Study summary · research use only
Afamelanotide, an agonistic analog of α-melanocyte-stimulating hormone, in dermal phototoxicity of erythropoietic protoporphyria
Plain-language summary
Paraphrased from the published abstract below — not a verdict on whether anything works.
This review discusses afamelanotide, an agonistic analog of α-melanocyte-stimulating hormone (MSH), as a first-in-class therapeutic for dermal phototoxicity in human patients with erythropoietic protoporphyria (PP) and X-linked dominant protoporphyria, conditions marked by sunlight intolerance. It covers the genetics and existing therapy for PP, α-MSH and afamelanotide's physiological and pharmacological actions including receptor-mediated intracellular signaling and effects of receptor polymorphisms, and adverse effects. The review discusses results from afamelanotide trials in PP, and states that early trial results were described by the authors as promising with a risk profile they characterized favorably, while noting an expectation that afamelanotide and related analogs could see future use in other light-related skin conditions.
Abstract
Afamelanotide, an α-melanocyte stimulating hormone (MSH) agonistic analog is a first-in-class therapeutic. Its application to protoporphyria (PP), a disease associated with absolute sunlight-intolerance is discussed. The genetics and existing therapy of the inherited disease PP comprising both erythropoietic protoporphyria and X-linked dominant protoporphyria. The physiological and pharmacological actions of α-MSH and afamelanotide including receptor-mediated intracellular signaling and effects of receptor polymorphisms. Adverse effects and safety issues. The clinical severity and the necessity for an effective therapy for the rare disease PP are illustrated by a short, up-to-date portrait. A condensed description of clinically important aspects of α-MSH signaling, physiological, pharmacological and safety issues of afamelanotide applied to humans and the rational for its potential efficacy in PP are given. The different trials of afamelanotide in PP and their most recent results are discussed. Although early, results of the first trials of afamelanotide for PP are promising and the risk-safety profile appears favorable today. We expect afamelanotide and analogs thereof to be a prospective therapeutic tool in light-related skin diseases, and in future this drug class might prove effectiveness in other medical conditions.
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