Advanced Cell Technology, Inc. (ACT), a leader in the field of regenerative medicine, announced the dosing of the first patients in each of its two phase I/II clinical trials for Stargardt's macular dystrophy and dry Age-related Macular Degeneration (dry AMD) using Retinal Pigment Epithelial (RPE) cells derived from human Embryonic Stem Cells (hESCs). The patients were treated Tuesday (July 12) by Steven Schwartz, MD, Ahmanson Professor of Ophthalmology at the David Geffen School of Medicine at UCLA and retina division chief at UCLA's Jules Stein Eye Institute . Robert Lanza, MD, chief scientific officer of ACT, attended the procedures. Both patients successfully underwent the outpatient transplantation surgeries and are recovering uneventfully.
Both the Stargardt's trial and the dry AMD trial will enroll 12 patients each, with cohorts of three patients each in an ascending dosage format. Both trials are prospective, open-label studies designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with Stargardt's and dry AMD at 12 months, the studies' primary endpoint.
“This first treatment milestone is welcomed by scientists, stem cell advocates and patients hoping for cures,” said Gary Rabin, interim chairman and chief executive officer of ACT. “The two trials could not have started any smoother, and we are very pleased to announce that the procedures went well. The dosing of the first patients represents an important milestone for ACT and opens the doors to a potentially significant new therapeutic approach to treating the many forms of macular degeneration. We believe that these procedures represent a key step forward in therapeutic stem cell research, and the capacity to treat a variety of devastating diseases.”
Dr Schwartz, the studies' principal investigator, explained, “One patient in each clinical trial, the Stargardt's trial and the dry AMD trial, has undergone surgical transplantation of a relatively small dose (50,000 cells) of fully-differentiated RPE cells derived from human embryonic stem cells. Early indications are that the patients tolerated the surgical procedures well. The primary objective of these phase I/II studies is to assess the safety and tolerability of these stem cell-derived transplants. We will be carefully monitoring our patients over the course of the trials. We are privileged to be collaborating with ACT and honoured to be working with these pioneering patients.”
Dry AMD, the most common form of macular degeneration, Stargardt's and other forms of atrophy-related macular degeneration are usually untreatable. Safe and effective therapies are greatly needed for the treatment of these common forms of blindness. Disease progression of both Stargardt's and dry AMD includes thinning of the layer of RPE cells in the patient's macula, the central portion of the retina and the anatomic location of central vision. With RPE cell death comes the loss of macular photoreceptors and loss of central vision. Currently both conditions are untreatable and often lead to legal blindness over a multi-year course. ACT's Stargardt's and dry AMD therapies treat these conditions by transplanting RPE cells in the patient's eyes before the RPE population is lost.
“Today -13 years after the discovery of human embryonic stem cells - the great promise of these cells is finally being put to the test,” said Dr Lanza. “The initiation of these two clinical trials marks an important turning point for the field. While we will continue writing research papers and carrying out more research, it's time to start moving these exciting new stem cell therapies out of the laboratory and into the clinic. Tens of thousands of people continue to die every day from diseases that could potentially be treated using stem cells. In the meantime, we intend to accelerate our efforts to translate new Embryonic Stem cell (ES) and induced Pluripotent Stem (iPS) cell therapies into the clinic. It has taken years of extensive research to get to this point. Our research and preclinical studies have demonstrated the safety and effectiveness of such therapies. We hope these cells may provide a treatment option not only for degenerative eye diseases, but for a wide spectrum of other debilitating conditions, ranging from diabetes to vascular and autoimmune diseases. Our team remains committed to moving the field of regenerative medicine forward from bench to bedside.”
The RPE is a highly specialized tissue located between the choroids and the neural retina. RPE cells support, protect and provide nutrition for the light-sensitive photoreceptors. Human embryonic stem cells differentiate into any cell type, including RPE cells, -- and have a similar expression of RPE-specific genes compared to human RPE cells and demonstrate the full transition from the hESC state.
Stargardt's Macular Dystrophy (SMD) is one of the most common forms of macular degeneration in the world. Stargardt's causes progressive vision loss, usually starting between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the Retinal Pigment Epithelium or RPE cell layer.
Degenerative diseases of the retina are among the most common causes of untreatable blindness in the world. As many as 30 million people in the United States and Europe suffer from macular degeneration, which represents a $ 25-30 billion worldwide market that has yet to be effectively addressed. Approximately 10 per cent of people ages 66 to 74 will have symptoms of macular degeneration, the vast majority suffering from the “dry” form of AMD - which is currently untreatable. The prevalence increases to 30 per cent in patients 75 to 85 years of age.
Advanced Cell Technology, Inc. is a biotechnology company applying cellular technology in the field of regenerative medicine.
Jules Stein Eye Institute is a world-renowned centre dedicated to the preservation of vision and the prevention of blindness and it has comprehensive programmes for the care of patients with eye disorders, research in the vision sciences, education in the field of ophthalmology and outreach to the community, coupled with its state-of-the art facilities, have brought national and international recognition to UCLA and the Institute, as it continues its mission to advance ophthalmology worldwide.