Prerequisites to launch neuroprotective trials in Parkinson's disease: an industry perspective.

摘要: Realizing that 60% to 80% of dopa- minergic nigrostriatal neurons are nonfunctional at the time clinical diagnosis, there is an emerging consen- sus disease-modifying treatments should be initi- ated in earliest stages Parkinson's disease (PD). To date, trial designs and metrics PD have been focused on motor symptoms as core feature disease. identify earlier or ''pre-motor'' populations PD, new markers proposed. We address prerequisites needed use these pre-motor trials for selection subjects, definition populations, monitoring dis- ease progression. This may require development diagnostic criteria potentially based non-motor signs, imaging techniques, biological features, all requiring discussion a regulatory framework. Ques- tions addressed include: Which steps must taken gain broad consensus field from academic opin- ion leaders, patient advocacy groups, bodies, industry? How do we prevent sub- which not representative full spectrum? Is way forward personalized medicine? balance risk benefit at- population? While many tools available, con- certed effort required develop integrated data sets, well achieve necessary standardization multicenter trials. this end, public-private sortia (including centers, industry) will crucial importance prospectively investigate define best treatment paradigms. V C 2012 Movement Disorder Society