September 22, 2011 5:42 pm

Stem cell trial offers hope to blind

By Clive Cookson

The first clinical trial outside the US of a treatment based on human embryonic stem cells – which could eventually cure blindness – is to take place at Moorfields Eye Hospital in London.

Young people suffering from Stargardt’s disease, a genetic disorder that destroys retinal tissue, leading to blindness at an early age, will receive new retinal cells derived from embryonic stem cells.

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IN Science

Advanced Cell Technology (ACT), a biotech company based in Massachusetts, has received approval from the UK Medicines and Healthcare Products Regulatory Agency to carry out a trial of its retinal cells at Moorfields. A similar trial is getting under way in the US.

Professor James Bainbridge, who will lead the Moorfields trial, will inject a suspension of 50,000 cells into the retina of each patient in an operation likely to take about an hour. The first procedure is expected before the end of this year.

“There is real potential that people with blinding disorders of the retina, including Stargardt’s disease and age-related macular degeneration, might benefit in the future from transplantation of retinal cells,” said Prof Bainbridge. “The ability to generate retinal cells from stem cells in the laboratory has been a significant advance and the opportunity to help translate such technology into new treatments for patients is hugely exciting.”

Robert Lanza, ACT chief scientist, said the announcement confirmed the UK’s place as the leading European centre for stem cell development, and Moorfields as the European leader for research into eye diseases.

Separately, scientists at Moorfields are also developing their own embryonic stem cell technology to treat dry age-related macular degeneration (AMD) – a much more common condition that affects older people – under the auspices of the London Project to Cure Blindness. That is scheduled to start clinical trials next year, led by Professor Pete Coffey.

“The two projects are independent but will be able to learn from one another,” said Prof Bainbridge. “There are similarities and differences between them.”

The ACT procedure is simpler: the retinal cells are injected in a suspension into the eye where, if all goes well, they will form themselves into a layer on the retina. The London Project aims first to generate a sheet of retinal cells in the lab and then insert that into the eye.

Although the ACT trial at Moorfields will start with Stargardt’s disease, the company is also beginning a clinical trial in the US that uses the same technology to treat AMD.

BACKGROUND NEWS

The first ACT trial is designed primarily to assess the safety of the therapy. For example, researchers want to confirm that the patients’ immune system will not reject the transplanted cells. They will proceed slowly and cautiously, treating a dozen patients over the course of a year or two.

● Stem cells are immature cells that can develop into a wide range of specialised tissues.

● Adult stem cells, found particularly in bone marrow, have been known for decades. But the discovery in 1998 that more versatile stem cells could be extracted from very early human embryos ignited medical interest in the field.

● Hundreds of scientists worldwide are working with human embryonic stem cells, despite stringent restrictions on research in many countries, imposed by politicians responding to moral and religious concerns.

● The first embryonic stem cell product to reach the clinic – in October 2010 – was a treatment for spinal cord injury from Geron of California.

● The retinal treatment for Stargardt’s disease from Advanced Cell Technology – the subject of the Moorfields trial – is the second embryonic stem cell product to reach the clinic.

● Foetuses aborted for medical reasons are another source of stem cells. ReNeuron of the UK is running a preliminary clinical trial of a product based on foetal stem cells to treat stroke.

● Induced pluripotent stem cells (iPSCs), first made in 2007, are embryonic-like stem cells generated directly from adult skin cells without involving embryos. They avoid some ethical and practical problems of embryonic stem cells but are still far from clinical use.

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