Advanced Cell Technology, Inc. announced that it completed discussions with the Food and Drug Administration (FDA) regarding its retinal pigmented epithelial (RPE) cell therapy. ACT is working with the agency to fulfill the FDA’s requirements to bring its RPE cell therapy into human clinical trials for the treatment of retinal degenerative diseases such as Retinitis Pigmentosa, Stargardt’s disease, and dry age-related macular degeneration (AMD).
Studies of the company’s proprietary RPE cells have shown that the therapy may ultimately provide effective treatment of degenerative retinal disorders including macular degeneration, which represents a $28 billion dollar market. AMD affects more than 30 million people worldwide and is the leading cause of blindness in people over 60 in the United States. The prevalence of AMD begins to increase after the age of 50. Approximately 15% of people over 75 have the condition. To date, AMD patients have had few if any effective therapies for treatment; thus, the need for novel therapies is clear.
In November at Neuroscience 2007, researchers at Oregon Health and Science University (OHSU) presented results of a study that used ACT RPE cells manufactured under Good Manufacturing Practices (GMP) conditions (21CFR211) at the company’s facility in Worcester, Massachusetts. The RPE cells were thoroughly characterized and cryo-preserved and shipped to researchers at OHSU for transplantation. The conclusions drawn by researchers were that visual function can be rescued and preserved in this animal model of disease utilizing ACT’s GMP-manufactured human ES-derived RPE cells with a functional dose threshold and that these cells may provide an effective donor cell source to rescue photoreceptors in conditions like AMD, where RPE function is compromised.
The next step in the development of the program will be to complete several safety studies utilizing the GMP-manufactured RPE cells. Pilot studies to date have shown the cells to be safe, well tolerated, and non-migratory. The Good Laboratory Practice (GLP) compliant studies are required prior to filing an Investigational New Drug (IND). The safety studies will include several key areas of examination, including, tumorigenicity potential, cell tolerability, cell survival, general safety, and potential for biodistribution.
“We are pleased to complete this key milestone as we move our RPE program toward the clinic,” said William Caldwell, IV, Chairman and CEO of Advanced Cell Technology. “Based upon our cell characterization data, pharmacology studies, and the overall safety profile, we are excited by the prospects for this program and the potential to treat a large unmet medical need. The successful completion of our pre-IND meeting represents another step forward towards the filing of the IND for our RPE Program and ultimately bringing the therapy to the bedside. We are one step closer to bringing our second stem cell therapy to patients in need.”
Illustration: Microsoft Clipart.
Advanced Cell Technology Press Release (02/01/08)
Medical News Today (02/06/08)