Trial reportScience translational medicine2021
A first-in-human phase 0 clinical study of RNA interference-based spherical nucleic acids in patients with recurrent glioblastoma.
Trial report in Science translational medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03020017 (A Phase 0 First-In-Human Study Using NU-0129), which is not on this map. Cited by 179 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
A Phase 0 First-In-Human Study Using NU-0129: A Spherical Nucleic Acid (SNA) Gold Nanoparticle Targeting BCL2L12 in Recurrent Glioblastoma Multiforme or Gliosarcoma Patients
Who cites it
179 citing papers in PubMed, 2 syntheses or guidelines pooled it, 307 citations in OpenAlex.
- Analyzing Molecular Determinants of Nanodrugs' Cytotoxic Effects.International journal of molecular sciences · 2025Pooled it
- A systematic review of non-coding RNA therapeutics in early clinical trials: a new perspective against cancer.Journal of translational medicine · 2024Pooled it
- Targeting cancer-associated fibroblasts: therapeutic strategies, translational challenges, and future perspectives.Journal of hematology & oncology · 2026Review
- Molecular Engineering of Aptamers for Glioblastoma Therapy: From Simple Antagonists to AI-Driven Approaches, a Narrative Review.International journal of molecular sciences · 2026Review
- Advances in vehicles for in situ delivery: From classical vectors to biologically inspired structures.Synthetic and systems biotechnology · 2026Review
- Applications of Bioinorganic Nano-Based Delivery Systems-Diagnostics and Therapeutics.Drug development research · 2026Review
- A metabolic amplification strategy for spherical nucleic acid immunogenicity driven by simvastatin-CpG codelivery.Materials today. Bio · 2026Article
- The Nucleolus in Human Disease: Ribosome Biogenesis, Nucleolar Surveillance, and Therapeutic Opportunities.Biomolecules · 2026Review
- BBB-aware stimuli-responsive and biomimetic nanomedicines for glioblastoma.Chinese neurosurgical journal · 2026Review
- Multistage nanomedicine engineering to overcome sequential barriers to glioblastoma treatment: a review.Journal of nanobiotechnology · 2026Review
- Gold Nanoparticles in Central Nervous System Diseases: Recent Progress, Challenges, and Therapeutic Opportunities.Nano letters · 2026Review
- PEGylated gold nanoparticles regulate metabolic flux in erythrocytes by inducing hemoglobin deoxygenation.International journal of pharmaceutics: X · 2026Article
- DNA Nanostructures for siRNA Delivery.Bioconjugate chemistry · 2026Review
- Transforming tumor microenvironments: nanotechnology and gene therapy in cellular signaling and epigenetic insight into chemo-resistance.Journal of experimental & clinical cancer research : CR · 2026Review
- Article
- Recent Developments in Lipid Nanoparticle-Mediated Delivery of Biotherapeutics and Gene Therapy Across the Blood-Brain Barrier.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2026Review
- Application Advances of Gold Nanoparticles in Cancer Theranostics: From Physicochemical Mechanisms to Multifunctional Nanoplatforms.International journal of molecular sciences · 2026Review
- Polysaccharide-functionalized gold, silver, and iron oxide nanoparticles for siRNA delivery: The role of artificial intelligence in design and optimization.Materials today. Bio · 2026Review
- Nonviral Gold Nanoparticle-Mediated Delivery of CRISPR-Cas9 Ribonucleoprotein and Long DNA Transgenes Into Primary Blood Cells.Advanced nanobiomed research · 2026Article
- siRNA Nanoparticle Delivery Strategies and Clinical Trial Advances in Tumor Therapy.International journal of molecular sciences · 2026Review
119 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
37 authors at 7 institutions in 1 country.
Funding
Abstract
Glioblastoma (GBM) is one of the most difficult cancers to effectively treat, in part because of the lack of precision therapies and limited therapeutic access to intracranial tumor sites due to the presence of the blood-brain and blood-tumor barriers. We have developed a precision medicine approach for GBM treatment that involves the use of brain-penetrant RNA interference-based spherical nucleic acids (SNAs), which consist of gold nanoparticle cores covalently conjugated with radially oriented and densely packed small interfering RNA (siRNA) oligonucleotides. On the basis of previous preclinical evaluation, we conducted toxicology and toxicokinetic studies in nonhuman primates and a single-arm, open-label phase 0 first-in-human trial (NCT03020017) to determine safety, pharmacokinetics, intratumoral accumulation and gene-suppressive activity of systemically administered SNAs carrying siRNA specific for the GBM oncogene Bcl2Like12 (Bcl2L12). Patients with recurrent GBM were treated with intravenous administration of siBcl2L12-SNAs (drug moniker: NU-0129), at a dose corresponding to 1/50th of the no-observed-adverse-event level, followed by tumor resection. Safety assessment revealed no grade 4 or 5 treatment-related toxicities. Inductively coupled plasma mass spectrometry, x-ray fluorescence microscopy, and silver staining of resected GBM tissue demonstrated that intravenously administered SNAs reached patient tumors, with gold enrichment observed in the tumor-associated endothelium, macrophages, and tumor cells. NU-0129 uptake into glioma cells correlated with a reduction in tumor-associated Bcl2L12 protein expression, as indicated by comparison of matched primary tumor and NU-0129-treated recurrent tumor. Our results establish SNA nanoconjugates as a potential brain-penetrant precision medicine approach for the systemic treatment of GBM.
Indexed as
Identifiers
What OpenQuestion holds
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.