DeCure's autonomous Dermatology AI scientist is researching a drug-repurposing hypothesis for ichthyosis — screening already-approved drugs against its 19-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleIchthyosis maps to a 19-gene Open Targets module — the target space DeCure's AI scientist screens approved drugs against.
DeCure.ai methodSignature reversal (LINCS) plus network proximity (STRING) rank already-approved drugs likely to perturb this module — the same engine that produces DeCure.ai's repurposing hypotheses.
Repurposing thesisScreening approved medicines against this disease module, then publishing the evidence for the strongest candidate. Known pharmacology and human exposure data make the first question sharper — they do not establish safety or efficacy in a new indication.
Research record
01
ResearchComing soon
Candidate research + dossier — target rationale, drug-repurposing thesis and evidence pack.proof: Published dossier + on-chain hash
02
ValidationComing soon
In-vitro biological validation at a contract research org (CRO).proof: CRO contract + in-vitro report
03
Peer review & paperComing soon
Peer-reviewed paper published open-access (preprint + journal).proof: DOI + open-access link + on-chain hash
Current lead
approvedAcitretinApproved drugapprovedIsotretinoinApproved drug
Structures already discussed alongside ichthyosis in the retrieved literature, rendered from public PubChem SMILES. Which drugs appear here reflects the evidence found, not a ranked prediction.
Molecular view
ST14 transmembrane serine protease matriptase (ST14) — ST14 is one of the genes in this disease's Open Targets module — part of the target space DeCure's repurposing candidates point at. The protein backbone is drawn as a cartoon. The structure has phenyl (4-carbamimidoylbenzyl)phosphonate bound in it, shown as sticks.
Loading structure…
helix sheet 4-carbamimidoylbenzyldrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 3NCL · 1.19 Å · ligand phenyl (4-carbamimidoylbenzyl)phosphonate (CCZ). Experimental structure, not a prediction.
What the evidence adds up to
The ichthyoses are a group of rare genetic diseases with a wide phenotypic spectrum, characterised most often by generalised hyperkeratosis and scaling with variable erythema. The highly visible scaling and frequent itch contribute to decreased quality of life. Ichthyoses follow patterns of Mendelian inheritance and cause symptoms from birth or shortly thereafter. Clinically they are divided into non-syndromic ichthyoses, where symptoms are caused exclusively by the epidermal barrier defect, and syndromic ichthyoses, where the causal gene also has extracutaneous functions that produce manifestations in other organs. All represent a disruption of the barrier formed during epidermal differentiation.
Management for ichthyosis focuses on symptomatic relief and scale reduction with emollients, keratolytics, and retinoids. A 1982 review noted that from the patient's point of view treatment is far from satisfactory, and that temporary improvement is relatively easy to achieve but treatment is often messy and unappealing, leading patients to abandon it altogether. That review based treatment on three main hypotheses for pathogenetic mechanisms acting in the ichthyotic disorders.
Recent advances in immune profiling and genotype-phenotype mapping have increased understanding of ichthyosis and shifted focus to pathogenesis-based targeted therapies with emerging biologics, small molecular inhibitors, and gene therapy. Knowledge of molecular mechanisms has improved dramatically over the past few years, and most causal genes are now known, along with the functions of the encoded proteins and their impact on skin barrier formation. No concrete survival or response rates from clinical trials of these emerging agents are reported in the provided abstracts.
