Identifying Effective Treatments for Dystonia in Patients With Cerebral Palsy: A Precision Therapeutics Approach.

Abstract

Recent focus on improving the recognition of dystonia in cerebral palsy (DCP) has highlighted the need for more effective treatments. Evidence supports improved functional outcomes with early interventions for patients with cerebral palsy, but it is not known which interventions are most effective for DCP. Current pharmacologic recommendations for DCP are based largely on anecdotal evidence, with medications demonstrating minimal to moderate improvements in dystonia and variable efficacy between patients. Patients, families, and clinicians have identified the need for new and improved treatments in DCP, naming this as the top research theme in a recent Neurology® publication. Precision therapeutics focuses on providing early effective interventions that are individualized to every patient and can guide research priorities to improve treatments for DCP. This commentary outlines current obstacles to improving treatment of DCP and addresses how precision therapeutics can address each of these obstacles through 4 key components: (1) identification of predictive biomarkers to select patients likely to develop DCP in the future and for whom early intervention may be appropriate to delay or prevent full manifestation of dystonia, (2) stratification of patients with DCP into subgroups according to shared features (clinical, functional, biochemical, etc) to provide a targeted intervention based on those shared features, (3) administration of an individualized dose of an effective intervention to ensure adequate concentrations of the therapeutic entity at the site of action, and (4) monitoring of objective biomarkers of response to intervention. With implementation of each of these components of precision therapeutics, new and more effective treatments for every person with DCP can be realized.