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Stem cell breakthrough restores sight in world-first treatment

Reprogrammed cells from healthy donors give hope to patients with severe corneal damage

08-Nov-2024

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In a ground-breaking study published in The Lancet, a new stem-cell therapy has successfully restored vision in people with severe corneal damage, offering hope to those suffering from limbal stem-cell deficiency (LSCD), a condition that leads to corneal scarring and blindness. Four participants, aged 39 to 72, received corneal transplants made from reprogrammed stem cells — a world-first treatment. Three of the participants experienced significant and lasting improvements in their vision over a year, while one showed initial gains that did not persist. This approach involved using induced pluripotent stem (iPS) cells derived from healthy donor blood, which were reprogrammed into a stem-cell-like state and transformed into corneal epithelial cells.

The procedure, led by ophthalmologist Kohji Nishida from Osaka University, involved removing scar tissue from the damaged corneas and transplanting the newly generated epithelial cells. Remarkably, two years after the transplants, none of the participants experienced severe side effects or immune rejection, even without the use of immunosuppressant drugs. This success is particularly notable as iPS cell therapies carry risks of tumour formation, but the patients’ grafts showed no such complications. The researchers caution that more trials are needed to confirm the safety and long-term effectiveness of this treatment.

Kapil Bharti, a stem-cell researcher at the US National Eye Institute, praised the results, describing them as "exciting." He emphasized the need for further research to ensure the treatment’s safety. Nishida and his team plan to launch clinical trials in March to assess the therapy’s efficacy further. This study, along with other global iPS-cell-based trials for eye diseases, suggests that stem-cell therapies could play a major role in treating vision impairment in the future.

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Kohji Nishida

Professor of ophthalmology at Osaka University

Osaka University

Public Research university.

The Lancet

Medical journal covering general medicine

Topics mentioned on this page:
Stem Cells, Vision (health)
Stem cell breakthrough restores sight in world-first treatment