What is still missing is evidence from completed clinical trials of the emerging biologics, small molecular inhibitors, and gene therapies — the abstracts only describe them as promising or emerging. No trial design, patient stratification strategy, or funding commitment for such trials is mentioned.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Current Opinion in Pediatrics · 2023 · 6 citations
Ichthyosis: presentation and management
AbstractPURPOSE OF REVIEW: This review focuses on the presentation and management of ichthyoses and highlights recent advances in treatment that hold promise for better targeted therapy. RECENT FINDINGS: The ichthyoses are a group of rare genetic diseases with a wide phenotypic spectrum, characterized most often by generalized hyperkeratosis and scaling with variable erythema. The highly visible scaling and frequent itch contribute to decreased quality of life. Management for ichthyosis focuses on symptomatic relief and scale reduction with emollients, keratolytics, and retinoids. Recent advances in immune profiling and genotype-phenotype mapping have increased understanding of ichthyosis and shifted focus to pathogenesis-based targeted therapies with emerging biologics, small molecular inhibitors, and gene therapy. SUMMARY: This article discusses clinical assessment and genotyping to make the diagnosis of specific forms of ichthyosis, provides guidance for management, and reviews new treatment options with systemic agents.
The Internet Journal of Pediatrics and Neonatology · 2008 · 5 citations
Treatment Of Congenital Ichthyosis With Acitretin
AbstractWe assessed the clinical efficacy, tolerability and safety of acitretin in a patient with ichthyosis. A newborn infant with ichthyosis who presented at birth with collodion baby appearance, was treated with acitretin. A moderate response to acitretin therapy (1 mg/kg/day) administered for 6 months was observed, with improvement in cutaneous lesions. Clinical improvement was achieved shortly after treatment. The treatment resulted in a satisfactory improvement in the skin condition of the case. The tolerance to the drug was good. Side effects were not observed. The oral acitretin treatment is efficient in severe congenital ichthyosis.
AbstractA multicenter study of the effectiveness of 13-cis-retinoic acid (isotretinoin) in lamellar ichthyosis and epidermolytic hyperkeratosis has been conducted. A dose of the drug which produced maximum clearing with minimum side effects was chosen; this varied among different patients, the mean dose being about 2 mg/kg/day. Almost all of the patients in both groups were clearly improved, as evaluated both by the physicians and the patients. The degree of improvement seemed higher in the group of patients with lamellar ichthyosis.
Clinical and Experimental Dermatology · 1982 · 1 citations
Ichthyosis and dry skin*
AbstractIn this short review I have endeavoured to be highly selective and have included only those aspects of the subject which can be regarded as advances or growing points. In many instances the wealth of data appearing are scientifically exciting and offer the prospect of major steps forward in the management of ichthyosis. The treatment of ichthyosis from the patient's point of view is far from satisfactory (Editorial, 1978). Temporary improvement in the ichthyotic disorders is relatively easy to achieve, but so often treatment is messy and unappealing to the patient who will frequently abandon treatment altogether. Treatment is based upon the three main hypotheses for the pathogenetic mechanisms acting in the ichthyotic disorders.
Actas Dermo-Sifiliográficas · 2025 · 0 citations · open access
ICTIOSIS: Actualización clínica y molecular. Parte 1: introducción e ictiosis no sindrómicas
AbstractLas ictiosis son un grupo heterogéneo de enfermedades que comparten síntomas y un mismo mecanismo etiopatogénico. Desde el punto de vista clínico estas enfermedades se caracterizan por la presencia de eritema y diferentes grados de engrosamiento y descamación cutáneas. Aunque el área afectada, la gravedad y el sustrato molecular son muy variables, todas ellas representan la manifestación de una disrupción de la barrera que se forma durante el proceso de diferenciación epidérmica. Las ictiosis siguen patrones de herencia Mendeliana y causan síntomas desde el nacimiento o poco tiempo después. Desde el punto de vista clínico se dividen en ictiosis no sindrómicas (cuando los síntomas están causados únicamente por el defecto de la barrera epidérmica) e ictiosis sindrómicas (cuando el gen causal también tiene funciones extracutáneas que determinan manifestaciones en otros órganos). El conocimiento de las bases moleculares ha avanzado extraordinariamente en los últimos años, y conocemos no sólo la mayoría de los genes que las ocasionan, sino la función de las proteínas que codifican y el impacto en la formación de la barrera cutánea. En la primera parte de este trabajo hacemos una introducción a la fisiopatología de las ictiosis, así como una actualización clínica y genética de las entidades no sindrómicas, tanto las incluidas en la última clasificación consenso como otras que se han caracterizado clínica y/o molecularmente a lo largo de los últimos años. Ichthyoses are a heterogeneous group of diseases sharing symptoms and a common etiopathogenic mechanism. Clinically, these diseases are characterized by the presence of erythema and variable degrees of skin thickening and desquamation. Although the affected area, severity, and molecular substrate are very variable, they are all signs of a disruption of the barrier formed during epidermal differentiation. Ichthyoses follow patterns of Mendelian inheritance and present symptoms since birth or shortly thereafter. Clinically, they can be categorized into non-syndromic (when symptoms are caused exclusively by the epidermal barrier dysfunction) and syndromic ichthyoses (when the causal gene has extracutaneous functions impacting other organs).Knowledge of molecular mechanisms has improved dramatically over the past few years, and we currently know not only most causal genes, but also the functions of the encoded proteins and their impact on skin barrier formation. In the first part of this review, we’ll be introducing ichthyosis physiopathology, along with a clinical and genetic update of non-syndromic entities (those included in the consensus classification and those clinically and/or molecularly characterized since then).
