DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for hepatic angiomyolipoma — screening already-approved drugs against its 7-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleHepatic angiomyolipoma maps to a 7-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 hepatic angiomyolipoma 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
Rho GTPase activating protein 35 (ARHGAP35) — ARHGAP35 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 gnpdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 3C5H · 1.8 Å · ligand PHOSPHOAMINOPHOSPHONIC ACID-GUANYLATE ESTER (GNP). Experimental structure, not a prediction.
What the evidence adds up to
Hepatic angiomyolipoma is a rare mesenchymal liver tumour considered predominantly benign, but malignant behaviour including invasive growth, recurrence after resection and metastases has been reported. A systematic review of 292 patients found that of 247 treated with surgery, recurrence occurred in 6 (2.4%), with metastases and death in 2 (0.8% mortality rate). Among 35 patients managed conservatively, progression was described in 6 (21.4%). In a single-centre series of 79 patients who underwent liver resection, one patient had tumour recurrence 6 years after surgery and died 1 year later; median survival was 90 months. Spontaneous rupture occurred in one patient. Symptom relief was achieved in 92% of symptomatic patients. Accurate preoperative diagnosis was made in only 52% of cases, and tumours smaller than 6 cm were more frequently misdiagnosed.
One case report describes a 26-year-old woman with tuberous sclerosis complex and massive hepatic angiomyolipoma causing severe malnutrition and abdominal pain. After 6 months of tamoxifen (20 mg twice daily), she had greatly improved quality of life and significant weight gain. After 12 months, MRI showed a significant reduction of the largest lesion with a liquid central area and diminished gastric compression. The authors note this is the first report of tamoxifen used successfully in a tuberous sclerosis patient with multiple liver angiomyolipomas, and they suggest the possible benefit is based on expression of oestrogen and progesterone receptors in these tumours.
Another case report describes a 47-year-old woman whose hepatic angiomyolipoma was initially misdiagnosed as clear cell carcinoma by needle biopsy, leading to partial hepatectomy. She was well with no recurrence 6 months after operation. The systematic review notes that biopsy is indicated when imaging is inconclusive, and that conservative management with annual imaging is justified when the diagnosis is certain on imaging. Resection should be considered for symptoms, inconclusive biopsy, or growth during follow-up. The review also notes that of 12 patients with malignant behaviour, only 2 had epithelioid-type histology; the subtype was undefined in the remaining 10.
What is still missing are prospective trials comparing conservative management, surgery, and medical therapies such as tamoxifen or sirolimus (the latter mentioned in the systematic review as used in 7 patients, but with no outcome data reported). The rarity of the disease makes randomised trials difficult, and no standardised criteria exist for when to start systemic treatment or which patients are at highest risk of malignant transformation. Patient stratification by histologic subtype and hormone receptor status is not routinely performed.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Liver International · 2017 · 91 citations · open access
Management of hepatic angiomyolipoma: A systematic review
AbstractHepatic Angiomyolipoma (HAML) is a rare mesenchymal liver tumour assumed to be predominantly benign, although incidental cases with malignant behaviour such as invasive growth, recurrence after resection and metastases have been reported. The aim of this systematic review was to assess the biological behaviour, estimate the risk of HAML related mortality and recommend on a justifiable management strategy. We performed a systematic literature search in Embase, Medline, Web-of-Science, Scopus, Pubmed Publisher, Cochrane and Google Scholar. We included all articles published from inception until March 2016 which reported on follow-up of various treatment strategies. We included 18 articles reporting on 292 patients. Male:female ratio was estimated at 1:3 with gender not reported in 31 cases. Of 292 patients 247 were treated with surgery, including one liver transplant, seven with chemotherapy or Sirolimus, three with embolization, and 35 conservatively. Recurrence after resection was described in 6/247 (2.4%) with pathologically proven HAML resulting in metastases and death in 2/247 (mortality rate 0.8%). Progression was described in 6/35 patients treated conservatively (21.4%). Two of 12 patients with malignant behaviour of HAML had an epithelioid-type HAML, of the remaining 10 histological subtype was undefined. With a risk estimate of 0.8% in surgically treated patients HAML related mortality is very low. Biopsy is indicated when imaging is inconclusive. In case of certain HAML diagnosis on imaging conservative management with annual imaging is justified. Resection should be considered in case of symptoms, inconclusive biopsy or growth in follow-up.
