Evaluate the safety and tolerability of transplanting human pluripotent stem cell derived neural stem cells (ISC-hpNSC) into patients with Parkinson’s disease (PD).

In this open label, single arm, dose escalating, Phase 1 clinical trial, 12 participants are recruited into three cohorts of four participants each. Patients receive stereotactic bilateral injections into the caudate nucleus, putamen and substantia nigra of 3x10⁷ (1^st cohort), 5x10⁷ (2^nd cohort), and 7x10⁷ (3^rd cohort) ISC-hpNSC cells. Participants are evaluated for 12 months in the active phase and five years in the safety follow-up phase. The primary endpoint evaluates the incidence of treatment-emergent adverse events (AEs) and serious adverse events (SAEs). Secondary endpoints evaluate preliminary efficacy by comparing neurological scores to baseline.

Ten subjects have been successfully transplanted with ISC-hpNSC and eight subjects (full 1^st and 2^nd cohorts) have completed the 12 month active phase and entered the five year safety follow-up phase. The transplanted cells seem to be well tolerated and no tumors, infection, or test article related SAEs have been reported thus far. Preliminary efficacy measures show a dose-dependent improvement, with the 2^nd cohort on average outperforming the 1^st cohort, in Hauser Motor Diary, PD Quality of Life Score-39, and Clinical Global Impression at six months post-transplantation. 

Interim results of the world’s first human pluripotent stem cell based therapy for PD show that transplantation of ISC-hpNSC is safe, well tolerated, and can potentially improve the quality of life of patients.