In 2006 gene therapy (voretigene neparvovec of Novartis) was the subject of a phase I trial carried out in collaboration between the Vanvitelli University, the Telethon Foundation and the Philadelphia Children Hospital.
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Naples center of excellence – The success of the treatments makes Naples a national reference point for the treatment of rare retinal diseases and, underlines the university, shows the effectiveness of the collaboration between public and private in guaranteeing access to the most innovative therapies to patients who up to recently they had no possibility of a cure.
The ten patients treated – explains Francesca Simonelli, full professor of Ophthalmology and director of the Ophthalmology Clinic of the University of Campania Luigi Vanvitelli – “today they can write, read and move independently. The results we have obtained of enlargement of the visual field, increase of vision at near, far and in low light conditions have a profound scientific and clinical value. And they also testify that, in a degenerative pathology, the way of early treatment is the winning one “.
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In 2019, authorized by Aifa, “we treated the first two children in Italy, and today almost two years after the therapy we can confirm an absolute stability of the results and a good safety profile. These are data that make us confident that what has been achieved in terms of visual capacity
last in the long term “, added the expert.
“An extraordinary result”, says the Rector of the Vanvitelli University, Gianfranco Nicoletti. Ten visually impaired children “have regained their sight, I must thank the Campania Region which has invested in gene therapy and our Ophthalmology for the excellent work done that places us among the
first centers in Europe for treatment in children “.
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Treat a patient with hereditary retinal dystrophy with gene therapy requires an extremely complex preparatory course and administration process that requires a long preparation. The Eye Clinic of the Vanvitelli University of Naples is the first center in Italy to be certified for the administration of gene therapy. “We now have a one-off therapy that allows us to offer real hope to patients and their families,” Professor Simonelli points out.
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