Promising Preliminary Results in Trials of Embryonic Stem Cell Based Treatment for Macular Degeneration

24 January 2012

The report published today in The Lancet provides hope for patients suffering from dry macular degeneration, a very common cause of blindness. Although the work by scientists at University of California, Los Angeles and US company Advanced Cell Technologies is preliminary, the study shows that replacement of retinal pigment epithelial cells (the cell type that is lost in this disease) by grafts derived from human embryonic stem cells appears to be safe, and may lead to an improvement in vision.   

Professor Martin Pera, Program Leader of Stem Cells Australia and Chair of Stem Cell Sciences at the University of Melbourne commented that, "This landmark study is the first published account of a human trial of embryonic stem cell based therapy.  The authors described results from only two patients, and it is possible that the improvements that they observed may be related to other factors apart from the graft itself.  Nonetheless the early findings are most encouraging."

Macular degeneration is a very promising target for stem cell therapies, because the required cell type can be efficiently produced from embryonic stem cell cultures, and because the eye is easily accessible for surgery and for monitoring the progress of the graft. Centers in California, the United Kingdom, and elsewhere are also progression towards clinical trials of stem cell therapies in this disease. 

Professor Pera also said, "It will soon be clear whether these preliminary results published today are a harbinger of a new era in cell therapy for this devastating medical condition."

For more information:
Embryonic stem cell trials for macular degeneration: a preliminary report The Lancet [Published online 
 23 January 2012; DOI:10.1016/S0140-6736(12)60028-2]

Media coverage:
Stem cell treatments reverses blindness THE AUSTRALIAN 25 January 2012
First stem cell treatment in humans helps recover sight SYDNEY MORNING HERALD 25 January 2012
Stem cells used to treat macular degeneration ABC NEWS PM 24 January 2012
Therapy based on embryonic stem cells improved patient's vision LOS ANGELES TIMES 23 January 2012
First patients shown to improve with embryonic stem cells REUTERS 23 January 2012