ACT Files IND With FDA For First Human Clinical Trial Using Embryonic Stem Cells to Treat Eye Disease
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While there is currently no treatment for SMD, several years ago ACT and its collaborators found that human embryonic stem cells could be a source of RPE cells. Subsequent studies found that the cells could restore vision in animal models of macular degeneration.
In the Royal College of Surgeon (RCS) rat model, implantation of RPE cells resulted in 100% improvement in visual performance over untreated controls without any adverse effects. The cells survived for more than 220 days and sustained extensive photoreceptor rescue. Functional rescue was also achieved in the ‘Stargardt’s’ mouse with near-normal functional measurements recorded at more than 70 days.
“It has been over a decade since human embryonic stem cells were first discovered,” said Dr. Robert Lanza, ACT’s Chief Scientific Officer. “The field desperately needs a big clinical success. After years of research and political debate, we’re finally on the verge of showing the potential clinical value of embryonic stem cell research. Our research clearly shows that stem cell-derived retinal cells can rescue visual function in animals that otherwise would have gone blind. We are hopeful that the cells will be similarly efficacious in patients.”
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 advanced patients with SMD.
A total of twelve patients will be enrolled into the study at three clinical sites, including 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, Worcester, Massachusetts (headed by Dr. Shalesh Kaushal, Chair of the Department of Ophthalmology); and the UMDNJ – New Jersey Medical School, Newark, New Jersey (headed by Dr. Marco Zarbin, Chair, Institute of Ophthalmology and Visual Science).
“It is an honor for us to be working with ophthalmology researchers of such high caliber,” said Edmund V. Mickunas, Advanced Cell’s Vice President of Regulatory Affairs. “They have been instrumental as collaborators on the clinical trial design and their input has been invaluable.”