StemCells Inc. today announced that the U.S. Food and Drug Administration (FDA) has authorized the initiation of a Phase I/II clinical trial of the Company’s proprietary HuCNS-SC® product candidate (purified human neural stem cells) in dry age-related macular degeneration (AMD), the most common form of AMD. AMD is the leading cause of vision loss and blindness in people over 55 years of age, and approximately 30 million people worldwide are afflicted with the disease. There are no approved treatments for dry AMD.
“With the approval of this trial, we have accomplished something truly unique in the stem cell field, which is the extension of clinical testing of our proprietary human neural stem cell platform to all three elements of the central nervous system: the brain, spinal cord and eye,” said Martin McGlynn, President and CEO of StemCells, Inc. “The preclinical data supporting our IND is particularly compelling and we look forward to getting this trial underway.”
The Phase I/II trial will evaluate the safety and preliminary efficacy of HuCNS-SC cells as a treatment for dry AMD. The trial will be an open-label, dose-escalation study, and is expected to enroll a total of 16 patients. The HuCNS-SC cells will be administered by a single injection into the space beneath the retina. Patients’ vision will be evaluated using conventional methods of ophthalmological assessment at predetermined intervals over a one-year period. Patients will then be followed for an additional four years in a separate observational study.
Preclinical data submitted as part of the Company’s Investigative New Drug application demonstrated that HuCNS-SC cells protect host photoreceptors and preserve vision in a well-established animal model of retinal disease that is relevant to dry AMD. HuCNS-SC transplants significantly protect against the degeneration of photoreceptors, the key cells of the eye involved in vision. Moreover, the number of cone photoreceptors, which are responsible for central vision, remain constant over an extended period, consistent with the sustained visual acuity and light sensitivity observed. In humans, degeneration of the cone photoreceptors account for the unique pattern of visual loss in dry AMD. A summary of the Company’s preclinical data was published in the February issue of the international peer-reviewed European Journal of Neuroscience, and is available online at http://onlinelibrary.wiley.com/doi/10.1111/j.1460-9568.2011.07970.x/abstract.
“We have published the preclinical evidence demonstrating that our human neural stem cells might offer a safe, effective and simple approach to treating AMD and other retinal diseases,” said Stephen Huhn, MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc. “Our approach is to provide durable protection of photoreceptors, thereby preserving vision, as opposed to approaches that aim to replace photoreceptors or the retinal pigmented epithelial cells. Furthermore, our preclinical data supports our hypothesis that we can achieve clinical benefit with a single transplant in AMD patients.”