Significant advancements in somewhat controversial stem cell research continue in 2011. Having no current treatments available, the leading cause of vision loss may be able to be treated using human embryonic stem cells. Just today, it was announced that the US Food and Drug Administration has approved clinical trials of an application to treat Dry Age-Related Macular Degeneration (AMD) using retinal pigment epithelial cells derived from human embryonic stem cells. Dry AMD afflicts between 10-15 million Americans.

Advanced Cell Technology (ACT) announced that they are now permitted to initiate a Phase I/II multicenter clinical trial to treat patients with Dry AMD, the most common form of macular degeneration in the world. Age-Related Macular Degeneration has two predominant forms, wet and dry. Dry AMD is the most common form, accounting for almost 90% of all cases. The progress of Dry AMD includes a breakdown or thinning of the layer of RPE cells in the patient's macula, the region at the center of the retina responsible for high acuity vision. Over time, the progressive loss of RPE cells and accompanying loss of photoreceptors can cause severe vision loss and even blindness.

Dry macular degeneration is diagnosed when yellowish spots known as drusen, begin to accumulate in and around the macula, an oval-shaped highly pigmented yellow spot near the center of the retina of the human eye. The macula absorbs excess blue and ultraviolet light that enter the eye, and acts as a natural sun block for this area of the retina. It is believed these spots are deposits or debris from deteriorating tissue. Dry AMD through a period of years slowly can progress into a gradual degradation of retinal cells that also can cause severe vision loss. There are currently no treatments available for this prevalent disease of an aging global population.

Gary Rabin, Interim Chairman and CEO of ACT, a biotechnology company applying cellular technology in the field of regenerative medicine, in a statement says: "ACT is now the first company to receive FDA clearance for two hESC trials, and is now a true translational leader in the field of regenerative medicine. It marks a major step forward, not just within the stem cell sector, but, potentially for modern healthcare techniques. We plan to proceed into the clinic with both of our hESC-based programs as quickly as possible." The Phase I/II trial will be a prospective, open-label study that is designed to determine the safety and tolerability of the RPE cells following sub-retinal transplantation into patients with Dry AMD. Twelve patients will be enrolled in the study at multiple clinical sites. Sites currently under consideration are the Jules Stein Eye Institute at UCLA, and the Ophthalmology Department at Stanford University School of Medicine. Additional sites may be considered.

Robert Lanza, MD, ACT's Chief Scientific Officer provides some more background: "Dry AMD is the leading cause of blindness in individuals over the age of 55. As the population ages, the incidence of AMD is expected to double over the next 20 years, further exacerbating this unmet medical need. Using our clinical-grade hESC lines, we are able to generate a virtually unlimited and reproducible supply of healthy RPE cells. Because only a small number of cells (50-200K) are needed to treat each patient, manufacturing and distribution of the therapeutic product is scalable with many similarities to the drug businesses that pharmaceutical companies understand well. Based on our animal model studies, we are very excited about the opportunity to treat patients. In a rat model of macular degeneration, we have seen a remarkable improvement in visual performance over untreated animals, without any adverse effects. We have also maintained near-normal function in a mouse model of Stargardt's Disease, a form of juvenile macular degeneration. In addition to this trial, we plan to concurrently use our RPE cells in our Phase I/II Clinical Trial for Stargardt's Disease, which received the green light from the FDA in November. We hope to see a similar benefit in both Stargardt's Disease and Dry AMD patients." Stargardt's disease (also known as fundus flavimaculatus and Stargardt macular dystrophy) is the most common form of inherited juvenile macular degeneration. Inherited as an autosomal recessive trait, it is a severe form of MD that begins in late childhood, leading to legal blindness. Stargardt disease is symptomatically similar to age-related macular degeneration, and it affects approximately one in 10,000 children.

While the initial portion of the clinical trial will focus on safety, in subsequent clinical trials the Company hopes to demonstrate that the retinal pigment epithelial cells injected into the retinal space will be capable of slowing or halting progression of the disease, and potentially even restoring some visual acuity to patients.

Edmund Mickunas, ACT's Vice President of Regulatory Affairs concludes: "It is estimated that over ten million Europeans suffer from Age-Related Macular Degeneration, representing a vast unmet need and a significant market opportunity. We are moving ahead aggressively to seek regulatory clearance from the European Medicines Agency to conduct clinical trials in Europe." Specific inclusion and exclusion requirements and Investigator contact information will be posted at clinicaltrials.

Sy Kraft, B.A.

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