GAD is the rate-limiting enzyme for GABA production; the activity of both GABA efferents to the subthalamic nucleus and its targets within the basal ganglia are both affected in PD. A muscimol infusion (a GABAergic agonist) during deep brain stimulation surgery into the subthalamic nucleus of patients affected with PD, resulted in suppression of neuronal firing rates, in turn resulting in a temporary improvement in PD symptoms. In fact, AAV2-GAD treatment led to an improvement of 8·1 (SE 1·7, 23·1%; p<0·0001) in off-medication UPDRS motor score at the study endpoint of 6 months, compared with 4·7 (1·5, 12·7%; p=0·003) with sham treatment.

(17), These benefits also persist after 12 months. (18)

So, with such a promising array of treatments on the horizon, they certainly warrant some serious Applause(3)

References

1- Le-Witt 2011

2- Niethammer-2017

3- Lady Gaga "Applause"- Stefani Germanotta, Paul "DJ White Shadow" Blair, Dino Zisis, Nick Monson, Martin Bresso, Nicolas Mercier, Julien Arias, William Grigahcine , released 2013, interscope records.