The Surgical Approaches Working Group encompasses new and emerging treatments for HD for which the delivery will require a neurosurgical intervention.
- cell replacement therapy
- delivery of neurotrophic factors
- deep brain stimulation (DBS)
- genetic manipulation (e.g. RNA interference).
The surgical approach which has progressed furthest, to date, has been transplantation of developing striatal tissue into the striatum of individuals with HD. This work was predicated on many decades of experimental evidence and has now resulted in a small number of clinical studies. Although a firm conclusion is premature, preliminary data suggest that this approach can provide functional benefit.Description
Clinical studies of neurosurgical interventions tend to be complex, difficult, and require a close relationship between specialists from several disciplines, including neurosurgeons, neurologists, psychologists, psychiatrists, and neuroscientists. Hence, the Surgical Approaches Working Group consults with others (for example, the Motor Phenotype and the Cognitive Phenotype WGs) for the development of outcome measures.Current projects
Some neurosurgical research areas already have a forum for discussion (NECTAR for cell replacement therapy and neurotrophic delivery), whereas others are not yet ready to translate preliminary results to clinical studies (e.g. RNA interference).
Following the kick-off meeting, the Surgical Approaches Working Group decided to focus on deep brain stimulation, as this appeared to be the area which could most benefit from the activities of the working group at the present time. Deep brain stimulation (DBS) is a surgical treatment involving the implantation of a medical device which sends electrical impulses to specific parts of the brain, particularly the globus pallidus.
Based on very promising results seen in individual case studies from
colleagues in Canada, and in collaboration with these colleagues, we
plan to study whether DBS may have a significant role in the management
of HD. Consequently, discussions within the working group have focused
over the last two years on developing a protocol for a clinical pilot
trial of DBS in HD patients with marked uncontrolled chorea. A pilot is
planned for the near future and it is anticipated that a multicentre
trial will follow once sufficient pilot data is available to guide and
power the full study. This study is co-chaired by Prof. Jan Vesper,
University of Freiburg, Germany.