Biotechnology Company Geron has been given the all clear for the world's first clinical trial involving human embryonic stem cells (hESCs). This comes a year after the US Food and Drug Administration (FDA) placed the study on hold amid safety fears.
The Phase I trial will use stem cells derived from human embryos to attempt to regenerate neural support tissue in patients with severe spinal cord injury. Geron are hopeful this could restore some sensation and movement in patients.
The treatment, called GRNOPC1, was initially given the go-ahead by the FDA back in January 2009. The following August however, the FDA banned the trial after the appearance of cysts in some of the animals given the treatment in a preliminary study.
During the last year, Geron have developed new markers and assays, which improve the guidelines for treatment. On completing a confirmatory preclinical trial in animals using the new markers and assays, Geron submitted the results to the FDA and the hold on the human study was lifted on July 30th.
Geron's president and CEO, Dr Okarma, said: 'Our goals for the application of GRNOPC1 in sub-acute spinal cord injury are unchanged - to achieve restoration of spinal cord function by the injection of hESC-derived oligodendrocyte progenitor cells directly into the lesion site of the patient's injured spinal cord'.
The primary aim of the Phase I trial is to assess safety of the treatment in human patients, although Geron will also be assessing how well the treatment improves muscle function and feeling in the patients' upper body or lower extremities.
'We are pleased with the FDA's decision to allow our planned clinical trial of GRNOPC1 in spinal cord injury to proceed', said Dr Okarma. 'Additionally, we are now formally exploring the utility of GRNOPC1 in other degenerative CNS (central nervous system) disorders including Alzheimer's, multiple sclerosis, and Canavan disease'.