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Published on: August 24, 2022
[Pharmacological concepts to treat hereditary retinal degenerations]
1Universitäts-Augenklinik Freiburg, Killianstr. 5, 79106, Freiburg, Deutschland. charlotte.poloschek@uniklinik-freiburg.de
Abstract:
This article reviews the current pharmacological strategies to treat inherited retinal degeneration. To date there is no causal therapy despite growing knowledge of the particular pathomechanisms. However, treatment is available for complications, such as cystic macular changes and cystoid macular edema. To reduce retinal thickness systemic or topical carboanhydrase inhibitors can be applied and in rare cases combined with steroids when indicated, however reduction of retinal thickness is not always accompanied by improvement of visual acuity. Regular follow-up with optical coherence tomography is required. In some cases, potentially neuroprotective agents (valproic acid, ciliary neurotrophic factor and Ca(2+ ) channel inhibitors) or food supplementation (vitamin A, lutein, synthetic retinoids and decosahexaenoic acid) may have a positive impact on disease progression (e.g. reduction in progression of visual field loss or individual electrophysiological parameters). However, beneficial effects and side effects, e.g. of vitamin A substitution, depend not only on the disease phenotype (such as retinitis pigmentosa) but also on the actual genotype. Furthermore, no data are available regarding the application of pharmaceuticals in the pediatric population.
Insights
Current pharmacological strategies for inherited retinal degeneration offer symptomatic relief for complications like macular edema but lack causal therapies. Some treatments may slow progression, but efficacy depends on genetic factors and disease type.
Area of Science:
- Ophthalmology
- Medical Genetics
- Pharmacology
Context:
- Inherited retinal degenerations (IRDs) represent a group of genetic disorders leading to progressive vision loss.
- Despite advances in understanding IRD pathomechanisms, causal treatments remain elusive.
- Current management focuses on addressing complications and potentially slowing disease progression.
Purpose:
- To review existing and emerging pharmacological strategies for treating inherited retinal degenerations.
- To evaluate the efficacy and limitations of current treatments for IRDs.
- To highlight the need for genotype-specific and age-appropriate therapeutic approaches.
Summary:
- No causal therapies exist for IRDs, but treatments for complications like cystoid macular edema are available.
- Systemic or topical carboanhydrase inhibitors, sometimes with steroids, can reduce retinal thickness, though visual acuity improvement is not guaranteed.
- Potential neuroprotective agents and nutritional supplements may impact disease progression, but their effectiveness is highly dependent on the specific IRD phenotype and genotype.
- Data on pharmaceutical applications in pediatric populations are lacking.
Impact:
- Provides a comprehensive overview of current treatment options for IRDs, informing clinical practice.
- Identifies gaps in knowledge and therapeutic strategies, guiding future research directions.
- Emphasizes the importance of personalized medicine in IRD treatment based on genetic and phenotypic factors.
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