Stem cells used to improve low vision

11:00am Wednesday 15th October 2014

content supplied by NHS Choices

After 12 months:

In the Stargardt's macular dystrophy group, after six months:

After 12 months:

The higher dose of cells - 150,000 - gave better results in the age-related macular degeneration group. A lower dose of 50,000 gave the best results in the Stargardt's macular dystrophy group.

Participants also had, on average, improved quality of life, as measured by a questionnaire.

Nobody had acute transplant rejection, abnormal excess growth of the transplanted stem cells, or tumour formation. In addition, they did not experience retinal detachment or changes in the blood vessels of the retina.

There were some side effects from the treatment, however, including:

 

How did the researchers interpret the results?

The researchers concluded that they "have shown the medium-term to long-term safety, graft survival, and possible biological activity of pluripotent stem cell progeny in individuals with atrophic age-related macular degeneration and Stargardt's macular dystrophy.

They go on to say that, "Our results suggest that hESC-derived cells [human embryonic stem cells] could provide a potentially safe new source of cells for the treatment of a variety of unmet medical conditions caused by tissue loss or dysfunction.

"The goal should be to treat patients early in the disease, potentially increasing the likelihood of photoreceptor and central visual maintenance or rescue in amenable retinal disorders."

 

Conclusion

These phase one/two studies have shown human embryonic stem cells can be developed into retinal cells in the laboratory and successfully transplanted into the eye, causing clinically significant visual improvements.

The technique does come with the usual potential surgical complications, but other major side effects were not found.

Limitations of the study include the small size, but this is normal in early trials whose primary aim is to determine safety.

Larger studies will be required to determine the optimal dose and the most appropriate candidates for the technique, as it was detrimental in one person and did not improve - or gave minimal improvement to - six other people.

There will also be ongoing ethical considerations for the technique, which currently uses cells left over from in vitro fertilisation (IVF).

Overall, these studies show a promising treatment for two of the commonest causes of visual impairment in the developed world, though it will take a few more years of trials to optimise the technique.

Analysis by Bazian. Edited by NHS Choices. Follow Behind the Headlines on Twitter. Join the Healthy Evidence forum.

Summary

"Embryonic stem cells transplanted into eyes of blind restore sight," The Daily Telegraph reports, covering a study where human stem cells were transplanted into the eyes of people with visual impairment.

Links to Headlines

Embryonic stem cells transplanted into eyes of blind restore sight. The Daily Telegraph, October 15 2014

Stem cell therapy success in treatment of sight loss from macular degeneration. The Guardian, October 15 2014

Stem cell trial cures blindness for many patients - with no side effects. The Independent, October 15 2014

Links to Science

Schwartz SD, Regillo CD, Lam BL, et al. Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt's macular dystrophy: follow-up of two open-label phase 1/2 studies (PDF, 2Mb). The Lancet. Published online October 15 2014

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