DeCure for Autoimmune lymphoproliferative syndrome type 4
DeCure's autonomous Immuno AI scientist is researching a drug-repurposing hypothesis for autoimmune lymphoproliferative syndrome type 4 — 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 moduleAutoimmune lymphoproliferative syndrome type 4 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 autoimmune lymphoproliferative syndrome type 4 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
NRAS proto-oncogene, GTPase (NRAS) — NRAS 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 gdpdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6ZIO · 1.55 Å · ligand GUANOSINE-5'-DIPHOSPHATE (GDP). Experimental structure, not a prediction.
What the evidence adds up to
Two cousins with autoimmune lymphoproliferative syndrome (ALPS) were described in a 2018 report that discussed the therapeutic potential of low-dose sirolimus. The abstract gives no numbers for response, survival, or sample size beyond the two cousins, and it does not state whether sirolimus was actually administered or what the outcome was. The same year, a separate case report described a 19-year-old girl diagnosed with ALPS after presenting with autoimmune cytopenia, lymphadenopathy, and splenomegaly, with accumulation of double-negative T cells in peripheral blood; no treatment or outcome data are given.
A 2017 review emphasised that ALPS diagnosis follows published criteria and requires excluding infections, autoimmune disorders, and malignancies. It noted that ALPS carries significant morbidity and is best managed through a multidisciplinary approach, but provided no specific treatment results. A 2018 paper on methotrexate-associated lymphoproliferative disorders in patients on immunosuppressants reported sustained regression after simply stopping the immunosuppressive agent, but this is a different condition (drug-induced, not inherited ALPS) and the abstract gives no patient numbers or follow-up duration.
No abstract provides evidence that sirolimus or any other drug changes the course of ALPS type 4. The 2018 sirolimus report is limited to two cousins and does not confirm efficacy. What is still missing is a prospective trial with a defined dose, a control group, and objective endpoints such as reduction in lymphoproliferation or autoimmune cytopenia, as well as genetic stratification to distinguish ALPS subtypes.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Pediatrics International · 2018 · 7 citations · open access
Low‐dose sirolimus in two cousins with autoimmune lymphoproliferative syndrome‐associated infection
AbstractAutoimmune lymphoproliferative syndrome (ALPS) is characterized by non-malignant lymphoproliferation and autoimmunity, with a wide spectrum of clinical manifestations (OMIM 601859). Typical features include enlarged spleen and lymph nodes and autoimmune cytopenia. We describe a family with ALPS in which two cousins independently presented to their physicians with infection and discuss the therapeutic potential of sirolimus.
Allergy and Asthma Proceedings · 2017 · 3 citations
Pearls and pitfalls: Autoimmune lymphoproliferative syndrome and autoimmune lymphoproliferative syndrome‐like disease
AbstractA case of autoimmune lymphoproliferative syndrome (ALPS) was presented, followed by a discussion of the clinical characteristics, pathophysiology, diagnosis, and management of this disease. Clinical pearls and pitfalls are emphasized for the use of the practicing allergist and the fellow in-training. The diagnosis of ALPS was guided by published criteria. A careful history and workup were needed to exclude other possible etiologies for the patient's symptoms and physical findings. ALPS often carries significant morbidity and is best managed through a multidisciplinary approach.
Electronic Journal of General Medicine · 2018 · 3 citations · open access
The lymphoproliferative auto-immune syndrome: a rare cause of peripheral cytopenia
AbstractAutoimmune Lymphoproliferative syndrome is an inherited disorder manifesting with autoimmune cytopenia, lymphadenopathy and splenomegaly. The differential diagnosis includes infections, autoimmune disorders or malignancies. The disease is characterized by accumulation of double negative (CD3+ CD4- CD8-) T cells (DNT) in the peripheral blood. Here we report the case of 19 years-old girl that was diagnosed as autoimmune Lymphoproliferative syndrome with a literature review.
BMJ Case Reports · 2018 · 2 citations · open access
Treatment of advanced stage methotrexate-associated lymphoproliferative disorders (MTX-LPDs) with methotrexate discontinuation
AbstractWe present two cases of patient's with long-standing autoimmune diseases being treated with immunosuppressants that developed aggressive lymphoproliferative disorders. Immunosuppressants have a well-known association with disorders. Sustained regression of these lymphoproliferative disorders occurred with simple discontinuation of these immunosuppressive agents.
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.
DeCure is a research and publication project, not medical advice and not a treatment. "DeCure for X" describes a research goal, not a claim that a cure exists. Backing a cure is a contribution to fund the research — it is not an investment, and confers no yield, royalty, equity or IP ownership. Papers are published open-access by the DeCure.ai DAO.
