First human stem cell trial using IVF embryos will treat patients facing blindness



Patients facing blindness with a rare and incurable eye disease are expected to become the first ever people to benefit from an embryonic stem-cell therapy.

Sufferers of Stargardt’s disease, which causes sight loss in early adulthood, are set to undergo the first clinical trials of the controversial treatment.

American scientists want to carry out transplant operations using stem cells from spare human embryos left over from IVF treatment.

Hope: Eyes suffering macular degeneration (above) could have retinas regrown using stem cells

If the research is cleared by the U.S. Food and Drug Administration (FDA), the 12 patients could undergo the procedures early in the new year.

‘The treatment for eye disease uses stem cells to re-create a type of cell in the retina that supports the photoreceptors needed for vision,’ said Dr Robert Lanza of Massachusetts-based Advanced Cell Technology.

‘These cells, called retinal pigment epithelium (RPE), are often the first to die off in Stargardt’s macular dystrophy.’

Stem cells are the body’s master cells. Embryonic stem cells, taken from a days-old embryo, are the most pliable as each one has the power to morph into any of the body’s cell types.

Scientists are trying to use them as the basis of a new field called regenerative medicine.



Embryonic stem cells are cultured in a lab to develop mature retinal pigment epithelium cells. These will be injected into damaged RPE cells in the retinas of people with macular degeneration. This should help them to focus their vision

Embryonic stem cells can be used for tailor-made treatments that a patient’s body is less likely to reject.

The hope is that they could be used to repair the damaged organs and tissues of patients with a relatively simple transplant procedure.

U.S. President Barack Obama lifted some of the restrictions imposed on their use by his predecessor George W Bush soon after he took office.

Another American biotechnology firm Geron won approval in January to test human embryonic stem cells to treat paraplegic patients who can use their upper bodies but cannot walk.



However, that trial is on hold while the company and FDA check on some side-effects seen in animals.

Stem cells: The body's master cells, taken from embryos, can morph into any of the body's cell types

But Dr Lanza is hopeful his trial will be able to start sooner – and make his patients the first in the world to receive any kind of treatment based on embryonic stem cells.

‘It has been over a decade since human embryonic stem cells were first discovered. The field desperately needs a big clinical success,’ he said.

‘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.’

The development is highly controversial because many pro-life groups are opposed to using human embryos in any kind of medical research.

But scientists believe that the benefits could revolutionise the treatment of many incurable disorders ranging from Parkinson’s to heart disease.

And Dr Lanza is confident of its success.

‘We've seen absolutely no adverse effects whatsoever in any of the preclinical experiments and our cells are more than 99.9 per cent pure,’ he told The Independent.

‘We certainly expect the FDA to come back with comments and questions but our hope is that we will start sometime early next year.



The condition: Stargardt’s disease causes macular degeneration so that sight is gradually lost over time







‘We’re optimistic and certainly confident in our own data. We”ve been in dialogue and we know what was on their mind and what they wanted us to do.

‘We’re hoping, assuming no hitches, to begin early next year, perhaps March.’

The first to benefit will be sufferers of Stargardt’s macular degeneration, which destroys the central part of the retina involved in recognising faces and reading words on a page.

ACT scientists also intend to follow this with an application to treat age-related macular degeneration, which affects more than 500,000 people in Britain and is the most common cause of blindness.

The treatment for eye disease uses stem cells to recreate a type of cell in the retina that supports the photoreceptors needed for vision.

These cells form the retinal pigment epithelium – which keep the light-sensing cells of the retina alive – which are often the first to die off in macular degeneration, which in turn leads to loss of vision, he said.

A single cell from a human embryo left over from IVF treatment was used in the creation of the stem cell ‘line’ that Dr Lanza and his colleagues cultivated in the laboratory.

By bathing the stem cells in a suite of chemical messengers, they were able to stimulate them to develop into fully mature retinal pigment epithelium cells.

Tests on animals found that transplants of the human cells into rats with macular degeneration resulted in a ‘100 per cent improvement’ in vision with no side-effects, Dr Lanza said.

Transplants into the 12 human volunteers chosen as guinea pigs for the first clinical trial will involve giving them mild immuno-suppressant drugs to prevent tissue rejection.