Objective: To follow the clinical status of 4 PD patients transplanted with NTCELL out to 81-130 weeks.

Background: NTCELL comprises neonatal porcine choroid plexus cells encapsulated in alginate microcapsules to protect from immune rejection. NTCELL produces an array of neurotrophic factors shown to promote survival of neuronal tissue. NTCELL implanted into rat brains survive rejection for over a year. Following NTCELL implantation into the brains of non-human primates with MPTP-induced parkinsonism, there was improved motor function and histological changes consistent with amelioration of the lesion. These studies have demonstrated an absence of PERV transmission. NTCELL has the potential to deliver neurotrophic agents for the treatment of Parkinson’s disease.

Methods: In this open label phase 1a study, 40 NTCELL capsules were implanted into 2 sites in the putamen contralateral to the worse side of 4 patients with PD who had been previously selected for deep brain stimulation. The patients were followed with clinical measures including Unified Parkinson’s disease Rating Scale (UPDRS) off and on, Unified Dyskinesia Rating Scale (UDysRS), and Quality of Life (PDQ-39). The 26 weeks results were presented at this conference in 2015. This presentation follows the patients for up to 130 weeks. Fluorodopa PET was performed at baseline and 6 months. There were no significant changes in uptake (as previously reported).

Results: At transplantation, the mean age was 62.4 (SD 4.3, Range 59.6 to 68.9) years and disease duration 12.5 (SD 7.3, Range 6 to 23) years. NTCELL was well tolerated. There was no evidence of PERV. At 42 weeks, UPDRS total off improved by a mean of 16.0 (SD 3.4, Range 11 to 18), the UDysRS improved by 13.5 (SD 7.3, Range 8 to 24), and the PDQ-39 improved by 8.9 (SD 4.0, Range 3.3 to 11.8). Updated data will be presented in the poster.

Conclusions: There has been a clinically significant improvement in the parkinsonism, dyskinesias and qualify of life of all 4 patients who received unilateral NTCELL transplantation. The biological basis remains unclear. The lack of PET change argues against increased nigrostriatal dopaminergic nerve endings. The sustained benefit argues against a purely placebo effect. The results encourage further study of this novel treatment for PD.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/safety-and-clinical-effects-of-ntcell-immunoprotected-alginate-encapsulated-porcine-choroid-plexus-cells-for-xenotransplantation-in-patients-with-parkinsons-disease-pd-81-to-130-weeks-fol/