In previous work we did together with Dr. Jianrong Tang at our institute, we learned that deep brain stimulation (DBS) could reverse certain neurological deficits (hippocampal learning and memory, plasticity and neurogenesis) in a mouse model of Rett syndrome. The recent discovery that DBS corrects the abnormal circuit defect of specific hippocampal neurons gives us more confidence about the positive effects of DBS in the Rett mouse model.
There are, however, several important research steps that we must do before we can contemplate testing DBS in patients with Rett. Firstly, we must establish the duration of the benefit from a two-week DBS intervention. Secondly, we need to determine the ideal frequency of DBS. Thirdly, we must establish the long-term benefits and safety of such an intervention when used over 12 months or longer.
Historically DBS has been used successfully for movement disorders such as Parkinson’s, essential tremor and dystonia. Now that we know the brain of a Rett syndrome model is receptive to neuromodulation, we wish to explore DBS for motor dysfunction which is such a core symptom of the disorder. Our hope is that through DBS we can target both cognitive and motor deficits.
I know that research progress is never fast enough for families and for those struggling with Rett Syndrome. I am encouraged by the results thus far with DBS and will address the steps outlined above with the goal of moving to the clinic as quickly as possible.