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Biologics and Small-Molecule Therapies in Netherton Syndrome: A Comprehensive Review
Shin Morizane1, Tomoyuki Mukai2, Ko Sunagawa1
1Department of Dermatology, Okayama University Graduate School of Medicine, Dentistry and Pharmaceutical Sciences, Okayama, Japan.
The Journal of Dermatology
|September 1, 2025
Summary
Netherton syndrome (NS), a rare genetic skin disorder, involves inflammation and barrier dysfunction. Emerging biologic and small-molecule therapies targeting specific immune pathways show promise for managing this condition.
Area of Science:
- Dermatology and Immunology
- Genetics and Molecular Biology
Background:
- Netherton syndrome (NS) is a rare congenital ichthyosis stemming from SPINK5 gene mutations, impacting LEKTI expression and leading to skin barrier defects and inflammation.
- Patients exhibit distinct immune profiles, often involving IL-17 and IL-36, with variations based on clinical subtypes like ichthyosis linearis circumflexa and scaly erythroderma.
Purpose of the Study:
- To review the current therapeutic landscape for Netherton syndrome, focusing on novel biologic and small-molecule immunomodulators.
- To explore the potential of targeted therapies in addressing the complex immune dysregulation characteristic of NS.
Main Methods:
- Literature review of current clinical management and emerging treatments for Netherton syndrome.
- Analysis of preliminary data on biologics (e.g., dupilumab, secukinumab) and JAK inhibitors (e.g., tofacitinib) in NS treatment.
Main Results:
- Symptomatic treatments remain standard, but targeted therapies are emerging as promising options.
- Preliminary evidence suggests benefits from interventions inhibiting IL-4, IL-13, IL-17, IL-36, and JAK pathways in NS patients.
Conclusions:
- Biologics and small-molecule immunomodulators targeting specific cytokines and signaling pathways offer potential new treatment avenues for Netherton syndrome.
- Further well-designed clinical trials are essential to validate the efficacy and safety of these novel therapies in NS management.
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