First patient enters trial to test safety of stem cells in spinal injuryBMJ 2010; 341 doi: https://doi.org/10.1136/bmj.c5724 (Published 12 October 2010) Cite this as: BMJ 2010;341:c5724
The first patient has been recruited to a clinical trial to assess the safety of oligodendrocyte progenitor cells derived from human embryonic stem cells to treat complete spinal cord injury, a US company, Geron, reported on Monday 11 October.
The phase I study is investigating the safety and tolerability of stem cell derived oligodendrocyte progenitor cells. The aim is to recruit a group of patients with injuries graded as A on the American Spinal Injury Association impairment scale, which means that they have complete loss of sensory and motor function, with the last fully preserved neurological level from the T3 to T10 thoracic vertebrae.
Patients who meet these criteria are being recruited at seven US medical centres. Two million of the cells will be injected into the site of each patient’s spinal cord lesion at a single time point between seven and 14 days after the injury occurs.
The trial’s primary end point is safety, measured by the frequency and severity of adverse events related to the injected stem cells, to the injection procedure, or to the concomitant immunosuppression administered during the first year after treatment. Neurological function will also be assessed as a secondary end point.
The stem cells being used in the trials have been developed by Geron. These cells are precursors to oligodendrocytes, which have several functions in the nervous system, including producing myelin that enables efficient conduction of nerve impulses.
Oligodendrocytes are lost in spinal cord injury, resulting in loss of myelin and neurones, which disrupts transmission of nerve impulses and results in paralysis.
Preclinical studies in which oligodendrocyte progenitor cells were injected into the site of spinal cord injuries in animals showed that the cells migrated throughout the lesion site. They then matured into functional oligodendrocytes that remyelinated axons and produced neurotrophic factors, resulting in improved locomotion in the treated animals.
The ultimate goal for the use of these cells in humans is to repair spinal cord injuries by injecting the cells directly into the spinal cord lesion.
Chris Mason, professor of regenerative medicine bioprocessing at the Advanced Centre for Biochemical Engineering, University College London, said, “This first in man study marks the dawn of the ‘stem cell age.’ The essential transition, from bench to bedside, is a critical step change in the progression of embryonic stem cells towards eventual cures.”
He added, “There are still many years of rigorous testing ahead, and no doubt there will be setbacks and failures before we have safe and effective cell based therapies. This pivotal clinical trial is a major morale boost for scientists, clinicians, and, most of all, patients by finally commencing the transformation of stem cells from a scientific curiosity into advanced health care.”
Thomas Okarma, president and chief executive office of Geron, said, “When we started working with human embryonic stem cells in 1999, many predicted that it would be a number of decades before a cell therapy would be approved for human clinical trials. This accomplishment results from a succession of inventive steps to enable production of master cell banks [to the necessary standard for use in humans] and preclinical studies in vitro and in animal models of spinal cord injury, leading to [authorisation] by the FDA to initiate the clinical trial.”
The study is planned to be completed by October 2012.
Cite this as: BMJ 2010;341:c5724