Last week, StemCells, Inc. announced the initiation of a Phase I/II clinical trial of its human neural stem cell product for the treatment of dry macular degeneration, to be conducted at the Retina Foundation of the Southwest’s (RFSW) Anderson Vision Research Center in Dallas, Texas. A summary of StemCell Inc.’s pre-clinical data was featured in the February 2012 issue of the European Journal of Neuroscience.
About StemCells, Inc.
StemCells, Inc., located in the United Kingdom and in Newark, California in the United States, is engaged in the research, development, and commercialization of cell-based therapies for use in stem cell-based research and drug discovery. The company is dedicated to addressing unmet medical needs through the development of stem cells as therapeutic agents to treat damage to, or degeneration of, major organ systems.
The company’s stem cell product is HuCNS-SC®, a highly purified composition of human neural stem cells, also called “tissue-derived” or “adult” stem cells. HuCNS-SC is an abbreviated version of Human Central Nervous System Stem Cells. HuCNS-SC is also in development as a potential treatment for a wide range of central nervous system disorders, including spinal cord injury and Alzheimer’s disease.
About the Retina Foundation of the Southwest
The Retina Foundation of the Southwest (RFSW) is an independent, non-profit research organization. The Foundation established the Anderson Vision Research Center in 1982, with a mission to discover the causes, treatments, and potential cures for hereditary and acquired blinding retinal disorders, including retinitis pigmentosa and age-related macular degeneration. Because the Center is funded by philanthropic and federal grants, patient services are free.
About Dry Age-Related Macular Degeneration
There are two types of age-related macular degeneration (AMD): dry (atrophic) and wet (neovascular). It’s possible to experience the wet type in one eye and the dry type in the other; in addition, the dry type can progress to wet in approximately 10-15% of cases.
The dry/atrophic type affects approximately 85-90% of individuals with AMD. Its cause is unknown, it tends to progress more slowly than the wet type, and there is not yet an approved treatment or cure. In dry AMD, small white or yellowish deposits, called drusen, form on the retina, beneath the macula, causing it to deteriorate or degenerate over time.
Although there is no approved treatment or cure for dry AMD at present, several clinical trials are in progress to assess potential therapies. I’ve blogged about several of the most promising, including Stem Cell Therapy for Dry Age-Related Macular Degeneration and Update on Stem Cell Clinical Trials for Dry Macular Degeneration and Stargardt’s Disease.
About the StemCells, Inc. 2012 Clinical Trial
From the StemCells, Inc website:
StemCells, Inc. is developing HuCNS-SC® human neural stem cells for the treatment of retinal degenerative diseases such as AMD, and is targeting initiation of a clinical trial in 2012. The Phase I/II trial will evaluate the safety and preliminary efficacy of HuCNS-SC cells as a treatment for dry AMD. The trial … 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 in the most affected eye. Patients’ vision will be evaluated using both conventional and advanced state-of-the-art methods of ophthalmological assessment. Evaluations will be performed at predetermined intervals over a one-year period to assess safety and signs of visual benefit. Patients will then be followed for an additional four years in a separate observational study.
More About Clinical Trials
Most clinical trials are designated as Phase I, II, or III, based on the questions the study is seeking to answer:
- In Phase I clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe and effective dosage range, and identify possible side effects.
- In Phase II clinical trials, the study drug or treatment is given to a larger group of people (100-300) to determine if it is effective and to further evaluate its safety.
- In Phase III studies, the study drug or treatment is given to even larger groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
- In Phase IV studies, after the Food and Drug Administration (FDA) has approved the drug, continuing studies will determine additional information, such as the drug’s risks, side effects, benefits, and optimal use.
VisionAware will provide updates on this important stem cell research as they become available.
Sources: NASDAQ Stock Market; ProactiveInvestors; MarketWatch; RTT News.