gms | German Medical Science

Objective: At present, there is no effective treatment or cure for Huntington’s disease (HD) patients. Therefore, neural stem cell transplantation seemed to offer a potential treatment for HD patients that may slowdown this devastating illness. However there remain major concerns in transplantation. Therefore our group looked for alternatives, utilizing Deep Brain stimulation (DBS), based on the long-lasting successful treatment of other neurodegenerative movement disorders like Parkinson’s disease (PD). Questions remained concerning the optimal target.

Methods: This phase I clinical trial is based on the hypothesis that deep brain stimulation of the internal pallidum can reduce choreatic symptoms in HD patients. In addition, this trial should demonstrate which target point within the pallidum can be used effectively for specific features of HD in order to further refine this promising strategy for a phase II multicenter trial approach. Four consecutive cases with DBS of the Pallidum (GPi/GPe region) are reported. Electrodes were stereotactically implanted under general anesthesia, followed by the implantation of a neurostimulation system.

Results: No complications occurred. The coordinates for the active contacts in the GPi/GPe range were adapted to individual anatomical changes. Under DBS of the pallidum choreatic movements could be reduced by 50 to 80% (UHDRS). The quality of life, which was measured by ADL, was significantly improved in the three patients. Since the effects are delayed, the adjustment and testing of the remaining contacts took place in the course of 6 months postoperatively. The most effective active contacts were in projection of the border of GPi and GPe.

Conclusions: Systematic positive influence of DBS in Huntington’s disease patients is reported for the first time. In the context of the following study it will have to be clarified whether the internal or the external part or other targets are suitable for DBS and which long-term results can be obtained.