A recent international study has shown that lenadogene nolparvovec, a gene therapy based on adeno-associated virus (AAV), can provide long-lasting visual improvements for patients with Leber hereditary optic neuropathy (LHON). This genetic condition, caused by a mutation in the MT-ND4 gene, typically leads to severe vision loss. The study followed patients who received the therapy and found significant improvements in vision even five years after treatment.
The therapy involves a single injection of the gene therapy into one eye, with the other eye receiving a sham injection. Remarkably, patients showed improvements in both eyes, even though only one was treated. This suggests a possible bilateral effect, where the treatment in one eye could help the other, an outcome that was previously hypothesized by researchers. These findings offer hope for better treatment of LHON, a condition with few effective options.
The study measured visual acuity using standardized tests and found that patients’ vision significantly improved. The results were consistent over the five-year period, with many participants showing improvements of three or more lines on the vision chart. In addition to visual improvements, patients also reported enhanced quality of life, with notable gains in areas like mental health and daily functioning.
While the therapy showed promising results, some patients experienced mild side effects, including cataracts and intraocular inflammation. However, these side effects were less frequent after the first two years of treatment. Overall, the results from this study suggest that lenadogene nolparvovec is a promising option for treating LHON, providing lasting benefits for patients and offering hope for future treatments of similar genetic conditions. Further studies will be needed to understand its full potential, especially in younger patients.
Created: Dec 27th, 2024
Citations:
Jackson, J. (2024, December 27). Sustained visual improvements in LHON patients treated with AAV gene therapy. Medical Xpress. https://medicalxpress.com/news/2024-12-sustained-visual-lhon-patients-aav.html
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