DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for platelet storage pool deficiency — screening already-approved drugs against its 3-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease modulePlatelet storage pool deficiency maps to a 3-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 platelet storage pool deficiency 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
RUNX family transcription factor 1 (RUNX1) — RUNX1 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 1E50 · 2.6 Å · ligand none (apo structure). Experimental structure, not a prediction.
What the evidence adds up to
The 1970 abstract describes a method for preparing platelet concentrates that can be stored for up to three days at 22°C with adequate viability for transfusion, provided concentrate volume and pH are controlled and agitation is used. Twelve studies in thrombocytopenic recipients suggested the stored concentrates would be safe and effective. No data on platelet storage pool deficiency specifically are given.
A 1989 study evaluated pooled platelet concentrates stored for up to five days using sterile connecting devices. In vitro assays showed acceptable storage values regardless of ABO type; pool pH on day 5 was 6.83 ± 0.3. In vivo, four-unit pools of ABO-compatible random-donor concentrates stored for up to 96 hours were transfused into thrombocytopenic patients with bone marrow failure. The mean one-hour corrected count increment was 11,368 ± 5,824 for pooled stored platelets versus 7,819 ± 5,189 for unpooled controls, a difference that was not statistically significant (p > 0.05). No evidence of mixed lymphocyte reactions was found in the pooling bag during storage. Again, no patients with platelet storage pool deficiency were studied.
A 2025 review article notes that the impacts of donor variability, manufacturing processes, and storage conditions on platelet transfusion efficacy and safety remain incompletely evaluated. It highlights critical gaps in understanding platelet product function in hemostasis, vascular integrity, and thromboinflammation, and states that the impact of manufacturing and storage on these domains is not yet understood. The review calls for improved preclinical models and better evaluation of clinical outcomes associated with the platelet storage lesion.
No abstract provides any data on drug repurposing for platelet storage pool deficiency. What is missing is any clinical trial in patients with that specific diagnosis, any study of drug interventions to improve platelet function or storage in that population, and any funding or trial design aimed at stratifying patients by the underlying molecular defect.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Blood · 1970 · 256 citations · open access
Storage of Platelet Concentrates at 22°C
AbstractAbstract A technique has been proposed which allows easy preparation of platelet concentrates within a closed, sterile system and maximal utilization of other blood components. Lifespan studies have been performed to demonstrate the viability of platelets prepared in this manner. Such concentrates can be stored for as long as three days with adequate maintenance of viability for tranfusion purposes if certain technical requirements are met. Important among these are attention to concentrate volume and pH; agitation of the concentrate is helpful for maintenance of viability. Twelve studies in thrombocytopenic recipients suggest that stored concentrates will be safe and effective in transfusion practice.
AbstractThe use of sterile connecting devices will permit up to 5-day storage of pooled platelet concentrates (PCs). However, there are no data evaluating long-term storage of PCs pooled from multiple donors. Four units of ABO-compatible or -incompatible PCs were pooled and stored in single 300-ml PL-732 storage bags for up to 5 days. Results of in vitro assays showed acceptable storage values regardless of the ABO types in the pool. Pool pH on Day 5 was 6.83 +/- 0.3 (mean +/- 1 SD). The in vitro storage characteristics were comparable to those of unpooled age-matched platelets reported previously from our laboratory. For in vivo studies, 4-unit pools of ABO-compatible random-donor PCs stored for up to 96 hours in 1000-ml PL-732 bags were transfused into patients who were thrombocytopenic due to bone marrow failure, and the correct count increments (CCI) were determined. In vivo results showed a mean 1-hour CCI of 11,368 +/- 5824 for the pooled stored platelets and 7819 +/- 5189 for unpooled controls (p greater than 0.05). To evaluate the possibility that passenger lymphocytes in the concentrates would generate mixed lymphocyte reactions (MLR) in the pooling bag during storage, lymphocytes were studied over 5 days of storage by the use of monoclonal antibodies against activated T-cell markers and by 3H thymidine uptake. Results failed to show evidence of either the generation of activated T-cell markers or the uptake of 3H thymidine.(ABSTRACT TRUNCATED AT 250 WORDS)
A Case of Chronic Thrombocytopenia in a 17-Year-Old Female
AbstractStorage pool deficiency (SPD) is a group of rare platelet disorders that result from deficiencies in α-granules, δ-granules, or both. One type of α-SPD is gray platelet syndrome (GPS), caused by mutations in the neurobeachin-like 2 (NBEAL2) gene that results in a bleeding diathesis, thrombocytopenia, splenomegaly, and progressive myelofibrosis. Due to the lack of α-granules, platelets have a gray and degranulated appearance by light microscopy. However, definitive diagnosis of GPS requires confirmation of α-granule deficiency by electron microscopy. Treatment is nonspecific, with the conservative utilization of platelet transfusions being the most important form of therapy. We present a case of a 17-year-old female with a past medical history of thrombocytopenia, first identified at the age of five. Her clinical symptomatology included chronic fatigue, gingival bleeding, bruising, menorrhagia, and leg pain. This report will discuss both the clinical and the pathophysiologic aspects of this rare platelet disorder.
Leveraging the Multifaceted Platelet Storage Lesion to Advance Precision Platelet Transfusion Medicine
AbstractPlatelet transfusions are a cornerstone of modern medical care, yet the impacts of donor variability, manufacturing processes, and storage conditions on efficacy and safety remain to be comprehensively evaluated. In this review, the key factors contributing to the platelet storage lesion and recent clinical findings on cold-stored platelets are explored. Critical gaps in our understanding of platelet product function in hemostasis and beyond, including the roles of platelets in vascular integrity and thromboinflammation, are also highlighted. The impact of platelet product manufacturing and storage conditions on these domains is not yet understood. Furthermore, the need for improved preclinical models to assess storage-related functional changes is discussed and the importance of evaluating clinical outcomes associated with platelet storage lesions is emphasized. By addressing these challenges, clinicians can refine transfusion strategies through precision medicine and optimize platelet product selection and manufacturing to improve patient outcomes.
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.
