DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for angiomyolipoma — 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 moduleAngiomyolipoma 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
approvedEverolimusApproved drug
Structures already discussed alongside angiomyolipoma in the retrieved literature, rendered from public PubChem SMILES. Which drugs appear here reflects the evidence found, not a ranked prediction.
Molecular view
Cytochrome P450 107G1 (RapN) — Everolimus has a real, experimentally solved structure in complex with this target (PDB 6L3A, 3.0 Å). This is the drug's own deposited structure, not a prediction, and confirms it is a structurally characterised molecule rather than an untested guess.
Loading structure…
helix sheet e53drag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6L3A · 3.0 Å · ligand Everolimus (E53). Experimental structure, not a prediction.
What the evidence adds up to
A systematic review of transarterial embolisation for renal angiomyolipoma covering 524 cases found a mean size reduction of 3.4 cm (38.3% of diameter) at a mean follow-up of 39 months. Self-limiting post-embolisation syndrome occurred after 35.9% of procedures, and further morbidity in 6.9%. No procedural mortality was reported. However, 20.9% of patients required unplanned repeat embolisation or surgery during follow-up, most often because of revascularisation (30.0%), unchanged or increasing size (22.6%), refractory or recurring symptoms (16.7%), or acute retroperitoneal haemorrhage (14.3%). The review notes that longitudinal data on long-term size reduction and re-treatment rates are lacking.
A case report describes a 53-year-old woman with a giant renal angiomyolipoma (25 × 18 × 8 cm) that ruptured spontaneously, causing hypovolemic shock (Wunderlich’s syndrome). She was managed with urgent total nephrectomy and recovered. The authors state that for giant angiomyolipoma with intratumoral haemorrhage and symptoms of Wunderlich’s syndrome, partial or total nephrectomy is a treatment option to save life.
A phase II trial tested everolimus 10 mg daily in 20 patients with sporadic angiomyolipomas 3 cm or larger who were candidates for surgery or embolisation. After four 28-day cycles, 10 of 18 evaluable patients (55.6%) showed a 25% or greater volumetric reduction (median reduction 58.5%). After six cycles, 10 of 14 evaluable patients (71.4%) showed a 25% or greater reduction (median 58.2%). However, 4 patients (20%) were withdrawn due to protocol-defined toxicities, and 8 (40%) withdrew themselves because of side effects. Only 11 patients (55%) completed four cycles and 7 (35%) completed six cycles. The trial was small, had no control arm, and the high discontinuation rate limits conclusions about tolerability.
What is still missing are large, randomised trials comparing embolisation, everolimus, and surveillance in sporadic angiomyolipoma, with standardised long-term follow-up protocols. The phase II everolimus data lack a placebo group and do not address whether volumetric reduction translates into fewer ruptures or less need for intervention. The embolisation review highlights the absence of longitudinal data needed to guide indications and follow-up schedules. No drug has been shown to reduce the risk of rupture in a prospective controlled setting.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
The Journal of Urology · 2015 · 119 citations · open access
Transarterial Embolization of Angiomyolipoma: A Systematic Review
AbstractINTRODUCTION: Transarterial embolization is increasingly used in the management of renal angiomyolipoma. The level of evidence establishing the safety and efficacy of transarterial embolization has not increased in parallel. MATERIALS AND METHODS: Using the MOOSE (Meta-analysis Of Observational Studies in Epidemiology) criteria a systematic review of transarterial embolization of angiomyolipoma was performed to establish procedural safety and efficacy. A MEDLINE® PubMed® search revealed 1,739 publications, of which 31 studies met eligibility criteria. RESULTS: A total of 524 cases of transarterial embolization of angiomyolipoma were included in analysis. Self-limiting post-embolization syndrome developed following 35.9% of embolizations and further morbidity developed in 6.9%. No procedural mortality was reported. At a mean followup of 39 months the mean size reduction was 3.4 cm (-38.3% of angiomyolipoma diameter). Unplanned repeat embolization or surgery was required in 20.9% of cases during this period. The most frequent indications for repeat procedures included angiomyolipoma revascularization in 30.0% of cases, unchanged or increasing size in 22.6%, refractory or recurring symptoms in 16.7% and representation with acute retroperitoneal hemorrhage in 14.3%. Treatment included a combination of 2 or more embolic agents in 46.8% of cases, ethanol monotherapy in 41.7%, coil monotherapy in 6.2% and foam or microparticle monotherapy in 5.2%. CONCLUSIONS: Transarterial embolization of angiomyolipoma demonstrates low rates of mortality and serious complications. Re-treatment rates and size reduction at a mean followup of 39 months are presented. Longitudinal data assessing long-term size reduction and re-treatment rates are lacking. Recommendations guiding the indications for transarterial embolization and clear followup require further longitudinal data.
