DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for desmoplastic small round cell tumor — screening already-approved drugs against its 30-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleDesmoplastic small round cell tumor maps to a 30-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 desmoplastic small round cell tumor 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
neurotrophic receptor tyrosine kinase 3 (NTRK3) — NTRK3 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 4-aminophenyldrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6KZD · 1.708 Å · ligand 3-[2-[6-(4-aminophenyl)imidazo[1,2-a]pyrazin-3-yl]ethynyl]-2-methyl-~{N}-[3-(4-methylpiperazin-1-yl)-5-propan-2-yl-phenyl]benzamide (DZ6). Experimental structure, not a prediction.
What the evidence adds up to
Five young adults with intraabdominal desmoplastic small cell tumour were treated between 1990 and 1995 with aggressive combination chemotherapy using alternating regimens similar to testis cancer salvage therapy. Four of the five showed a partial response. Surgery to remove all residual disease was attempted in two of the responding patients but succeeded in only one. All five patients died from their cancer. The authors concluded that the tumour is sensitive to chemotherapy, that treatment offers palliation and may prolong survival in isolated cases, but that even the most aggressive medical and surgical approaches do not cure and patients ultimately die.
A separate series of five children with diffuse intraabdominal desmoplastic small round cell tumour, managed over a decade with aggressive chemotherapy, surgery, radiotherapy and peripheral blood stem cell transplantation, reported that three patients who had radical excision plus adjuvant chemotherapy recurred within two to six months. The two patients who had initial debulking recurred after two and eighteen months respectively. All but one patient died within three years from diagnosis; the surviving patient was alive eight months after debulking at the time of reporting. The authors stated that multiagent chemotherapy usually leads to temporary regression but recurrence is the rule, and that radical surgery, radiotherapy and stem cell transplantation do not appear to improve prognosis significantly. They described the outcome as dismal and surgical efforts as only palliative.
A 2018 report of 34 cases of desmoplastic small round cell tumours with atypical presentations provided supplementary data but did not report treatment outcomes or survival figures in the available material. No drug names, response rates or survival durations beyond those already described were given in that supplement.
What remains missing is any evidence that current chemotherapy, surgery, radiotherapy or stem cell transplantation can produce durable remissions in this disease. No randomised trial has been conducted, no targeted therapy or immunotherapy has shown activity in a published series, and no biomarker exists to stratify patients by likelihood of response. The rarity of the tumour makes trial recruitment difficult, and no dedicated funding stream for a multi-centre trial is in place.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Cancer · 1996 · 46 citations · open access
Intraabdominal desmoplastic small cell tumor: Report and discussion of five cases
AbstractBACKGROUND: Intraabdominal desmoplastic small cell tumor is a rare malignancy that has only recently been described. In the adult population, it is usually seen in young males. The tumor appears to arise as a dominant mass or masses in the abdominal cavity, particularly in the pelvic region, without a clear visceral origin. Multiple small tumor nodules may be found attached to the peritoneal surface. Visceral metastases occur late in the clinical course, ultimately leading to the patient's death. Five young adults with intraabdominal desmoplastic small cell tumors were treated with aggressive chemotherapy. METHODS: Charts of five patients treated between 1990 and 1995 were reviewed. The diagnosis of desmoplastic small cell tumor was made on the basis of typical clinical and radiographic findings, light microscopy showing hyperchromatic cells surrounded by a desmoplastic stroma, and immunohistochemistry demonstrating markers of epithelial and mesenchymal differentiation. Patients were treated with aggressive combination chemotherapy consisting of alternating regimens similar to those used as testis cancer salvage therapy. RESULTS: Four of the five patients demonstrated a partial response to chemotherapy. Two of the responding patients had surgery with the intent of removing all residual disease, but this was successful in only one case. All patients have died from their cancer. CONCLUSIONS: Intraabdominal desmoplastic small cell tumors are sensitive to combination chemotherapy. It appears that treatment offers good palliation and may prolong survival in isolated cases. However, even the patients managed with the most aggressive medical and surgical approaches are not cured and ultimately die of their disease.
European Journal of Pediatric Surgery · 2006 · 33 citations
Diffuse Intraabdominal Desmoplastic Small Round Cell Tumor: A Ten-Year Experience
AbstractBACKGROUND: Intraabdominal desmoplastic small round cell tumors (IDSRCT) are rare in children and predominantly affect male adolescents and young adults. We present our experience in the management of five children with diffuse IDSRCT, managed with aggressive chemotherapy, surgery, radiotherapy and peripheral blood stem cell transplantation. MATERIAL AND METHODS: During the last decade five patients, four males and one female (mean age 9.6 years), with diffuse IDSRCT were managed in our department. The main symptoms were abdominal distention, vague abdominal pain, and vomiting. Three patients with inoperable tumor on admission were submitted initially to open biopsy followed by aggressive chemotherapy. Regression of the tumor was followed by a second laparotomy and radical excision of any macroscopically distinguishable masses, followed by chemotherapy. In the remaining two patients a debulking procedure was done initially, followed by chemotherapy. The accurate diagnosis of the disease was established by immunohistochemistry, additionally confirmed in the last two patients by molecular analysis. RESULTS: Three patients who had radical excision of the tumor and adjuvant chemotherapy had recurrence after two to six months. In the remaining two patients, recurrence was evident after two and eighteen months, respectively, following debulking. In addition, one patient with recurrence received radiotherapy and two others underwent peripheral blood stem cell transplantation. All but one patient died within three years from diagnosis. The last patient, who was submitted to a debulking procedure, is still alive eight months after the operation. CONCLUSIONS: Intrabdominal desmoplastic small round cell tumor is a highly aggressive malignancy with a very poor prognosis. Multiagent chemotherapy usually leads initially to a temporary regression of the tumor, but recurrence is the rule. Radical surgical excision, radiotherapy and peripheral blood stem cell transplantation does not seem to improve prognosis significantly. Despite all therapeutic modalities the outcome is dismal and surgical efforts can be considered only as palliative.
Sage Journals Data · 2018 · 0 citations · open access
Supplementary_table_1 – Supplemental material for Desmoplastic Small Round Cell Tumors With Atypical Presentations: A Report of 34 Cases
AbstractSupplemental material, Supplementary_table_1 for Desmoplastic Small Round Cell Tumors With Atypical Presentations: A Report of 34 Cases by Alyaa Al-Ibraheemi, Cory Broehm, Munir R. Tanas, Andrew E. Horvai, Brian P. Rubin, Alison L. Cheah, Khin Thway, Cyril Fisher, Armita Bahrami, Andrew L. Folpe and Karen J. Fritchie in International Journal of Surgical Pathology
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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