Trial reportInvestigational new drugs2014
A phase II trial of AS1411 (a novel nucleolin-targeted DNA aptamer) in metastatic renal cell carcinoma.
Trial report in Investigational new drugs, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 198 papers, 3 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
198 citing papers in PubMed, 3 syntheses or guidelines pooled it, 403 citations in OpenAlex.
- Advances in Aptamer Diagnostics Targeting Non-Small-Cell Lung Cancer: A Systematic Review.International journal of molecular sciences · 2026Pooled it
- Nucleolin‑based targeting strategies in cancer treatment: Focus on cancer immunotherapy (Review).International journal of molecular medicine · 2023Pooled it
- Cellular localization of nucleolin determines the prognosis in cancers: a meta-analysis.Journal of molecular medicine (Berlin, Germany) · 2022Pooled it
- Lipid nanoparticles with aptamers enable targeted mRNA delivery to CD4⁺ T cells.Drug delivery · 2026Article
- The cellular landscape of druggable RNA-binding proteins.Nature reviews. Drug discovery · 2026Review
- Aptamer-Functionalized Liposomes for Targeted Delivery of Anticancer Drugs in Lung Cancer.Molecules (Basel, Switzerland) · 2026Article
- Nucleic acid aptamers: new methods for selection, target validation, molecular diagnostics and therapeutics.Signal transduction and targeted therapy · 2026Review
- Targeting IGF2BP3 in Cancer: From Molecular Structure and Biology to Early Drug Discovery.International journal of molecular sciences · 2026Review
- Recent Advances in Lipid Nanoparticle-Mediated Respiratory and Gastrointestinal Mucosal Delivery of Nucleic Acids.Bioengineering (Basel, Switzerland) · 2026Review
- AS1411-Induced Lipidomic Alterations and Therapeutic Insights in U-87 Glioblastoma Cells.Biomolecules · 2026Article
- Tetrahedral DNA nano-PROTACs enable enhanced ocular penetration and efficient nucleolin degradation for choroidal neovascularization therapy.Signal transduction and targeted therapy · 2026Article
- Development of AS1411 Aptamer-Targeted Solid Lipid Nanoparticles to Improve Anticancer Efficiency of Lawsone.Advanced pharmaceutical bulletin · 2026Article
- Aptamers in cancer therapy: Why has clinical translation lagged behind preclinical promise?Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie · 2026Review
- Mechanisms and Design Principles of Proteolysis-Targeting Chimeras and Their Emerging Applications.ACS pharmacology & translational science · 2026Review
- Fulfilling multiple roles in PROTAC design: The emerging potential of oligonucleotides.European journal of medicinal chemistry · 2026Review
- Extracellular Vesicle-Mediated Nucleolin Transfer in Glioblastoma: A Targetable Axis Driving Blood-Tumour Barrier Formation.Journal of extracellular vesicles · 2026Article
- Macroscopic polarimetric discrimination and quantification of antiangiogenic effect of AGRO aptamer and GK1 peptide in a preclinical melanoma model.Scientific reports · 2026Article
- Aptamer-liposome targeted nanotherapeutics for cancer therapy: Bibliometric analysis, recent developments and future perspectives.Materials today. Bio · 2026Review
- Preliminary assessment of biodistribution and targeting of the fluorescent molecular probe Cy7-SYL3C in an EpCAM-positive colorectal cancer mouse model.Scientific reports · 2026Article
- Covalent aptamers: agents with promising therapeutic and diagnostic potential.RSC chemical biology · 2026Review
138 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
13 authors at 10 institutions in 7 countries.
Funding
Abstract
backgroundDNA aptamers represent a novel strategy in anti-cancer medicine. AS1411, a DNA aptamer targeting nucleolin (a protein which is overexpressed in many tumor types), was evaluated in patients with metastatic, clear-cell, renal cell carcinoma (RCC) who had failed treatment with ≥1 prior tyrosine kinase inhibitor.
methodsIn this phase II, single-arm study, AS1411 was administered at 40 mg/kg/day by continuous intravenous infusion on days 1-4 of a 28-day cycle, for two cycles. Primary endpoint was overall response rate; progression-free survival (PFS) and safety were secondary endpoints.
results35 patients were enrolled and treated. One patient (2.9 %) had a response to treatment. The response was dramatic (84 % reduction in tumor burden by RECIST 1.0 criteria) and durable (patient remains free of progression 2 years after completing therapy). Whole exome sequencing of this patient's tumor revealed missense mutations in the mTOR and FGFR2 genes which is of interest because nucleolin is known to upregulate mTOR pathway activity by enhancing AKT1 mRNA translation. No other responses were seen. Thirty-four percent of patients had an AS1411-related adverse event, all of which were mild or moderate.
conclusionsAS1411 appears to have minimal activity in unselected patients with metastatic RCC. However, rare, dramatic and durable responses can be observed and toxicity is low. One patient in this study had an excellent response and was found to have FGFR2 and mTOR mutations which will be of interest in future efforts to discover and validate predictive biomarkers of response to nucleolin targeted compounds. DNA aptamers represent a novel way to target cancer cells at a molecular level and continue to be developed with a view to improving treatment and imaging in cancer medicine.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.