The US Food and Drug Administration has authorised the initiation of a Phase I/II clinical trial of US company StemCells' purified human neural stem cells (HuCNS-SC) for the treatment of dry age-related macular degeneration (AMD).
The trial aims to evaluate the safety and preliminary efficacy of the HuCNS-SC cells in dry AMD treatment. Company president and chief executive Martin McGlynn stated: 'The preclinical data supporting our Investigational New Drug application is particularly compelling and we look forward to getting this trial under way.'
The company added that it expected to enrol 16 patients on the open-label, dose escalation study, who would then have the HuCNS-SC cells administered by a single injection in the space beneath the retina. Patients would be evaluated with ophthalmological assessment at predetermined intervals over a one-year period, and then followed for an additional four years in a separate observational study.
The company's pre-clinical data, published in February's European Journal of Neuroscience, showed that the cells protected host photoreceptors and preserved vision in a well-established animal model of retinal disease that was relevant to dry AMD.
Stephen Huhn, vice president at StemCells, said: 'Our approach is to provide durable protection of photoreceptors, thereby preserving vision, as opposed to approaches that aim to replace photoreceptors or retinal pigmented epithelial cells.'
