Advanced Cell enrolls first patients in 2 clinical trials for eye disease stem cell treatments Print E-mail
By Olivia D'Orazio -   
Thursday, 16 June 2011 16:45
Advanced Cell Technology (OTCBB:ACTC) announced Thursday the enrollment of the first patients in its two Phase 1/2 clinical trials for the treatment of Stargardt's Macular Dystrophy (SMD) and Dry Age-Related Macular Dystrophy (Dry AMD), both degenerative eye diseases. The regenerative medicine-focused biotech company will use retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs) to treat SMD and Dry AMD, which are degenerative diseases that cause the loss of photoreceptors in the retina of the eye, eventually resulting in blindness.

The treatment works by implanting hESCs in the patients' eyes, to replace degenerative RPE cells in the eye before all vision function is lost. The hESCs have the ability to differentiate into any cell type and can demonstrate the full transition in state from hESC to RPE cell.

Advanced Cell said the study is intended to determine the safety and tolerability of the RPE cells one year after transplantation. The patients were enrolled at the Jules Stein Eye Institute at the University of California, Los Angeles (UCLA).

"The enrollment of the first patients in our two clinical trials marks an important step forward for the field of regenerative medicine," said CEO Gary Rabin.

"If these therapies work as we hope they will, particularly with small volumes of cells, then we should be in an excellent position to take advantage of our patented techniques for manufacturing large numbers of doses of RPE cells that can be conveniently stored and shipped to clinicians, following the basic manufacturing and distribution systems already familiar to pharmaceutical and biotech companies."

Stargardt's is one of the most common forms of macular degeneration in the world, usually starting in children between 10 to 20 years of age.

Dry AMD, meanwhile, is seen in older people, typically starting between the ages of 66 to 74, with prevelance increasing with age.

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