Gene Therapy Trial Shows Promise for AMD Treatment
Researchers at UC Davis Health have conducted a successful gene therapy trial for the treatment of wet age-related macular degeneration (AMD). Wet AMD is a leading cause of vision loss in older adults and affects millions of people worldwide.
The trial utilized an experimental therapy called ABBV-RGX-314, which aims to reduce the need for frequent injections by allowing the eye to continuously produce the necessary medicine on its own. This approach could potentially maintain vision while reducing the burden of treatment for patients.
Dr. Glenn Yiu, the principal investigator for the clinical trial, explained that the current treatments for wet AMD often involve lifelong injections that can be as frequent as every month. The gene therapy solution, if approved, could significantly reduce the number of injections required and provide a more sustainable treatment option.
In wet AMD, abnormal blood vessels grow underneath the retina, leading to bleeding or fluid leakage in the eye and causing vision loss. The growth of these blood vessels is primarily driven by a growth factor called vascular endothelial growth factor (VEGF).
The gene therapy approach differs from other treatments for eye diseases, such as stem cell therapies. Instead of injecting cells with regenerative capabilities, gene therapy uses a viral vector to deliver specific genetic instructions for making a protein. In the case of ABBV-RGX-314, the therapy contains instructions for producing anti-VEGF proteins.
The experimental gene therapy procedure involves a more complex surgical technique compared to traditional injections. It includes a vitrectomy, where the gel in the eye is removed and replaced with a saline infusion. The gene therapy solution is then injected underneath the retina.
Although the therapy is still under investigation and has not yet been approved by the FDA, the initial trial results show promise for its efficacy and safety. Dr. Yiu and his team at UC Davis Health are closely monitoring the patient who received the gene therapy to assess the need for future anti-VEGF injections.
The success of this gene therapy trial is significant for patients with wet AMD. UC Davis Health is one of 93 sites across the United States participating in the clinical trial, and their enrollment of three patients so far demonstrates the potential benefits of this approach.
Dr. Paul Sieving, a professor of ophthalmology at UC Davis Health, emphasized the importance of this research and its potential to reduce the frequency of eye injections for wet AMD. The Center for Ocular Regenerative Therapy at UC Davis Health, established by Dr. Sieving, aims to explore therapies like gene therapy for eye diseases.
In conclusion, the successful gene therapy trial conducted at UC Davis Health holds promise for the treatment of wet age-related macular degeneration. If approved, this innovative approach could provide a more sustainable and effective treatment option for patients, reducing the need for frequent injections and potentially maintaining or improving vision. More research and clinical trials are needed to establish the long-term efficacy and safety of this experimental therapy.