Journal of Surgical Oncology · 2011 · 70 citations
Diagnosis and treatment of hepatic angiomyolipoma
AbstractBACKGROUND AND OBJECTIVE: Hepatic angiomyolipoma is more frequently encountered in clinical practice, its diagnosis is difficult, its treatment remains controversial. We review a single-center experience in the treatment of hepatic angiomyolipoma. METHOD: The clinical data of 79 patients with hepatic angiomyolipoma treated at the authors' institute between January 1992 and December 2006 were retrospectively reviewed. RESULTS: During a period of 15 years, a total of 79 patients with hepatic angiomyolipoma underwent liver resection at our hospital. There are 58 women and 21 men. The tumor size varied from 1 to 25 cm in diameter (6.1 ± 4.08). Fifty-four patients (68%) were asymptomatic. Accurate preoperative diagnosis was made in 41 patients (52%). Tumors less than 6 cm in size were more frequently misdiagnosed. Spontaneous rupture occurred in one patient. One patient had tumor recurrence 6 years after the surgery, and died of the disease 1 year later. Symptom relief was achieved in 92% of the symptomatic patients. Median survival was 90 months (90.12 ± 30.84). CONCLUSIONS: Hepatic angiomyolipoma poses a diagnostic challenge clinically and radiologically. Surgical resection is a safe and effective treatment option. It is important to realize the potential of malignant transformation and risk of rupture as life-threatening complications.
Journal of Hepatology · 2008 · 20 citations · open access
Massive hepatic angiomyolipoma in a young woman with tuberous sclerosis complex: Significant clinical improvement during tamoxifen treatment
AbstractBACKGROUND/AIMS: Isolated liver angiomyolipomas (AMLs) occur in about 40% of TSC patients. Because of their slow growth, these tumors are often asymptomatic. Since AMLs express estrogen and progesteron receptors we suggest the possible benefits of tamoxifen for the treatment of liver AMLs. METHODS: We report the case of a 26-year-old female affected by tuberous sclerosis (TSC2) with cerebral, renal and hepatic involvement admitted to the Liver Unit for severe malnutrition, anorexia and abdominal pain. MRI showed a grossly enlarged liver, causing severe gastric compression. The liver was entirely filled with multiple nodular lesions of different sizes. Liver biopsy showed tumoral tissue with microscopic and ultrastructural features of angiomyolipoma. All liver function tests were repeatedly normal. Prior to considering the patient for partial hepatectomy, she was administered tamoxifen (20mg b.i.d). RESULTS: After 6 months of tamoxifen treatment a greatly improved quality of life and a significant weight gain were observed. After 12 months the clinical conditions further improved and the MRI showed a significant reduction of the largest lesion with a liquid central area and a diminished compression of the stomach. CONCLUSIONS: This is to our knowledge the first report in which tamoxifen has been successfully used in a TSC patient with multiple liver angiomyolipomas.
AbstractAngiomyolipoma usually involves the kidney and rarely affects the liver. This study reports a case of angiomyolipoma of the liver in a 47-year-old Chinese woman. The patient did not present with abdominal pain and jaundice. Imaging showed a small mass in the right liver. The hepatic artery and portal vein were free from invasion. Partial hepatectomy was performed after a tentative diagnosis of clear cell carcinoma of the liver by needle biopsy. Histopathological examination of the resected specimen revealed angiomyolipoma originating in the liver. The post-operative clinical course was uneventful and, at the time of writing, the patient was well with no signs of recurrence 6 months after operation. To our knowledge this is the first documented case of an angiomyolipoma arising in the liver mimicking hepatic clear cell carcinoma.
Zeitschrift für Gastroenterologie · 2008 · 1 citations
Das Angiomyolipom der Leber - eine seltene Differenzialdiagnose der Leberraumforderung
AbstractA 22-year-old woman was treated for a hepatic lesion with a high suspicion of a liver adenoma at another hospital. The patient presented with unspecific abdominal pain. Further physical examination was unremarkable. A biopsy of the liver lesion revealed hepatic adenoma. Because of the increasing tumour size over a one-year period the patient was referred to our department for surgical therapy. On MRI scan, the liver mass measured 10 x 9 x 9 cm in the right liver lobe with contact to the right hilum. Because of the histological signs of adenoma a right hepatic lobectomy was performed. Postoperative follow-up was uneventful. The pathological diagnosis of hepatic angiomyolipoma was obtained. Angiomyolipoma of the liver is a rare benign mesenchymal tumour often mimicking other hepatic lesions. Histological features are thick-walled blood vessels, mature fat and smooth muscle in various proportions. The biological behaviour of the tumour is benign, although distant metastases are occasionally possible. Due to the potential for malignant transformation, tumour resection should be performed.
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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