StemCells Inc., (NASDAQ: STEM), based in Newark, California, has announced the dosing of a first patient with purified human neural stem cells directed at the treatment of geographic atrophy (GA), an advanced form of dry age-related macular degeneration (AMD). The study, formally titled “A Phase II proof-of-concept study of the safety and efficacy of HUCNS-SC subretinal transplantation in subjects with geographic atrophy of age-related macular degeneration”, aims to enrol approximately 63 patients and is scheduled for completion in December 2017.
To be eligible for inclusion in the study patients must have a diagnosis of geographic atrophy associated with AMD in both eyes and a BCVA of 20/320 or better in each eye at screening assessment. Five clinical sites in the US are participating in the study with clinics in Michigan, Illinois, Arizona and Texas however, the company is expecting as many as 20 centres to be enrolled over the duration of the trials. Known as the “Radiant Study”, the trial is designed to evaluate the safety and efficacy of human neural stem cells (“HuCNS-SC”) as a treatment for the degenerative retinopathy. According to the company, the trial will be run as a “fellow eye” controlled study, with all subjects receiving subretinal transplantation of HuCNS-SC cells via a single injection into the eye with the inferior BCVA. The better untreated eye will serve as the control and all patients will be followed for 12 months. The objective will be to demonstrate a reduction in the rate of disease progression in the treated eye versus the control eye. Previous work conducted by StemCells, Inc. has claimed to demonstrate safety data with HuCNS-SC cells transplanted directly into the brain, spinal cord and the eye. According to the company, such cells “have been shown to engraft and survive long-term [and] there is the possibility of a durable clinical effect following a single transplantation”. The experimental treatment comprises a highly purified composition of human neural stem cells “purified, expanded in culture, cryopreserved and then stored as banks of cells, ready to be made into individual patient doses when needed”.
Commenting on the milestone, Dr. Joel Naor, VP of Clinical Development at at the company stated that, “Initiation of this fellow eye controlled Phase II clinical trial re-affirms our leadership in the study of stem cells to treat disorders of the eye. AMD is a debilitating condition that severely impacts quality of life for millions of people and for which there is currently no treatment. HuCNS-SC cells may have the potential to preserve vision in those affected with dry AMD and possibly other degenerative