Furthering its lead in stem cell research in ophthalmology, Advanced Cell Technology Inc., announced today that it had filed an Investigational New Drug (IND) application with the U.S. Food and Drug Administration, to initiate a Phase I/II multicenter study for the treatment of dry Age-Related Macular Degeneration (dry AMD) using human embryonic stem cell (hESC) derived retinal pigment epithelial (RPE) cells.
Dry AMD reportedly afflicts more than 30 million people worldwide, including an estimated13-15 million Americans. Approximately 10% of people 66 to 74 years of age will have findings of macular degeneration, and this prevalence increases to 30% in patients 75 to 85 years of age.
Dry AMD, or "central geographic atrophy," is the "dry" form of advanced Age-Related Macular Degeneration. Dry AMD occurs when the light-sensitive cells (photoreceptors) in the macula slowly break down, gradually blurring central vision in the affected eye. Over time, as less of the macula functions, central vision is gradually lost in the affected eye, often progressing to blindness. The loss of photoreceptors is a direct result of a preceding degeneration of the RPE layer of cells just below the retina. Dry AMD is much more common than wet AMD, which results from aberrant blood vessel formation in the eye. Some 85-90 percent of all people with intermediate and advanced AMD combined suffer from the dry form. Despite representing a $25-30 Billion market opportunity, there are currently no FDA-approved treatments for Dry AMD available.
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.
"We can generate a virtually unlimited supply of healthy RPE cells," said Robert Lanza, MD, ACT's Chief Scientific Officer. "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."
On November 22, ACT announced that it had received FDA approval to begin treating patients as part of the company's Phase I/II Clinical Trial treat Stargardt's Disease, a form of juvenile macular degeneration. (See Stem Cells in Ophthalmology: Update 2) Company scientists view the use of the same hESC derived RPE cells for both trials as the most efficacious approach, as it permits the company to leverage its experience with the FDA that it gained through the process of obtaining approval for the Stargardt's clinical trial to expedite the approval of its clinical trial in Dry AMD.
"We are seeing the beginning of new era in medical treatment," continued Dr. Lanza. "The hope that stem cell therapies may one day repair and regenerate diseased organs and tissue goes far beyond what can be accomplished with traditional medicine. This approval shows an apparent readiness by the FDA to work with researchers to move exciting new stem cell based therapies out of the lab and into the clinic."
"Filing this IND represents the culmination of years of innovation and hard work by ACT's scientific team," said William M. Caldwell IV, Chairman and CEO of ACT. "With this second IND, and our plans to expand our studies in Europe, ACT is positioning itself as a true `translational' leader in the field of regenerative medicine. When you are the first, the whole world's eyes are on you, and in our case, most especially the eyes of the patients that suffer from AMD. We do not intend to let them down. We welcome this challenge, and of course plan to scale up our operations, as necessary, to continue to meet our milestones and help validate this technology platform."
Dry AMD reportedly afflicts more than 30 million people worldwide, including an estimated13-15 million Americans. Approximately 10% of people 66 to 74 years of age will have findings of macular degeneration, and this prevalence increases to 30% in patients 75 to 85 years of age.
Dry AMD, or "central geographic atrophy," is the "dry" form of advanced Age-Related Macular Degeneration. Dry AMD occurs when the light-sensitive cells (photoreceptors) in the macula slowly break down, gradually blurring central vision in the affected eye. Over time, as less of the macula functions, central vision is gradually lost in the affected eye, often progressing to blindness. The loss of photoreceptors is a direct result of a preceding degeneration of the RPE layer of cells just below the retina. Dry AMD is much more common than wet AMD, which results from aberrant blood vessel formation in the eye. Some 85-90 percent of all people with intermediate and advanced AMD combined suffer from the dry form. Despite representing a $25-30 Billion market opportunity, there are currently no FDA-approved treatments for Dry AMD available.
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.
"We can generate a virtually unlimited supply of healthy RPE cells," said Robert Lanza, MD, ACT's Chief Scientific Officer. "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."
On November 22, ACT announced that it had received FDA approval to begin treating patients as part of the company's Phase I/II Clinical Trial treat Stargardt's Disease, a form of juvenile macular degeneration. (See Stem Cells in Ophthalmology: Update 2) Company scientists view the use of the same hESC derived RPE cells for both trials as the most efficacious approach, as it permits the company to leverage its experience with the FDA that it gained through the process of obtaining approval for the Stargardt's clinical trial to expedite the approval of its clinical trial in Dry AMD.
"We are seeing the beginning of new era in medical treatment," continued Dr. Lanza. "The hope that stem cell therapies may one day repair and regenerate diseased organs and tissue goes far beyond what can be accomplished with traditional medicine. This approval shows an apparent readiness by the FDA to work with researchers to move exciting new stem cell based therapies out of the lab and into the clinic."
"Filing this IND represents the culmination of years of innovation and hard work by ACT's scientific team," said William M. Caldwell IV, Chairman and CEO of ACT. "With this second IND, and our plans to expand our studies in Europe, ACT is positioning itself as a true `translational' leader in the field of regenerative medicine. When you are the first, the whole world's eyes are on you, and in our case, most especially the eyes of the patients that suffer from AMD. We do not intend to let them down. We welcome this challenge, and of course plan to scale up our operations, as necessary, to continue to meet our milestones and help validate this technology platform."
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