A recognized developer and manufacturer of highly innovative stem cell treatments, StemCells Inc., has officially begun the Phase I/II dose-escalating clinical trial of its lead product candidate HuCNS-SC® cells. On September 11, 2013, the first patient in the Phase I/II clinical trial received a high dose of 1 million purified human neural stem cells for the management of dry AMD; marking the initiation of another developmental milestone in the study of efficacy and therapeutic benefits of HuCNS-SC® cells. This study participant — the fifth one of sixteen candidates involved in the study — is the first one to receive such a high dose (the previous four candidates received a smaller dose of 20,000 cells).
The enrollment for the Phase I/II clinical trial is already in progress at the Retina Foundation of the Southwest in Dallas, Texas and Byers Eye Institute at Stanford in Palo Alto, California.
Stephen Huhn, the Vice President of CNS Clinical Research at StemCells, recently commented:
“Advancing to the high dose, which is a five-fold increase from the low dose, is an important milestone in this trial. Testing a cell dose of this magnitude in all the remaining patients planned for the trial will enhance our ability to assess the effect of the cells on visual acuity.”
AMD is one of the leading causes of vision loss in adults over the age of 55 years. According to the latest statistics, almost 30 million patients worldwide suffer from AMD, with over 90% of patients suffering from the dry variety of the disease.
Mr. Huhn commented that StemCells is planning to further expand the number of research facilities involved in the clinical trials:
“Also, we are pleased to announce that we have received permission from the FDA to open three more U.S. trial sites in addition to the Retina Foundation of the Southwest and the Byers Eye Institute at Stanford, the two currently active centers. Expanding the number of sites will provide easier access to the trial for patients and help us achieve our goal of completing enrollment within the next nine months.”
About the clinical trial:
In order to determine the therapeutic safety of drug in human subjects and evaluate preliminary efficacy for the management of dry AMD, the Phase I/II trial of HuCNS-SC cells is currently in progress. The dose-escalating, open labeled study is being conducted on 16 patients, wherein a single dose of the HuCNS-SC cells is introduced into the affected eye (just beneath the retina via injection). The research team will be utilizing both traditional as well as innovative state-of-the-art methods for the assessment of results over a period of one year. Additionally, the company is planning to conduct a separate 4-year follow-up study to observe the long-term effects of the therapy.
The results of the preliminary study conducted on animal subjects was published in the European Journal of Neuroscience (February 2012 edition), and suggested that injected HuCNS-SC cells are capable of protecting and preserving vision by preserving photoreceptors from degenerative processes. The study was conducted on rat models with well-developed retinal disease (Royal College of Surgeons (RCS) rats) that are frequently used in research studies for degenerative retinal disorders. Most importantly, the study suggested that total number of cone receptors remained stable during the entire study period. Likewise, the primary cause of central visual loss in age related macular degeneration is degradation of cone photoreceptors.
About HuCNS-SC Cells:
The lead product candidate of StemCell Inc. is developed to be a highly innovative stem cell therapy that can be expanded and stored as a reserve bank of cells. It is derived from human neural stem cells, and the preliminary study conducted by the company suggests that once transplanted inside the nervous tissue, the cells are well-tolerated, with no signs of adverse effects or complications, such as tumor formation. Based on this behavior, researchers suggest that the transplanted neural stem cells, or HuCNS-SC cells, can survive for long period of time through engrafting. The research team also suggested that this response indicates that a single transplantation may produce promising results in the therapeutic management of a variety of disorders and diseases involving CNS. The data analysis about the drug safety in humans is yet to be determined.
If you are looking to participate in the ongoing clinical trial for the management of dry age-related macular degeneration (AMD), contact:
Retina Foundation of the Southwest, Dallas Texas at (214) 363-3911
Byers Eye Institute at Stanford, California at (650) 498-4486
Photo from http://www.stemcellsinc.com