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Stem cell trial for eye diseases begins

18 July 2011

By Dr Sophie Pryor

Appeared in BioNews 616

Doctors in the USA have begun treating patients in two clinical trials for degenerative eye diseases. The studies at the Jules Stein Eye Institute at the University of California, Los Angeles (UCLA), will test whether specialised eye cells, which have been produced from human embryonic stem cells (hESCs), can be used to treat dry age-related macular degeneration (dry AMD) and Stargardt's macular dystrophy.

First to take part was a 77-year-old woman affected by dry AMD and a 27-year-old woman with Stargardt's, who both received injections of around 50,000 eye cells called retinal pigment epithelium (RPE) cells into the back of one eye. Professor Steven Schwartz, who administered the treatments, said: 'Early indications are that the patients tolerated the surgical procedures well'.

Twelve patients suffering from each condition will be recruited to the trials and will each receive an injection of 50,000 to 200,000 RPE cells before being monitored for one year. Professor Schwartz said the primary objective of the studies is 'to assess the safety and tolerability of these stem cell-derived transplants'.

Dr Robert Lanza, chief scientific officer at Advanced Cell Technology (ACT), which is sponsoring the trial, said: 'The great promise of these cells is finally being put to the test... the initiation of these two clinical trials marks an important turning point for the field'. He said this type of stem cell research 'may provide a treatment option not only for degenerative eye disease, but for a wide spectrum of other debilitating conditions'.

Dry AMD and Stargardt's are common forms of blindness for which there is currently no cure. Both diseases are caused by degeneration of the RPE, the pigmented layer of the retina which provides nutrition and support for the light-sensitive cells of the eye. These cells eventually die due to loss of the RPE, leading to a loss of vision. ACT has engineered hESCs to form functional RPE cells hoping they will ultimately be capable of replacing those lost due to the disease, improving vision. Another round of treatment is due to begin in August.

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