First successful transplantation of iPSC-derived neural stem/progenitor cells into human participant to treat spinal cord injury

Written by Harriet Stanwix

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Researchers have completed the first successful transplantation of iPSC-derived neural stem/progenitor cells into a human participant during a clinical study at Keio University.

A landmark clinical study at Keio University (Tokyo, Japan) has completed the first successful transplantation of iPSC-derived neural stem/progenitor cells (iPSC-NS/PCs) into a human participant, with the aim of treating spinal cord injury (SCI). This clinical study aims to assess the safety of iPSC-NS/PC transplantation in patients with subacute SCI to understand the impact it could have on neurological function and quality-of-life outcomes.

Currently, treating subacute and chronic SCI is an extreme challenge for clinicians. SCI can occur due to a wide range of reasons and outcomes for patients can be difficult, sometimes resulting in irreversible damage to various systems in the body.

The team, led by Hideyuki Okano, previously undertook research in small animals during which human iPSC-NS/PCs were transplanted into mice with SCI to determine a safe benchmark for the number of cells to use in human patients. During this study, an ultrasound probe was used to establish the epicenter of the injury. The spinal cord membrane was then cut to uncover the damaged area, after which approximately two million iPSC-NS/PCs were transplanted into the epicenter of the injury.

Only one patient has undergone this process so far and the results will be closely monitored by an independent data monitoring committee who will evaluate the safety of the study based on data obtained up to about three months after the first transplant. If it is decided that the study should continue, the team will conduct transplantations for three additional participants.