ACT Receives FDA Clearance for the First Clinical Trial Using Embryonic Stem Cells to Treat Macular Degeneration
News Nov 24, 2010
Advanced Cell Technology, Inc. has announced that the US Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application to immediately initiate a Phase I/II multicenter clinical trial using retinal cells derived from human embryonic stem cells (hESCs) to treat patients with Stargardt’s Macular Dystrophy (SMD), one of the most common forms of juvenile macular degeneration in the world. The decision removes the clinical hold that the FDA had placed on the trial.
Stargardt’s Macular Dystrophy causes progressive vision loss, usually starting in children 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 (RPE).
“There is currently no treatment for Stargardt’s disease,” said Dr. Robert Lanza, ACT’s Chief Scientific Officer. “Using stem cells, we can generate a virtually unlimited supply of healthy RPE cells, which are the first cells to die off in SMD and other forms of macular degeneration. We’ve tested these cells in animal models of eye disease. In rats, we’ve seen 100% improvement in visual performance over untreated animals without any adverse effects. Our studies showed that the cells were capable of extensive rescue of photoreceptors in animals that otherwise would have gone blind. Near-normal function was also achieved in a mouse model of Stargardt’s disease. We hope to see a similar benefit in patients with various forms of macular degeneration.”
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 advanced SMD. A total of twelve 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 (headed by Dr. Steven Schwartz); the Casey Eye Institute in Portland, Oregon (headed by Dr. Peter Francis of the Oregon Health Sciences University); the University of Massachusetts Memorial Medical Center in Worcester, Massachusetts (headed by Dr. Shalesh Kaushal, Chair of the Department of Ophthalmology); the UMDNJ – New Jersey Medical School in Newark, New Jersey (headed by Dr. Marco Zarbin, Chair, Institute of Ophthalmology and Visual Science); additional sites may be considered.
Earlier this year the FDA also granted Orphan Drug designation for the company’s RPE cells. As a result, the company is eligible to receive a number of benefits, including tax credits, access to grant funding for clinical trials such as this one, accelerated FDA approval, and allowance for marketing exclusivity after drug approval for a period of as long as seven years.
Raymond Lund, Ph.D., a scientific collaborator with ACT, and considered one of the world’s foremost experts in retinal cell physiology and vision restoration, commented, “The study results of ACT’s RPE cells implanted in the various animal models of macular degeneration was phenomenal. If ACT observes even a fraction of that benefit in humans, it will be nothing short of a home run."