Researchers in the U.S. have begun the world's first clinical trial of a new therapy for patients with spinal cord injuries, using human embryonic stem cells.
The groundbreaking experiment, run by a private biotechnology firm called Geron Corp., is still in an early phase, meant primarily to test the treatment for safety. But it could mark a landmark for a therapy that could have the potential to treat a variety of diseases.
The unidentified patient with a crushed spinal cord in being treated at Shepherd Center, a spinal cord and brain injury center in Atlanta, Ga., Geron Corp. said.
The patient is being treated with GRNOPC1, a trademark therapy that contains cells called oligodendrocyte progenitor cells. The cells turn into oligodendrocytes, which are cells that produce myelin, a nerve coating that allows impulses to move along the nerves.
The hope is that if the therapy works, the progenitor cells will produce new oligodendrocytes in the injured area of the patient's spine, potentially allowing for new movement.
Earlier studies on lab rats showed that GRNOPC1 significantly improved movement in animals with spinal cord injuries when injected seven days after the injury.
The company has said it plans to enroll eight to 10 patients in the study at sites across the U.S. All of the patients enrolled in the study will try the treatment within two weeks of their spinal cord injury. The therapy will be injected into the patients' spines between their third and 10th thoracic vertebrae, or roughly the middle to upper back.
This trial phase will take about two years, with each patient being studied for one year. If the therapy is shown to be safe in humans, that could then lead to larger and longer studies that would be more focused on the effectiveness of the therapy.
Later trials might also include patients with less severe spinal injuries and damage to other parts of the spine.
"Initiating the GRNOPC1 clinical trial is a milestone for the field of human embryonic stem cell-based therapies," Dr. Thomas B. Okarma, Geron's president and chief executive officer, said in a statement.