Actas Dermo-Sifiliográficas · 2025 · 0 citations · open access
[Translated article] ICHTHYOSIS: Clinical and Molecular Update. Part 1: Introduction and Non-Syndromic Ichthyoses
AbstractLas ictiosis son un grupo heterogéneo de enfermedades que comparten síntomas y un mismo mecanismo etiopatogénico. Desde el punto de vista clínico estas enfermedades se caracterizan por la presencia de eritema y diferentes grados de engrosamiento y descamación cutáneas. Aunque el área afectada, la gravedad y el sustrato molecular son muy variables, todas ellas representan la manifestación de una disrupción de la barrera que se forma durante el proceso de diferenciación epidérmica. Las ictiosis siguen patrones de herencia Mendeliana y causan síntomas desde el nacimiento o poco tiempo después. Desde el punto de vista clínico se dividen en ictiosis no sindrómicas (cuando los síntomas están causados únicamente por el defecto de la barrera epidérmica) e ictiosis sindrómicas (cuando el gen causal también tiene funciones extracutáneas que determinan manifestaciones en otros órganos). El conocimiento de las bases moleculares ha avanzado extraordinariamente en los últimos años, y conocemos no sólo la mayoría de los genes que las ocasionan, sino la función de las proteínas que codifican y el impacto en la formación de la barrera cutánea. En la primera parte de este trabajo hacemos una introducción a la fisiopatología de las ictiosis, así como una actualización clínica y genética de las entidades no sindrómicas, tanto las incluidas en la última clasificación consenso como otras que se han caracterizado clínica y/o molecularmente a lo largo de los últimos años. Ichthyoses are a heterogeneous group of diseases sharing symptoms and a common etiopathogenic mechanism. Clinically, these diseases are characterized by the presence of erythema and variable degrees of skin thickening and desquamation. Although the affected area, severity, and molecular substrate are very variable, they are all signs of a disruption of the barrier formed during epidermal differentiation. Ichthyoses follow patterns of Mendelian inheritance and present symptoms since birth or shortly thereafter. Clinically, they can be categorized into non-syndromic (when symptoms are caused exclusively by the epidermal barrier dysfunction) and syndromic ichthyoses (when the causal gene has extracutaneous functions impacting other organs).Knowledge of molecular mechanisms has improved dramatically over the past few years, and we currently know not only most causal genes, but also the functions of the encoded proteins and their impact on skin barrier formation. In the first part of this review, we’ll be introducing ichthyosis physiopathology, along with a clinical and genetic update of non-syndromic entities (those included in the consensus classification and those clinically and/or molecularly characterized since then).
Disease module: DeepOracle (Open Targets). Structures: RDKit from PubChem SMILES. Literature: retrieved by DeepSearch across 234,678,978 indexed works (targeted per-candidate search), resolved on OpenAlex.
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