Objective: This study seeks to alleviate severe movement and tone disorders in cerebral palsy (CP) through chronic neuromodulation using deep brain stimulation (DBS), targeting motor dentate nuclei and cerebellar output pathways.

Background: CP, the most prevalent childhood physical disability, can encompass several movement disorders that manifest through dystonia and choreoathetosis in 15-17% of cases under a condition called dyskinetic CP (DCP). Current treatments for DCP often fall short due to low efficacy and side effect prevalence from oral medications and infection risk associated with intrathecal baclofen infusion. The cerebellum is considered an attractive target for DBS therapy in DCP due to its established role in dystonia pathophysiology and relative resistance to hypoxic-ischemic damage; small studies of DBS administered in the dentate nucleus for subjects with DCP have produced promising preliminary results, such as improvements in objective movement rating scales for dystonia/choreoathetosis.

Method: Ten participants aged 7-25 with DCP related to hypoxic-ischemic static encephalopathy will be recruited for implantation with a Medtronic Percept bidirectional neurostimulator into the dorsal dentate nucleus. By employing N-of-1 clinical trials with a cross-over design, this study will compare three months of effective stimulation versus three months of sham stimulation for each participant, randomly selecting stimulation type each month and evaluating weekly outcomes. Blinded conditions will be assigned to patients, caregivers and assessing clinicians. Videotaped automated movement recognition techniques and formal gait analysis will be piloted to evaluate kinematic metrics. Efficacy will be determined through analysis of cerebellar oscillatory activity as well as pre- and one-year postoperative structural/functional MRIs, which will serve to identify baseline disease severity as well as DBS response markers.

Results: So far, two candidates have been identified. The first DBS surgery will be in April 2024. The study is registered at clinicaltrials.gov (NCT06122675) and approved by the UCSF IRB.

Conclusion: This study seeks to create a safe and effective framework for cerebellar DBS therapy in patients with DCP, while providing greater understanding of this disorder via MRI-based disease marker identification and automated kinematic analysis.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/protocol-for-cerebellar-deep-brain-stimulation-in-children-and-young-adults-with-dyskinetic-cerebral-palsy-combined-n-of-1-trials-to-assess-safety-and-efficacy/