It was only eight days ago that Advanced Cell Technology (“ACT”) announced that they have been granted U.S. Food and Drug Administration approval to proceed with human studies in Phase I/II clinical trials to treat Stargardt’s Disease, a form of juvenile macular degeneration, using human embryonic stem cell (hESC) derived retinal pigment epithelial cells (RPE). This in itself was a history making moment as this approval was only the second approval ever granted by the FDA for clinical trials with embryonic stem cells.
The management of Advanced Cell Technology is now looking to broaden their footprint in the industry and usher in a new age of medical therapies as today it was announced that the Company has filed an Investigational New Drug (IND) Application with the FDA to initiate a Phase I/II multicenter study using hESC RPE cells to treat patients with Dry Age-Related Macular Degeneration (“Dry AMD” or “central geographic atrophy”). Using these same technologies is an efficient move by the management of ACT as it should help expedite the decision-making process with the FDA as this approach in research is the same that was recently approved for the Stargardt’s clinical trials.
While Stargardt’s is a devastating disease, dry AMD is found far more frequently, effecting more than 30 million people worldwide with nearly half of those affected being Americans. Statistics show that nearly 10% of all people aged 66 to 74 will experience some form of macular degeneration, with the percentage increasing to 30% in people 75 to 85 years of age. The disease occurs when the photoreceptors in the macula begin to break down, an irreversible process at this point. Blindness often results over time as the macula slowly loses all function over time. Research has shown that the loss of photoreceptors is a direct result of earlier degeneration of the RPE layer of cells located just below the retina.
Much like the Stargardt’s clinical trials, 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 to patients with Dry AMD. A total of 12 patients will be enrolled in the study at multiple clinical sites. The sites which are currently under consideration are the Jules Stein Eye Institute at UCLA; the Ophthalmology Department at Stanford University School of Medicine; and the Edward S. Harness Eye Institute at Columbia University College of Physicians and Surgeons. Additional sites may be considered.
The market potential for these therapies, especially Dry AMD because of its prevalence, is enormous. Approximately 85 to 90 percent of all patients suffering from advanced AMD suffer from the Dry form, which correlates to approximately $25 to $30 billion in market space. As debilitating as the diseases are, there are no FDA-approved treatments for Dry AMD or Stargardt’s available on the market today. Perhaps even more importantly, pre-clinical research has showed extremely promising results for the ACT technologies as a bona fide therapy to treat the diseases.
Dr. Robert Lanza, Chief Scientific Officer for ACT, commented, “We can generate a virtually unlimited supply of healthy RPE cells.” Dr. Lanza continued, “In our animal studies, we observed significant improvement in visual performance over untreated animals, and did not find any adverse effects of the injection of RPE cells. In extending our studies to human patients, it is our hope that we will show that the injected RPE cells will rescue photoreceptors and slow, if not stop, the progression of macular degeneration.”
More information on Advanced Cell Technology and its groundbreaking research can be found on the Company’s website at www.advancedcell.com
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