Human neural stem cells in patients with chronic ischaemic stroke (PISCES): a phase 1, first-in-man study

Area of Science:

• Neuroscience
• Regenerative Medicine
• Clinical Neurology

Background:

• CTX0E03 is a human neural stem-cell line used to develop CTX-DP for allogeneic therapy.
• Preclinical studies in rats showed dose-dependent sensorimotor function improvement after stroke with CTX-DP.
• These findings prompted an investigation into the safety and tolerability of CTX-DP in human stroke patients.

Purpose of the Study:

• To evaluate the safety and tolerability of CTX-DP in patients who have experienced an ischemic stroke.
• To assess neurological changes and adverse events following intracerebral administration of CTX-DP.

Main Methods:

• An open-label, single-site, dose-escalation study was conducted.
• 11 male patients (aged 60+) with stable disability post-ischemic stroke received single doses of CTX-DP (2-20 million cells) via stereotactic putamen injection.
• Safety endpoints included adverse events and neurological assessments over a 2-year follow-up period.

Main Results:

• No immunological or cell-related adverse events were observed in the 11 participants.
• Procedure-related adverse events or those due to comorbidities were noted.
• Five patients showed T2-weighted MRI hyperintensity around injection tracts.
• A median improvement of 2 points on the NIHSS score was observed at 2 years.

Conclusions:

• Single intracerebral doses of CTX-DP, up to 20 million cells, were found to be safe and well-tolerated in patients with chronic ischemic stroke.
• The observed neurological improvements suggest that CTX-DP holds promise for stroke patient treatment.
• Further clinical investigation of CTX-DP for stroke is warranted based on these safety and preliminary efficacy findings.