At last, a gene therapy for Parkinson's disease?

摘要

Surgical interventions to treat Parkinson's disease, specifically thalamotomy and pallidotomy, pre-date levodopa therapy. They were largely discontinuec after the introduction of levodopa, but reintroducec 20 years ago as adjunctive therapy for later stages o1 the disease. In 1998, deep brain stimulation (DBS) o1 the subthalamic nucleus was shown to be an effective alternative approach to pallidotomy. Since then severa new surgical approaches have been proposed, although only two have been tested in randomised double-blinc clinical trials: intraputamenal infusion of glial-derivec neurotrophic factor (GDNF) and intraputamena gene therapy with neurturin (a structural relative of GDNF). However, these surgical approaches have not shown the same level of efficacy in randomisec controlled trials as in the preceding open-label studies In the case of intraputamenal GDNF, explanations foi this failure included the possibility of a large placebc effect and differences in methodology and statistica underpowering (type 2 error)