For the first time in Hungary, an innovative gene therapy eye surgery for the long-term treatment of a widespread retinal disease, wet age-related macular degeneration (nAMD), has been performed at the Clinical Center of the University of Debrecen as part of clinical trials. This groundbreaking therapeutic approach could not only preserve vision but also significantly improve patients’ quality of life by reducing the need for continuous treatments.
Understanding Wet Macular Degeneration
Wet macular degeneration (nAMD) is a retinal disease characterized by abnormal blood vessel growth in the area of the retina responsible for sharp vision. It is a condition that primarily affects older adults and, over time, typically involves both eyes. If left untreated, patients may gradually lose their ability to read, recognize faces and objects, and experience a rapid decline in quality of life.
For nearly two decades, the most effective treatment for nAMD has been anti-VEGF therapy, which requires frequent injections of medication directly into the eye.
- “Conventional treatments are effective as long as they are strictly monitored in clinical trials. However, in real-world conditions, treatment sessions often become inconsistent or infrequent, leading to a decline in visual acuity. Moreover, the lifelong need for these treatments and constant check-ups can be burdensome for patients,” explained Dr. Attila Vajas, clinical chief physician at the University of Debrecen’s Department of Ophthalmology, in an interview with hirek.unideb.hu.
A New Approach: Gene Therapy
Dr. Vajas highlighted that gene therapy represents a novel long-term treatment approach. The University of Debrecen’s Department of Ophthalmology is the first institution in Hungary to perform this gene therapy procedure.
- “With this advanced surgical technique, we introduce a viral vector (AAV8 type) into specific retinal layers. The outer shell of the vector retains its ability to enter certain cells along with its carried genetic material. The vector delivers the desired genetic code, which activates within the target cells. The produced protein is an inhibitory molecule (anti-VEGF protein fragment), similar to what is administered via traditional therapies but now generated internally within the eye,” Dr. Vajas explained.
(Image description: The image shows the implantation process. The two small blister-like formations at the top contain the viral vector along with the delivered genetic code.)
Breakthrough Benefits
This new therapy has the potential not only to preserve vision but also to improve patients’ quality of life by significantly reducing the need for frequent treatments.
- “Following gene therapy, the levels of inhibitory molecules inside the eye remain stable, eliminating the risk of vision loss caused by inconsistent treatments. Based on available data, traditional injection-based therapies are no longer necessary or are required only on rare occasions. A previous similar study showed that, after gene therapy, 78% of patients remained injection-free for nine months, while the remaining cases required at most one conventional treatment. Throughout the study, visual acuity remained stable,” emphasized Dr. Vajas.
(Graph description: Each horizontal line represents a patient, and the colored circles indicate the different conventional treatments they received. The vertical line marks the day of the gene therapy procedure. The graph clearly demonstrates the significant reduction in the need for traditional treatments.)
Future Prospects
Dr. Vajas pointed out that gene therapy is currently in the clinical trial phase and is not yet part of routine medical practice. However, if ongoing results continue to show promise, this method could revolutionize the treatment of wet macular degeneration and other serious ophthalmic diseases.
(unideb.hu)
