Advancing human neural progenitor cells (hNPCs) to FDA IND approval

NIH RePORTER · VA · IU1 · · view on reporter.nih.gov ↗

Abstract

Our work to date has demonstrated that Neural Progenitor Cells (NPCs) and Neural Stem Cells (NSCs) survive grafting to the spinal cord and extend very large numbers of axons over long distances through the lesioned rodent and non-human primate (NHP) spinal cord. Host axons also regenerate into cell grafts occupying the lesion site, and form new synaptic connections that act as neural relays across the injury to support functional improvement in both rodents and primates. We have identified a lead candidate human NSC/NPC line for clinical translation: an H9 human embryonic stem cell (a federally approved human cell line) that is driven to a spinal cord NSC/NPC fate. We refer to these cells as H9-scNSCs. H9-scNSCs survive grafting to rhesus monkey cervical spinal cord hemisection and contusion lesions. This promising line of work is advancing on a translational path, supported extensively by the VA’s RR&D Gordon Mansfield Spinal Cord Injury Consortium. We aim to bring this work to the point of readiness for human clinical trials in both subacute SCI and, subsequently, chronic SCI. In addition, our ongoing work in the Consortium aims to improve the potency of this intervention through combinations with rehabilitation and neuromodulation (e.g., targeted epidural spinal stimulation). The goal of the current application is to advance this program to submission of an FDA pre-IND, with the eventual goal of a human clinical trial of this promising approach. For the one-year funding period, this project will support project management (75% effort) and the services of a regulatory expert with experience in FDA submissions.

Key facts

NIH application ID
10642228
Project number
1IU1RX004569-01
Recipient
VA SAN DIEGO HEALTHCARE SYSTEM
Principal Investigator
MARK H. TUSZYNSKI
Activity code
IU1
Funding institute
VA
Fiscal year
2023
Award amount
Award type
1
Project period
2023-04-01 → 2024-03-31