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Updated: Nov 29, 2025
Toxicity Screens in Human Retinal Organoids for Pharmaceutical Discovery
Published on: March 4, 2021
[Toxic retinopathies]
Ulrich Kellner1,2, Simone Kellner3,4, Silke Weinitz3,4
1Zentrum für seltene Netzhauterkrankungen, AugenZentrum Siegburg, MVZ Augenärztliches Diagnostik- und Therapiecentrum Siegburg GmbH, Europaplatz 3, 53721, Siegburg, Deutschland. kellneru@mac.com.
Abstract:
Toxic retinopathies are most frequently induced by external stimulants (e.g. nicotine, poppers, methanol) and are less frequently undesired side effects of systemic drugs (e.g. hydroxychloroquine, ethambutol, MEK, ERK, FLT3 or checkpoint inhibitors, didanosine, pentosan polysulfate sodium) or intravitreally applied drugs. The clinical symptoms of undesired side effects of drugs are often similar to retinal diseases from other causes, which interferes with the recognition of the undesired side effects of drugs. Clinical findings, pathophysiological mechanisms and if advisable strategies for screening are discussed. The focus is on the presentation of confirmed undesirable side effects with established associations for medications which have long been approved. For novel medications, in addition potential but not proven associations are presented to facilitate the recognition of additional cases with side effects for these medications.
Insights
Drug-induced toxic retinopathies, often mimicking other retinal diseases, can arise from systemic or intravitreal medications. Early recognition is crucial for managing these adverse effects, especially with novel drug therapies.
Area of Science:
- Ophthalmology
- Toxicology
- Pharmacology
Background:
- Toxic retinopathies are frequently caused by external agents like nicotine and methanol.
- Adverse drug reactions (ADRs) from systemic or intravitreal medications can also induce toxic retinopathies.
- Clinical presentation of drug-induced retinopathy often overlaps with other retinal diseases, complicating diagnosis.
Purpose of the Study:
- To review clinical findings and pathophysiological mechanisms of drug-induced toxic retinopathies.
- To discuss screening strategies for identifying adverse drug effects on the retina.
- To highlight confirmed associations for approved medications and potential links for novel drugs.
Main Methods:
- Literature review focusing on established and potential drug-induced retinopathies.
- Analysis of clinical presentations and known etiological agents.
- Discussion of diagnostic challenges and screening recommendations.
Main Results:
- External stimulants are common causes of toxic retinopathy.
- Approved systemic drugs (e.g., hydroxychloroquine, ethambutol) and intravitreal drugs are associated with retinopathy.
- Novel medications may also present potential, though unproven, associations with retinopathy.
Conclusions:
- Recognizing drug-induced toxic retinopathies requires understanding their varied clinical presentations.
- Distinguishing ADRs from other retinal diseases is critical for timely intervention.
- Awareness of both established and potential drug associations aids in early detection and management.
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