RT Journal Article SR Electronic T1 Direct delivery of an investigational cell therapy in patients with Parkinson’s disease: an interim analysis of feasibility and safety of an open-label study using DBS-Plus clinical trial design JF BMJ Neurology Open JO BMJ Neurol Open FD BMJ Publishing Group Ltd SP e000301 DO 10.1136/bmjno-2022-000301 VO 4 IS 2 A1 Jorge E Quintero A1 John T Slevin A1 Julie A Gurwell A1 Christopher J McLouth A1 Riham El Khouli A1 Monica J Chau A1 Zain Guduru A1 Greg A Gerhardt A1 Craig G van Horne YR 2022 UL http://neurologyopen.bmj.com/content/4/2/e000301.abstract AB Objective To evaluate the interim feasibility, safety and clinical measures data of direct delivery of regenerating peripheral nerve tissue (PNT) to the substantia nigra (SN) in participants with Parkinson’s disease (PD).Methods Eighteen (13 men/5 women) participants were unilaterally implanted with PNT to the SN, contralateral to the most affected side during the same surgery they were receiving deep brain stimulation (DBS) surgery. Autologous PNT was collected from the sural nerve. Participants were followed for safety and clinical outcomes for 2 years (including off-state Unified Parkinson’s Disease Rating Scale (UPDRS) Part III assessments) with study visits every 6 months.Results All 18 participants scheduled to receive PNT implantation received targeted delivery to the SN in addition to their DBS. All subjects were discharged the following day except for two: post-op day 2; post-op day 3. The most common study-related adverse events were hypoaesthesia and hyperaesthesias to the lateral aspect of the foot and ankle of the biopsied nerve (6 of 18 participants experienced). Clinical measures did not identify any hastening of PD measures providing evidence of safety and tolerability. Off-state UPDRS Part III mean difference scores were reduced at 12 months compared with baseline (difference=−8.1, 95% CI −2.4 to −13.9 points, p=0.005). No complications involving dyskinesias were observed.Conclusions Targeting the SN for direct delivery of PNT was feasible with no serious adverse events related to the study intervention. Interim clinical outcomes show promising results meriting continued examination of this investigational approach.Trial registration number NCT02369003.No data are available.