Albendazole

證據等級: L5

預測適應症: 10 個

Albendazole: From Intestinal Helminthiasis to Alveolar Echinococcosis

One-Sentence Summary

Albendazole is a broad-spectrum benzimidazole antiparasitic agent, originally established as the WHO first-line treatment for intestinal helminthiasis and soil-transmitted helminth infections. The TxGNN model predicts it may be effective for Alveolar Echinococcosis, with 5 clinical trials and 20 publications currently supporting this direction.

Quick Overview

Item	Content
Original Indication	Intestinal helminthiasis (soil-transmitted helminth infections)
Predicted New Indication	Alveolar Echinococcosis
TxGNN Prediction Score	99.97%
Evidence Level	L2
India Market Status	✗ Not Marketed (0 registrations in dataset)
Number of Registrations	0
Recommended Decision	Proceed with Guardrails

Why is This Prediction Reasonable?

Detailed mechanism of action data is not available in this Evidence Pack (Data Gap DG002). Based on known pharmacological information, Albendazole is a benzimidazole compound whose active metabolite — albendazole sulfoxide — selectively binds to parasite β-tubulin with approximately 1,000-fold greater affinity than mammalian tubulin, inhibiting microtubule polymerization and disrupting cyst wall cytoskeletal integrity. It simultaneously blocks parasite glucose uptake via GLUT inhibition, exploiting a critical vulnerability: Echinococcus multilocularis larvae cannot perform gluconeogenesis and rely entirely on external glucose, leading to ATP depletion and eventual parasite death or stasis. Albendazole sulfoxide penetrates the metacestode cyst wall to achieve therapeutic concentrations at the target site.

Alveolar echinococcosis (AE) is a severe, potentially fatal zoonosis caused by the larval stage of Echinococcus multilocularis, a cestode tapeworm. The same β-tubulin inhibition pathway that governs albendazole’s efficacy in intestinal helminthiasis applies directly to cestode larval stages — the key difference is that in AE the drug acts parasitostatic rather than parasiticidal, halting disease progression rather than eliminating the parasite outright. This means long-term or lifelong treatment is often required, particularly when surgical resection is not feasible (approximately 60–70% of cases).

The WHO-IWGE Expert Consensus (PMID 19931502) formally lists albendazole as the first-line continuous pharmaceutical treatment for AE, supported by a Phase 2 trial with 194 patients (NCT07182305), multiple observational cohort studies, and an extensive review literature. The 99.97% TxGNN prediction score reflects genuine clinical and biological evidence rather than a network artefact.

Clinical Trial Evidence

Trial Number	Phase	Status	Enrollment	Key Findings
NCT07182305	Phase 2	Completed	194	Direct AE Phase 2 trial in Kyrgyzstan (2017–2023); the largest prospective trial to date evaluating albendazole as parasitostatic treatment for early-stage alveolar echinococcosis caused by E. multilocularis. High relevance (Grade A).
NCT02876146	NA	Completed	50	EchinoVISTA prospective study (2012–2021); defines improved management of hepatic AE patients on albendazole by identifying biological and imaging markers of parasite viability to guide treatment withdrawal decisions. Observational design (Grade B).
NCT06483880	NA	Unknown	24	Randomised controlled trial evaluating adjuvant albendazole after pulmonary hydatid cyst (cystic echinococcosis, CE) resection vs placebo for 6-month recurrence reduction. Targets CE rather than AE; small sample size (Grade B).
NCT05824442	NA	Recruiting	43	Evaluation of a multiplex quantitative PCR technique for echinococcosis diagnosis; albendazole referenced as the treatment backbone. Diagnostic study without direct efficacy assessment (Grade C).
NCT07176598	N/A	Completed	1	Case report of a misdiagnosed primary intramuscular hydatid cyst treated with albendazole; narrative reference only, no statistical power (Grade C).

