A woman in her 80s has become the first person to be successfully treated with induced pluripotent stem (iPS) cells
Laboratory made retinal cells has protected the eyesight of a woman

fighting her age related macular degeneration – a common form of progressive blindness.

Such stem cells can be coaxed to form many other types of cell. Unlike other types of stem cell, such as those found in an embryo, induced pluripotent ones can be made from adult non-stem cells – a discovery that earned a Nobel prize in 2012.

Now, more than a decade after they were created, these stem cells have helped someone. Masayo Takahashi at the RIKEN Laboratory for Retinal Regeneration in Kobe, Japan, and her team took skin cells from the woman and turned them into iPS cells. They then encouraged these to form retinal pigment epithelial cells, which are important for supporting and nourishing the retina cells that capture light for vision.

The researchers made a slither of cells measuring just 1 by 3 millimetres.

Before transplanting this into the woman’s eye in 2014, they first removed diseased tissue on her retina that was gradually destroying her sight. They then inserted the small patch of cells they had created, hoping they would become a part of her eye and stop her eyesight from degenerating.

Now the results are in. Published today, they show that the treatment hasn’t made the woman’s vision any sharper, but it does seem to have prevented further deterioration – with her vision now stable for more than two years. Since the graft, the woman says her vision is “brighter”.

“Takahashi and her team have done incredible work, and deserve all the praise they get for this project,” says Shinya Yamanaka, director of the Center for iPS Cell Research and Application at Kyoto University, who won the Nobel prize for inventing iPS cells and collaborated on this work. “This is a landmark study and opens the door to similar treatments for many diseases,” he says.

“This first iPSC-derived retinal graft is an important landmark in the field of retinal regeneration,” says James Bainbridge at University College London, and head of a trial at Moorfields Eye Hospital in London of similar grafts made instead from human embryonic stem cells.

One worry about this approach is that turning the stem cells into new tissues could lead to cancer-causing genetic mutations – though the team found no evidence of this in the treated woman. However, a trial of the technique in another person was cancelled in 2015, after tests revealed that the cells intended to be given to the man had developed genetic abnormalities.

But although it has taken many years to bring proven stem cell therapies to the clinic, many private centres around the world have been advertising unregulated treatments purporting to use stem cells for some time.

A second study published today shows just how badly some unregulated treatments described as stem cell therapies can go wrong. Three case reports of women given such treatments for age-related macular degeneration detail how one woman went blind and the vision of the other two became much worse.

All three ended up seeking emergency treatment in 2015, after each paid $5000 to a private clinic to receive injections of their own fatty tissue into their eyes.

“Patients and physicians in the US should be made aware that not all ‘stem cell’ clinics are safe, and that ‘stem therapy’ as provided in private clinics in the US is unproven and potentially harmful,” says Thomas Albini at the University of Miami’s Bascom Palmer Eye Institute, Florida, who subsequently treated two of the women.

Albini advises people to be suspicious of any procedure involving payment. “Most legitimate research in the US does not require patients to pay for the experimental procedures,” he says, adding that people should check whether a trial has been registered with the US Food and Drug Administration. “Be aware that if it sounds too good to be true, it may indeed not be true.”

By Andy Coghlan

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