You are hereOptiStem scientists set gold standard for translational research

OptiStem scientists set gold standard for translational research

By Emma Kemp - Posted on 31 August 2010

A study involving 112 patients has shown that stem cells grown in the laboratory can be used to restore sight after certain kinds of eye damage.

In research published in the New England Journal of Medicine, scientists from the University of Modena and Reggio Emilia and the San Raffaele Scientific Institute in Italy grew stem cells in the laboratory then grafted them onto patients' eyes to restore their sight.

The eyeball is covered by a layer called the cornea, which is essential for sight. The cornea is usually maintained and repaired by stem cells called limbal stem cells, found in an area at the edge of the eye. However, severe damage to the eye by an injury such as a chemical burn can destroy both the cornea and the limbal stem cells, leaving patients blind.

Graziella Pellegrini, Michele De Luca, Paolo Rama and their colleagues took a small number of limbal stem cells from the healthy eye of 112 patients with severe burns to their other eye. The researchers then cultivated the limbal stem cells in the lab to grow a larger number of cells. They then grafted these stem cells onto the patient’s damaged eye.

The results are exciting. Patients were carefully monitored after the treatment, some for as long as 10 years. Three quarters of them not only regained their sight, but were found to have developed a healthy new cornea that is naturally maintained in the long term – just as it is in an undamaged eye.

This initial study provides an important step towards stem cell therapies, both for blindness and for other diseases. There is still work to be done to make this treatment widely available. Nevertheless, the work demonstrates an important principle. The researchers used knowledge of one kind of stem cell  –  a type found in the skin – to help them develop methods for growing the limbal stem cells they needed to repair the eye. This approach enabled them to develop and carefully test a new kind of treatment. In an article published in the influential journal Nature, Elena Ezhkova and Elaine Fuchs of the Howard Hughes Medical Institute in New York, USA, praised the research:

‘Their paper sets the gold standard for the level of scientific proof that is needed for each new stem-cell therapy, and provides a blueprint that can be applied to the development of other adult stem cells for clinical therapies.’


Related links

Elena Ezhkova and Elaine Fuchs, An eye to treating blindness, Nature, 466, 567–568 (29 July 2010), doi:10.1038/466567

Rama et al, Limbal Stem-Cell Therapy and Long-Term Corneal Regeneration, N Engl J Med, 2010, 363:147-55, doi: 10.1056/NEJMoa0905955



This research was supported by grants from the Italian Ministry for Education, University and Research and Ministry of Health; l’Association Française contre les Myopathies et les Maladies Neuromuscu-laires (AFM) Telethon; the European Commission Sixth Framework Program for Research and Technological Development (corneal engineering) and Seventh Framework Program; Optimization of Stem Cell Therapy for Degenerative Epithelial and Muscle Diseases (OptiStem, HEALTH-F5-2009-223098); Regione Emilia–Romagna (area 1b, medicina rigenerativa); the Italian Ministry of Health; Ricerca finalizzata nazionale 'Diagnosi e trattamento di disabilità visiva dovuta a deficit limbare bilaterale totale'.