Embryonic Stem Cell Therapy For Stargardt's Disease On The Anvil

Stargardt’s disease, a rare, incurable eye disease, may be cured by embryonic stem cell therapy, according to a path-breaking research.

Clinical trials of the treatment for the disease that causes blindness early in adulthood, are expected to begin next year, according to the reports of British newspaper 'The Times'.

If the research is passed by the US Food and Drug Administration, 12 patients with Stargardt's disease, involved in the trial, could become the world's first to receive such treatment by embryonic stem cells.

Lead scientist Robert Lanza of the Advanced Cell Technology portrayed the application as a vital medical advancement.

"After years of research and political debate, we're finally on the verge of showing the potential clinical value of embryonic stem cells.”

"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."

Embryonic stem cells are master cells found in embryos that can form any of the specialised tissue types in the adult human body. They have great prospects of being a source of substitute tissue for treating disease or injury.

Advanced cell technology has employed a line of embryonic stem cells grown from an embryo to generate retinal pigment epithelial cells, a type of eye tissue that fails to function in Stargardt’s disease and age-related macular degeneration.

In studies of rats with respect to Stargardt's, embedding these cells showed the way to a considerable improvement in eyesight. The animals showed no adverse effects.

Dr Lanza said that the trial would involve 12 patients at three centres in the US, and is designed to appraise safety and tolerability at first.

If this succeeds, a larger study to scrutinise effectiveness will follow and if everything goes well a treatment could be agreed upon for more extensive use within three to five years.