DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for pityriasis rubra pilaris — screening already-approved drugs against its 2-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease modulePityriasis rubra pilaris maps to a 2-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
No approved-drug candidate for pityriasis rubra pilaris is corroborated in the literature DeepSearch retrieved. Some conditions are managed with non-pharmacological care — a device, surgery or physical therapy — rather than a medicine; that may be the case here, or the literature we found may simply be too sparse yet to support a drug-repurposing angle.
Molecular view
N-sulfoglucosamine sulfohydrolase (SGSH) — SGSH is one of the genes genetically linked to this disease in Open Targets — shown as context, not as a drug target we're pursuing: no approved-drug candidate for this disease is yet corroborated in the literature we found.
Loading structure…
helix sheet apo structuredrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 4MHX · 2.0 Å · ligand none (apo structure). Experimental structure, not a prediction.
What the evidence adds up to
In a 2016 survey of 100 patients with a putative diagnosis of pityriasis rubra pilaris, only 50 met criteria for classic, unquestionable PRP. Among those 50, the median age was 61 years, 46% were female, and only 13 (26%) were correctly diagnosed at initial presentation. Diagnosis was delayed by an average of 29 months, and 27 patients (54%) had undergone two or more biopsies. At enrollment, symptoms had persisted in 36 patients (72%) for an average of 58 months. Comorbidities were present in 31 patients (62%), with hypothyroidism in 20%. Nearly all patients (98%) received some form of therapy; patients cited topical emollients, corticosteroids, salicylic acid, oral retinoids, methotrexate, and tumour necrosis factor inhibitors as most helpful. The authors concluded that PRP remains a challenging diagnosis without established and specific treatment.
A 2018 case series and literature review examined methotrexate treatment in a total of 116 patients. The overall response rate was 65.5%, with complete clearing in 23.3% and excellent improvement in 17.2%. After excluding studies with other concurrent systemic therapies or low reliability, the overall response rate increased to 90.9%, with complete clearing in 40.9% and excellent improvement in 31.8%. Sixteen adverse reactions, of which 11 were mild, were observed in 15 patients (12.9%). The authors concluded that the available literature supports good response rates and safety of methotrexate in PRP, but noted that no randomised controlled trials are available.
A 1978 case report described a 32-year-old man treated with 1% aminonicotinamide cream applied once daily. Improvement was noted within four days, and almost complete clearing occurred after two weeks of therapy. No side-effects were reported. A 1977 study of 93 patients described clinical features and natural history but provided no treatment outcomes or response rates.
What is still missing are randomised controlled trials, prospective data on long-term outcomes, validated diagnostic criteria, and any stratification of patients by disease subtype or biomarker that might predict response to a given drug.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
JAMA Dermatology · 2016 · 86 citations · open access
Epidemiologic, Clinicopathologic, Diagnostic, and Management Challenges of Pityriasis Rubra Pilaris
AbstractIMPORTANCE: Pityriasis rubra pilaris (PRP) is a rare papulosquamous disorder with limited epidemiologic and clinicopathologic data. Little information is available on long-term outcomes, comorbidities, and treatment efficacy. OBJECTIVE: To evaluate objective and subjective disease experience metrics from the perspectives of patients and clinicians. DESIGN, SETTING, AND PARTICIPANTS: One hundred patients with a putative diagnosis of PRP and who elected to participate completed a comprehensive survey, followed by acquisition of their medical records, including histopathology slides and reports. The data were analyzed separately from the health care clinician and the patient perspectives. Two academic dermatologists examined clinical notes, pathology reports, and photographs, confirming diagnoses via predetermined criteria. Patients were categorized into 4 levels of diagnostic certainty to allow stratification of the findings for subgroup analysis. Patients with a diagnosis of PRP were solicited through patient support organization websites. MAIN OUTCOMES AND MEASURES: Clinical outcomes, unexpected association of comorbidities, and efficacy (or lack of it) of various treatment modalities. RESULTS: Among the 100 patients, 50 were diagnosed as having classic, unquestionable PRP. The patients were a median of 61 years old (range, 5-87 years), and 46% were female. Fifty were categorized as level 1 diagnostic certainty, 15 as level 2, 30 as level 3, and 5 as level 4. Of the level 1 patients, 13 (26%) were correctly diagnosed at initial presentation; diagnosis was delayed, on average, by 29 months (range, 0.25-288 months; median, 2 months); and 27 (54%) having undergone 2 or more biopsies. At enrollment, PRP symptoms had persisted in 36 patients (72%) for an average of 58 months (range, 1-300 months; median, 30 months). Thirty-one patients (62%) had comorbidities, including hypothyroidism (20%). Nearly all patients (98%) received some form of therapy. Patients cited topical emollients, corticosteroids, and salicylic acid along with oral retinoids, methotrexate, and tumor necrosis factor inhibitors as most helpful. CONCLUSIONS AND RELEVANCE: Pityriasis rubra pilaris remains a challenging diagnosis without established and specific treatment. Our data highlight new potential avenues for research with therapeutic perspective.
Methotrexate Treatment for Pityriasis Rubra Pilaris: A Case Series and Literature Review
AbstractTreatment recommendations for pityriasis rubra pilaris (PRP) are based solely on case reports and small case series, as to-date no randomized controlled trials are available. We present here a case series of 3 patients and a literature review of 28 studies treating a total of 116 patients, with the aim of providing data regarding efficacy and safety of methotrexate in the treatment of PRP. Methotrexate was effective in our patients; the review showed an overall response rate of 65.5% with complete clearing in 23.3% and excellent improvement in 17.2%, respectively. After excluding studies with other concurrent systemic therapies or low reliability, the overall response rate increased to 90.9%, with complete clearing in 40.9% and excellent improvement in 31.8%, respectively. Sixteen adverse reactions, of which 11 were mild, were observed in 15 patients (12.9%). In conclusion, the available literature supports good response rates and safety of methotrexate in PRP.
Pityriasis Rubra Pilaris Responding to Aminonicotinamide
AbstractA 32-year-old man with pityriasis rubra pilaris diagnosed by clinical appearance and histologic examination was treated with 1% aminonicotinamide cream applied once daily. Improvement was noted within four days, and almost complete clearing occurred after two weeks of therapy. No side-effects were noted.
British Journal of Dermatology · 1977 · 16 citations
Pityriasis rubra pilaris: clinical features and natural history in a study of 93 patients
AbstractJournal Article Pityriasis rubra pilaris: clinical features and natural history in a study of 93 patients Get access W.A.D. Griffiths W.A.D. Griffiths Department of Dermatology, University of Liverpool Search for other works by this author on: Oxford Academic Google Scholar British Journal of Dermatology, Volume 97, Issue s15, 1 December 1977, Page 18, https://doi.org/10.1111/j.1365-2133.1977.tb14302.x Published: 01 December 1977
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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