Tuesday, November 23, 2010

Stem Cells in Ophthalmology Update 2: ACT Gets Go-ahead to Treat Stargardt’s

As I noted in my September report on the Use of Stem Cells in Ophthalmology, it was anticipated that either the program at The London Project to Cure Blindness or Advanced Cell Technology’s program to treat Stargardt’s disease would be the first to get the go-ahead to begin approved human trials. I have not heard any news out of London, but earlier this week ACT received notification from the FDA that it was cleared to begin its human trials with human embryonic stem cells.

The multi-center trial will involve patients with Stargardt's Macular Dystrophy, one of the more common forms of juvenile macular degeneration.

Stargardt's Macular Dystrophy causes progressive vision loss. "There is currently no treatment for Stargardt's disease," said Robert Lanza, ACT's Chief Scientific Officer. "Using stem cells, we can generate a virtually unlimited supply of healthy (retinal pigment epithelium ) 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 percent 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.

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."

"Initiating our macular degeneration clinical trial represents a significant milestone in the progress of developing human embryonic stem cell-based therapies aimed at large worldwide markets," said William M. Caldwell IV, ACT's Chairman and CEO. "I think generations will look back at this time as one of the most exciting in the history of medicine. With the initiation of this clinical trial, and that of Geron's earlier this fall (for treatment of spinal disease), the field of regenerative medicine is poised to take embryonic stem cell therapies from the realm of nebulous potential to that of tangible and real treatments that will make a significant difference in the lives of millions of people worldwide. This is truly a `game changer' for the medical community."

Among the most common causes of untreatable blindness in the world are degenerative diseases of the retina. As many as thirty 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% of people ages 66 to 74 will have symptoms of macular degeneration, the vast majority the "dry" form of AMD - which is currently untreatable. The prevalence increases to 30% in patients 75 to 85 years of age.

"These patient numbers are staggering in size," stated Edmund Mickunas, ACT's Vice President of Regulatory, "and when the impact on health and quality-of-life is considered in that context, macular degeneration represents one of the more significant unmet medical needs in our society. With the momentum of this Stargardt's trial, and the unique experience we have gained as one of the few companies in the world having succeeded in taking an hESC program into the clinic, we are preparing to extend our lead with an IND in the use for treating age-related macular degeneration, as well as filing to begin clinical trials in Europe, in the very near future."


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