Literature Evidence

PMID	Year	Type	Journal	Key Findings
19931502	2010	Consensus Guideline	Acta Tropica	WHO-IWGE expert consensus on diagnosis and treatment of cystic and alveolar echinococcosis; recommends albendazole as the first-line pharmacological agent for AE
30760475	2019	Comprehensive Review	Clinical Microbiology Reviews	Major 21st-century advances in echinococcosis covering genetics, molecular epidemiology, diagnostic tools, and treatment; confirms albendazole central role
39311470	2024	Review	Parasite (Paris)	Current status of benzimidazole chemotherapy for AE; characterises parasitostatic limitations, liver toxicity risks, and the urgent need for alternative agents
40093668	2025	Clinical Review	World Journal of Gastroenterology	Management of liver echinococcosis; albendazole positioned as essential perioperative and long-term adjunct to surgical treatment
25526545	2014	Review	Parasite (Paris)	Novel therapeutic options for AE and CE beyond albendazole/mebendazole; large-scale drug screening strategies and albendazole as the reference comparator
36974024	2022	Review	Chinese Journal of Schistosomiasis Control	Progress of albendazole research specifically for AE treatment; reviews evidence for parasitostatic effects and recent clinical studies
38501660	2024	Pharmacology Study	Antimicrobial Agents and Chemotherapy	Metabolic mechanism and pharmacological study of albendazole in a secondary hepatic AE rat model; evaluates enhanced-solubility formulations (ABZ-CSD, TABZ-HCl-H) to overcome poor oral bioavailability
34808118	2022	Review	Acta Tropica	Status and prospects of novel treatments for AE and CE; confirms albendazole and mebendazole remain the only two licensed non-surgical treatment options globally
34161992	2021	Clinical Review	Seminars in Liver Disease	Hepatic alveolar echinococcosis review; documents the pseudotumoral presentation and role of lifelong albendazole in non-resectable cases
39254012	2024	Review	Tidsskrift for den Norske laegeforening	Current clinical overview of AE in a non-endemic European setting; highlights prolonged albendazole therapy as standard management combined with surgery

India Market Information

No registrations for Albendazole were found in this dataset for the Indian market (CDSCO). This likely reflects a data collection gap rather than the actual availability of the drug: Albendazole is included in the WHO Model List of Essential Medicines and is widely used in global mass drug administration programmes.

Note: It is strongly recommended to verify current CDSCO approval status directly via the Indian drug regulatory authority database before proceeding with any clinical or procurement activities.

Safety Considerations

Drug Interactions: Albendazole has 96 documented interactions in the DDInter database. Key interactions to monitor:

Interacting Drug	Level	Potential Clinical Impact
Aprepitant	Moderate	CYP3A4/CYP2C19 modulation may alter albendazole sulfoxide plasma exposure
Cobicistat	Moderate	CYP enzyme inhibition may increase active metabolite levels
Apalutamide	Moderate	CYP3A4 induction may reduce albendazole efficacy
Bosentan	Moderate	Potential additive hepatotoxicity risk
Boceprevir	Moderate	CYP3A4 inhibition may elevate albendazole metabolite concentrations
Bendamustine	Moderate	Possible additive hepatotoxic effects
Fostamatinib	Moderate	Pharmacokinetic interaction via CYP pathways
Deferasirox	Moderate	Potential drug-drug interaction affecting systemic exposure
Dexamethasone	Minor	May increase albendazole sulfoxide levels — notably relevant in AE treatment protocols where corticosteroids are co-administered
Cimetidine	Minor	May increase albendazole plasma concentrations via CYP inhibition

Please refer to the package insert for complete safety information including key warnings and contraindications, as TFDA/CDSCO prescribing information was not available in this data pack (Data Gap DG001).

Conclusion and Next Steps

Decision: Proceed with Guardrails

Rationale: Albendazole is supported by WHO expert consensus guidelines and a completed Phase 2 prospective trial with 194 patients (NCT07182305) as the primary pharmacological treatment for alveolar echinococcosis — making this prediction a genuine clinical application rather than speculative repurposing. The biological mechanism is well-characterised, the drug is already used off-label for AE in multiple countries, and the evidence base is substantial enough to justify continued development under a structured oversight framework.

To proceed, the following is needed:

Regulatory data (Blocking): Obtain CDSCO prescribing information to assess key warnings and contraindications (Data Gap DG001 — currently blocking Safety Stage 1 evaluation)
Mechanism documentation: Retrieve formal MOA data from DrugBank API to complete mechanistic link analysis (Data Gap DG002)
India regulatory pathway: Verify current CDSCO registration status; determine whether import authorization, named-patient supply, or new registration is required given 0 licenses found in dataset
Long-term monitoring protocol: Establish hepatotoxicity monitoring plan (ALT/AST, bilirubin, alkaline phosphatase at baseline and every 4–8 weeks) given documented risk of liver dysfunction during prolonged albendazole therapy
Formulation optimization assessment: Evaluate enhanced bioavailability formulations (e.g., albendazole crystal dispersion systems) given known poor and erratic oral absorption due to low aqueous solubility — this is particularly critical for achieving therapeutic concentrations within echinococcal cysts
Surgical candidacy criteria: Define patient selection criteria distinguishing resectable from non-resectable AE to position albendazole correctly as definitive therapy vs. perioperative adjunct

⚠️ Disclaimer: This report is for research reference only and does not constitute medical advice. All drug repurposing candidates require clinical validation before application. Results generated by the TxGNN model are computational predictions and must be interpreted in conjunction with clinical expertise.

Disclaimer

This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.