Ongoing Research and Future Outlook
Medications and Novel Therapies
Geographic atrophy (GA) currently does not have a cure. However, therapeutic approaches such as complement system inhibition, gene therapy and cell-based therapies offer potential in slowing disease progression and improving patient outcomes. Currently, only two medications are approved for the treatment of GA – pegcetacoplan and avacincaptad pegol. Both target the complement system, with pegcetacoplan inhibiting C3 activation, and avacincaptad pegol inhibiting C5 activation (for more details, including the data that supports their use in GA, see Treatment Options and Therapies).
Several novel therapies are under investigation for the treatment of GA, including additional complement inhibitors, gene therapies and cell-based therapies; just a few will be mentioned here – for more information, see Treatment Options and Therapies.Danicopan (ALXN2040, AstraZeneca) is a complement factor D inhibitor, currently being investigated as monotherapy for…
To continue reading
Log in or register to continue reading. It's free!
OR
By signing up to create an account, I accept Healio's Terms of Use and Privacy Policy.
Medications and Novel Therapies
Geographic atrophy (GA) currently does not have a cure. However, therapeutic approaches such as complement system inhibition, gene therapy and cell-based therapies offer potential in slowing disease progression and improving patient outcomes. Currently, only two medications are approved for the treatment of GA – pegcetacoplan and avacincaptad pegol. Both target the complement system, with pegcetacoplan inhibiting C3 activation, and avacincaptad pegol inhibiting C5 activation (for more details, including the data that supports their use in GA, see Treatment Options and Therapies).
Several novel therapies are under investigation for the treatment of GA, including additional complement inhibitors, gene therapies and cell-based therapies; just a few will be mentioned here – for more information, see Treatment Options and Therapies. Danicopan (ALXN2040, AstraZeneca) is a complement factor D inhibitor, currently being investigated as monotherapy for GA in a phase 2 trial. Gene therapy may allow for gene replacement or durable expression of therapeutic proteins to treat GA. One such gene therapy, OCU410 (Ocugen), has been developed for treating dry age-related macular degeneration. The efficacy and safety of OCU410 is being investigated in a phase 1/2 study. Cell-based therapies are another promising approach GA treatment, aiming to replace degenerated retinal pigment epithelium (RPE) cells or introduce cells that release factors supporting photoreceptor survival and function. Several ongoing phase 1/2 studies are exploring the potential of these therapies. One study focuses on a composite subretinal implant known as California Project to Cure Blindness—Retinal Pigment Epithelium 1. Another study is investigating OpRegen (Lineage Cell Therapeutics), a cell-based product composed of RPE cells derived from human embryonic stem cells, which may support the remaining retinal cells in the atrophic area by counteracting RPE cell loss and dysfunction. Another example is RPESC-RPE-4W cells, which are derived from allogeneic RPE stem cells isolated from human cadaveric eyes, and which are currently being investigated in a phase 2 trial.
Beyond therapeutic options, advances in imaging biomarkers and genomic technologies are enabling earlier detection and a better prognosis for GA. Identifying genes linked to GA development, such as CFH, HTRA1 or ARMS2, can help identify patients at higher risk or those who might respond better to certain treatments. Advanced imaging tools like fundus autofluorescence and optical coherence tomography provide clearer and more detailed pictures of the eye, helping health care providers keep track of GA progression over time. Additionally, AI is being used to analyze these images, predict how the disease might develop and even determine which treatments might be best for each person. Combining all of these tools is expected to enhance early diagnosis and monitoring, making personalized approaches possible and further improving the clinical outlook for patients with GA.
References
- Abdelsalam A, Del Priore L, Zarbin MA. Drusen in age-related macular degeneration: pathogenesis, natural course, and laser photocoagulation-induced regression. Surv Ophthalmol. 1999;44(1):1-29.
- Age-Related Eye Disease Study 2 Research Group. Lutein + zeaxanthin and omega-3 fatty acids for age-related macular degeneration: the Age-Related Eye Disease Study 2 (AREDS2) randomized clinical trial. JAMA. 2013;309(19):2005-2015.
