Authors: Lauren Pulling, Editor
We’re now seeing cell-based therapies being investigated for a number of disorders, with neurodegenerative diseases being no exception. In 2015, the first-in-human clinical study of neural stem cells in patients with Parkinson’s disease (PD) began, with promising 6-month results now being reported. In light of this, we spoke to Russell Kern, CSO at International Stem Cell Corporation (ISCO; CA, USA), which developed the ISC-hpNSC cell line being used in the trial. In this interview, Russell tells us more about the trial, next steps, and how these cell lines are also being used to tackle other conditions including traumatic brain injury (TBI).
The ISCO is pioneering the use of ISC-hpNSCs in a number of trials – can you tell us about the first-in-human clinical study of neural stem cells in patients with PD?
ISCO received approval of the world’s first human pluripotent stem cell-based therapy for PD. This is an open-label, single center, uncontrolled Phase I study evaluating the safety and tolerability of intracranial transplantation of ISC-hpNSC in PD patients. This study is conducted at the Royal Melbourne Hospital in Australia and the principal investigator is Andrew Evans (Royal Melbourne Hospital, Australia). Three different dose regimens of ISC-hpNSC (30, 50 and 70 million cells) are tested in 12 patients divided into three cohorts of four patients each. So far four patients from the first cohort and three patients from the second cohort have been dosed with 30 and 50 million cells, respectively. No serious adverse events associated with ISC-hpNSC have been detected thus far.
Six month interim data from the first cohort shows improvements of 24% in the % OFF-Time and 19% in the % ON-Time without dyskinesia. There was an improvement of 35% in the Beck Depression Inventory and 33% in the Emotional Wellbeing dimension of the Parkinson’s Disease Quality of Life Score-39 (PDQ-39). The PDQ-39 score improved in several other dimensions including activities of daily living, mobility, bodily discomfort and cognitive impairment. Impulsive and compulsive disorders were diminished, as demonstrated by the 53% reduction in the Questionnaire for Impulsive-Compulsive Disorders in Parkinson’s Disease Rating Scale. These results are very encouraging because the first dose was sub-therapeutic and was used to test the safety and feasibility of the procedure. Patients from the second and third cohort receive higher cell doses shown to be therapeutic in our preclinical studies.
The next step now is to complete the Phase I study and analyze all the safety and efficacy data. After that, we are planning to file an IND to the US FDA to conduct a double-blind, placebo-controlled, multi-center Phase II study in PD patients with sites in the USA and Australia.
You’re also investigating the use of these cells to treat TBI – can you tell us more about this?