DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for poikiloderma with neutropenia — screening already-approved drugs against its 1-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease modulePoikiloderma with neutropenia maps to a 1-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 poikiloderma with neutropenia 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
U6 snRNA biogenesis phosphodiesterase 1 (USB1) — USB1 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 ampdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6D31 · 1.2 Å · ligand ADENOSINE MONOPHOSPHATE (AMP). Experimental structure, not a prediction.
What the evidence adds up to
A 1931 report noted that poikiloderma-like symptoms can appear in many chronic inflammatory skin diseases, and that some authorities doubted whether poikiloderma atrophicans vasculare was a separate disease at all. A 2012 case report described a 37-year-old man with dermatomyositis and pruritic poikiloderma who was treated with oral dapsone. The authors stated that treatment of poikiloderma in dermatomyositis is usually difficult, and they reported that this patient’s condition was successfully treated. No sample size, response rate, or survival data were given.
A 2021 case report described a patient with confirmed poikiloderma with neutropenia, a rare autosomal recessive condition, who had chronic bilateral lower limb cellulitis and associated osteomyelitis that did not respond to extensive antibiotic regimens. The patient was treated with hyperbaric oxygen therapy, and the authors reported that this treatment was successful. They noted that no previous report had described hyperbaric oxygen therapy for recurrent cellulitis in this patient population. No numbers of patients, response rates, or survival data were provided.
The 2012 and 2021 reports are single cases, so no generalisable efficacy can be drawn. The 1931 paper does not address poikiloderma with neutropenia at all. No controlled trials, no randomised data, and no validated biomarkers exist for this disease. What is missing is any prospective trial design, any patient stratification, and any funding for systematic investigation of treatments in poikiloderma with neutropenia.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Archives of Dermatology · 1936 · 24 citations
MYCOSIS FUNGOIDES WITH POIKILODERMA-LIKE SYMPTOMS
AbstractWithin the last few years increasing attention has been given the cutaneous picture poikiloderma atrophicans vasculare. A number of articles on this subject have appeared in the foreign journals, and published transactions of proceedings of dermatologic societies in this country show a number of cases presented with that diagnosis. Among authorities there is considerable difference of opinion as to what symptoms represent the picture of poikiloderma atrophicans vasculare. Some authors doubt whether it is a separate disease, while others believe firmly that it is a definite clinical entity with a clearcut symptomatology. It is not generally known that poikiloderma-like symptoms may be part of the picture of many chronic inflammatory dermatoses, and this probably is the reason why so many patients are now being shown with poikiloderma as the diagnosis. Dr. Otto H. Foerster, in 1931, first called attention to this fact. The following report deals
European Journal of Dermatology · 2012 · 10 citations
Pruritic poikilodermatous eruption associated with dermatomyositis: successful treatment with dapsone
Abstractejd.2012.1669 Auteur(s) : Yasuhiro Kawachi [email protected], Yasuhiro Fujisawa, Junichi Furuta, Yasuhiro Nakamura, Yoshiyuki Ishii, Fujio Otsuka Department of Dermatology, University of Tsukuba, 1-1-1, Ten-nodai Tsukuba, Ibaraki 305-8575, Japan Treatment of poikiloderma in patients with dermatomyositis is usually difficult. We report a case of poikiloderma associated with dermatomyositis with severe pruritus, which was successfully treated with oral dapsone. A 37-year-old man developed [...]
American Journal of Medical Genetics Part A · 2021 · 3 citations
Hyperbaric oxygen management of recurrent cellulitis in poikiloderma with neutropenia
AbstractPoikiloderma with neutropenia (PN), is a rare autosomal recessive condition with many associated complications and manifestations. Here we present a patient with confirmed PN who is of one-quarter Chucktaw or Cherokee heritage with no known descent from the Navajo tribe. The patient's condition was complicated by chronic bilateral lower limb cellulitis and associated osteomyelitis which was unresponsive to extensive antibiotic regimens. Subsequent treatment with hyperbaric oxygen therapy (HBOT) was successful. To date, no author has reported on the treatment of recurrent cellulitis using HBOT in this patient population. Based on our experience, HBOT should be considered in patients with PN.
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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