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Stem cell trial for blindness to begin

23rd November 2010

A US biotech company, wants to test stem cells as a treatment for a type of blindness that usually takes hold in small children, Stargardt's Macular Dystrophy.

stemcell1The US Food and Drug Administration (FDA) has given permission to Advanced Cell Technology (ACT) to begin its phase I study in humans.

The company will fund the trial to test the technique, which transforms human embryonic stem cells into retinal cells, in 12 blind people.

While the treatment has already been tested on rats and mice, it is the first time that the researchers will try to use the stem cells on human eyes.

Bob Lanza, the company's chief scientific officer, said that the cells had performed spectacularly well, halting the progression of blindness in rodents without causing any side effects.

But the use of embryonic stem cells in the treatment of cancer and other diseases has sparked controversy in some quarters.

Opponents say that stem cell treatments involve the destruction of living human embryos, and hence human life.

However, the current technique involves a process that does not actually destroy the embryo.

Instead, the scientists only need to extract a 'blastomere' from the stem cell, which stays alive.

The same procedure is already used by in vitro fertilization clinics to run a check on embryos prior to implantation in women.

Then, after the blastomeres have been turned into retinal stem cells, they are injected directly into the eyes.

Since the 12 subjects have an advanced form of the disease, they do not expect to regain their vision fully.

Stargardt's disease causes blindness by gradually corroding the eye's ability to detect light.

People who have the disease experience blurry vision, have trouble seeing in low light, and eventually lose their ability to see.

If two parents carry the gene mutation involved in the degenerative process, any offspring may inheirit the disease, beginning to go blind as early as age six.

The purpose of the planned trial is to gauge the safety of ACT's technique, and the company hopes to eventually use the technique to prevent blindness in such children.

Lanza said that the disease was horrific, causing children to go blind as teenagers, and that he hoped his company could prevent the blindness from happening.

In rodents, the treatment created an abundance of retinal cells, which Stargardt's disease must first destroy before it can erode the eyes' photoreceptors, where photons begin the cascade of electrochemical changes that cause us to see.

ACT hopes that the treatment will be able to deter the progress of Stargardt's disease over the human lifespan, so that the disease will not make its way to people's photoreceptors.


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