The effect of everolimus on renal angiomyolipoma in patients with tuberous sclerosis complex being treated for subependymal giant cell astrocytoma: subgroup results from the randomized, placebo-controlled, Phase 3 trial EXIST-1
AbstractBACKGROUND: Tuberous sclerosis complex (TSC) is characterized by benign tumours in multiple organs, including the brain, kidneys, skin, lungs and heart. Our objective was to evaluate everolimus, an mTOR inhibitor, in the treatment of angiomyolipoma in patients with subependymal giant cell astrocytoma (SEGA) associated with TSC. METHODS: EXamining everolimus In a Study of Tuberous Sclerosis Complex-1 (NCT00789828), a prospective, double-blind, randomized, placebo-controlled, Phase 3 study, examined everolimus in treating SEGA associated with TSC. Patients with serial SEGA growth from pre-baseline to baseline scans were randomly assigned (2:1) to receive 4.5 mg/m(2)/day everolimus (target blood trough: 5-15 ng/mL; n = 78) or placebo (n = 39). Angiomyolipoma response rates were analysed in patients (n = 44) with target baseline angiomyolipoma lesions (≥1 angiomyolipoma; longest diameter ≥1.0 cm). An angiomyolipoma response rate, defined as the proportion of patients with confirmed angiomyolipoma response, was assessed by kidney CT or MRI screening at baseline, at 12, 24 and 48 weeks and annually. RESULTS: Angiomyolipoma response rates were 53.3% (16/30) and 0% (0/14) for everolimus- and placebo-treated patients, respectively. Angiomyolipoma reductions ≥50% in the sum of volumes of all target lesions were seen only in everolimus-treated patients (56.5, 78.3 and 80.0%) compared with placebo-treated patients (0% at each time point) at Weeks 12, 24 and 48, respectively. Greater percentages of everolimus-treated patients had angiomyolipoma reductions ≥30% at these same time points (82.6, 100 and 100% versus 8.3, 18.2 and 16.7% for everolimus versus placebo, respectively). CONCLUSIONS: Everolimus showed efficacy in reducing angiomyolipoma lesion volume in patients with SEGA associated with TSC.The trial is registered with ClinicalTrials.gov, number NCT00789828; http://clinicaltrials.gov/ct2/show/NCT00789828?term=EXIST-1&rank=1.
International Journal of Surgery Case Reports · 2015 · 39 citations · open access
Spontaneous rupture of a giant renal angiomyolipoma—Wunderlich’s syndrome: Report of a case
AbstractINTRODUCTION: Herein we present a rare case of pontaneous rupture of a giant renal angiomyolipoma (AML), with symptoms of hypovolemic shock (Wunderlich's syndrome), which was managed by urgent total nephrectomy. PRESENTATION OF CASE: A 53 year old female was transferred to the emergency room with progressive acute painful swelling of the left lateral abdominal area, duration of 5h. An emergent ultrasonic examination, revealed a heterogeneous-solid mass with maximum diameter of 23cm, with probable origin from the left kidney. Due to worsening of the clinical status (hypovolemic shock), loss of consciousness and acute drop of haematocrit level to 17.8%, the patient was urgently intubated in the emergency room and transferred to the operating theater. A giant haemorrhagic mass was found originating from the left kidney, which removed en-block with the left kidney. The patient was transferred to the intensive care unit. Her recovery was uneventful. The histopathologic examination revealed a giant renal angiomyolipoma (25×18×8cm) with extensive bleeding. DISCUSSION: Enlarged renal AMLs can rupture. This can be sudden and painful with manifestations of hypovolemic shock. The management of AMLs has been correlated with symptoms. Patients with life-threatening retroperitoneal haemorrhage, require urgent exploration as retroperitoneal bleeding can lead to severe complications, increasing morbidity. CONCLUSION: In case of giant angiomyolipoma with intratumoral haemorrhage, and symptoms of Wunderlich's syndrome, partial or total nephrectomy is a good treatment option in order to save the patient's life.
The Journal of Urology · 2020 · 15 citations · open access
Sporadic Angiomyolipomas Growth Kinetics While on Everolimus: Results of a Phase II Trial
AbstractPURPOSE: Everolimus decreases tumor volume of renal angiomyolipomas in patients with tuberous sclerosis. No prospective data are available regarding the effect of everolimus on the growth kinetics in patients with sporadic angiomyolipomas. We sought to determine the safety and efficacy of everolimus in the volumetric reduction of sporadic angiomyolipomas. MATERIALS AND METHODS: This multi-institutional, prospective, phase II trial, enrolled patients with 3 cm or larger sporadic angiomyolipomas who were candidates for surgical resection or percutaneous angioembolization. Patients received 10 mg everolimus daily for 4 planned 28-day cycles. Response was defined as a 25% or greater volumetric reduction of patient angiomyolipoma. Baseline, 4, 6 and 12-month volumetric analyses were performed using magnetic resonance imaging. Everolimus was discontinued in those with less than 25% volumetric reduction after 4 cycles. Those with 25% or greater volumetric reduction received 2 additional cycles. The primary outcomes were the efficacy of everolimus in the volumetric reduction of angiomyolipomas by 25% or more, and the safety and tolerability of everolimus. RESULTS: Overall 20 patients were enrolled at 5 centers. Of these patients 11 (55%) completed 4 cycles and 7 (35%) completed 6 cycles. Efficacy was demonstrated, with 10 of 18 (55.6%) patients exhibiting a 25% or greater reduction in tumor volume at 4 months (median 58.5%) and 10 of 14 (71.4%) patients exhibiting a 25% or greater reduction in tumor volume at 6 months (median 58.2%). Four (20%) patients were withdrawn due to protocol defined toxicities and 8 (40%) self-withdrew from the study due to side effects. CONCLUSIONS: Everolimus was effective in causing volumetric reduction of angiomyolipomas by 25% or greater in most patients but was associated with a high rate of treatment discontinuation.
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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