StemCells has transplanted the company's proprietary HuCNS-SC (purified human neural stem cells) into the first five patients in the final cohort of its 16-patient phase I/II trial for geographic atrophy of age related macular degeneration (GA-AMD). Each of the eight patients in this second cohort will receive a dose of 1 million stem cells into the most affected eye.
"We are very pleased with the rate of progress being made towards our goal of completing enrollment in this study by the end of this quarter," said Stephen Huhn, managing director, FACS, FAAP, vice president, CNS Clinical Research at StemCells, Inc. "We look forward to releasing the first interim results from this study next month and are planning to initiate a controlled phase II proof-of-concept study later this year."
An estimated 10 million people in the US either have age-related macular degeneration (AMD) or are at substantial risk for receiving the diagnosis, according to the Foundation Fighting Blindness. AMD is a degenerative retinal disease that typically strikes adults in their 50s or early 60s and gradually progresses to destroy central vision. Age-related macular degeneration refers to a loss of photoreceptors (rods and cones) from the macula, the central part of the retina. Overall, about 85% of patients with AMD suffer from the dry form of the disease, the advanced form of which is referred to as "geographic atrophy."
The phase I/II trial evaluates the safety and preliminary efficacy of HuCNS-SC cells as a treatment for dry AMD. The investigation is divided into two sequential cohorts. Subjects are enrolled into each cohort based on best-corrected visual acuity (BCVA), as determined by the Electronic Early Treatment Diabetic Retinopathy Study (E-ETDRS) acuity test. Patients with BCVA of less than or equal to 20/400 in the study eye were enrolled in Cohort I. Patients with less severe BCVA of 20/320 to 20/100 in the study eye are being enrolled in Cohort II. Cohort I consists of four subjects who were each transplanted with 200,000 stem cells, followed by four subjects who have each been transplanted with 1 million cells. Cohort II will consist of eight subjects who will undergo transplant with 1 million cells. The HuCNS-SC cells are administered by a single injection into the space behind the retina in the most affected eye. Patients' vision is being evaluated using both conventional and advanced state-of-the-art methods of ophthalmological assessment. Evaluations are being performed at predetermined intervals over a one-year period to assess safety and signs of vision improvement. Patients will be followed for an additional four years in a separate observational study.
StemCells, Inc. has demonstrated human safety data from completed and ongoing clinical studies of its proprietary HuCNS-SC cells. StemCells clinicians and scientists believe that HuCNS-SC cells may have broad therapeutic application for many diseases and disorders of the CNS. Because the transplanted HuCNS-SC cells have been shown to engraft and survive long-term, there is the possibility of a durable clinical effect following a single transplantation. The Company's preclinical research established that HuCNS-SC cells can be directly transplanted in the central nervous system (CNS) with no sign of tumor formation or adverse effects. The HuCNS-SC platform technology is a highly purified composition of human neural stem cells that are expanded and stored as banks of cells.
StemCells, Inc. is engaged in the research, development and commercialization of cell-based therapeutics and tools for use in stem cell-based research and drug discovery.