A large-scale, collaborative, systems biology approach is needed to expedite the discovery of treatments for dry AMD—a leading cause of blindness among people 65 and older for which is there is no treatment—according to a report by a working group of scientists appointed by the National Advisory Eye Council (NAEC).
Further understanding risk factors
There are multiple genetic and environmental factors at play in the development of AMD. More than 40 genetic variants have been associated with AMD, including APOE, CHF, and HTRA/ARMS2. The discovery of these gene variants have helped identify potential therapeutic targets, and gene therapy clinical trials are currently underway for the treatment of the wet subtype.3,4
“We propose that researchers utilize a systems biology approach, integrating the big data available from clinical registries and various fields of biology known as ‘omics’ to develop better models and ultimately treatments for patients with this blinding disease,” said report co-authorJoan W. Miller, MD, chairman, Harvard Medical School Department of Ophthalmology, Boston.
“This approach would integrate basic, genomic, pre-clinical, medical, pharmacological, and clinical data into mathematical models of pathological processes at different stages of dry AMD in order to ask how relevant individual components act together within the living system,” Dr. Miller said.
1. Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. The Lancet Global Health;2:e106-e116.
2. Handa JT, Bowes Rickman C, Dick AD, et al. A systems biology approach towards understanding and treating non-neovascular age-related macular degeneration. Nature communications 2019;10:3347.
3. REGENXBIO. REGENXBIO Announces Additional Positive Interim Phase I/IIa Trial Update for RGX-314 for the Treatment of Wet AMD at the American Academy of Ophthalmology 2019 Annual Meeting, 2019.
4. Grishanin R, Vuillemenot B, Sharma P, et al. Preclinical Evaluation of ADVM-022, a Novel Gene Therapy Approach to Treating Wet Age-Related Macular Degeneration. Molecular therapy : the journal of the American Society of Gene Therapy 2019;27:118-129.