Geron is planning to advance clinical development of its human embryonic stem cell (hESC)-based product, GRNOPC1, for the treatment of spinal cord injury. The plan is expected to enable Geron to re-initiate the phase I clinical trial of GRNOPC1 in patients with complete thoracic spinal cord injury and to support future expansion of the trial to patients with cervical injuries.
Geron has been performing a series of preclinical studies to expand the clinical program for spinal cord injury beyond patients with complete thoracic injuries.The goal is to test the safety and utility of GRNOPC1 in patients with complete and incomplete injuries in both thoracic and cervical regions.
As announced in one previous preclinical study, a higher frequency of animals developed cysts in the injury site than had been seen in numerous foregoing preclinical studies with clinical grade GRNOPC1. These cysts are non-proliferative, confined to the injury site, smaller than the injury cavity, and were not associated with adverse effects on the animals. As part of ongoing work to optimise GRNOPC1 manufacturing and product release, the company developed new candidate markers and assays. Data from studies using the new markers were submitted to the FDA. The IND for spinal cord injury was placed on clinical hold pending FDA review of the data.
Geron will complete a confirmatory preclinical study using GRNOPC1 that has been characterised by the new markers and assays, as agreed upon in discussions with the FDA. As part of the ongoing plan to advance clinical development to cervical patients, Geron had already initiated this preclinical study in an animal model of cervical injury.
FDA has advised that it concurs with Geron that positive data from this study can be used to support both release of the clinical hold and expansion to cervical patients. Geron expects the data from this study to enable re-initiation of the clinical trial in the third quarter of 2010.