ArticleMolecular therapy : the journal of the American Society of Gene Therapy2015
First-in-man phase 1 clinical trial of gene therapy for advanced pancreatic cancer: safety, biodistribution, and preliminary clinical findings.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 52 papers.
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Who cites it
52 citing papers in PubMed.
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- Biomolecule-Based Coacervation: Mechanisms, Applications, and Future Perspectives in Biomedical and Biotechnological Fields.Biomolecules · 2025Review
- Improving PD-1 blockade plus chemotherapy for complete remission of lung cancer by nanoPDLIM2.eLife · 2024Article
- Beyond Lipids: Exploring Advances in Polymeric Gene Delivery in the Lipid Nanoparticles Era.Advanced materials (Deerfield Beach, Fla.) · 2024Review
- Endoscopic Ultrasound-Guided Ablation of Premalignant Pancreatic Cysts and Pancreatic Cancer.Diagnostics (Basel, Switzerland) · 2024Review
- Review
- Therapeutic potential of gene therapy for gastrointestinal diseases: Advancements and future perspectives.Molecular therapy oncolytics · 2023Review
- Preclinical development of non-viral gene therapy for patients with advanced pancreatic cancer.Molecular therapy. Methods & clinical development · 2023Article
- Formulation and Evaluation of Polymer-Based Nanoparticles for Intravitreal Gene-Delivery Applications.Current protocols · 2022Article
- Therapeutic effect of multiple functional minicircle vector encoding anti-CD25/IL-10/CXCR3 in allograft rejection model.The Korean journal of internal medicine · 2022Article
- Endoscopic ultrasound as a reliable tool for assessment of pancreatic adenocarcinoma treatment: Example of in situ gene therapy.Endoscopy international open · 2022Article
- Endoscopic ultrasound role in pancreatic adenocarcinoma treatment: A review focusing on technical success, safety and efficacy.World journal of gastroenterology · 2022Review
- CD47-targeted cancer immunogene therapy: Secreted SIRPα-Fc fusion protein eradicates tumors by macrophage and NK cell activation.Molecular therapy oncolytics · 2021Article
- Nanoparticle-MediatedACS nano · 2021Article
- Immunogenicity of stabilized HIV-1 Env trimers delivered by self-amplifying mRNA.Molecular therapy. Nucleic acids · 2021Article
- A review of the tortuous path of nonviral gene delivery and recent progress.International journal of biological macromolecules · 2021Review
- Minicircles for a two-step blood biomarker and PET imaging early cancer detection strategy.Journal of controlled release : official journal of the Controlled Release Society · 2021Article
- Scalable Purification of Plasmid DNA Nanoparticles by Tangential Flow Filtration for Systemic Delivery.ACS applied materials & interfaces · 2021Article
- Overcoming the Tumor Microenvironmental Barriers of Pancreatic Ductal Adenocarcinomas for Achieving Better Treatment Outcomes.Advanced therapeutics · 2021Article
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Authors and funding
23 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
This phase 1 trial was aimed to determine the safety, pharmacokinetics, and preliminary clinical activity of CYL-02, a nonviral gene therapy product that sensitizes pancreatic cancer cells to chemotherapy. CYL-02 was administrated using endoscopic ultrasound in 22 patients with pancreatic cancer that concomitantly received chemotherapy (gemcitabine). The maximum-tolerated dose (MTD) exceeded the maximal feasible dose of CYL-02 and was not identified. Treatment-related toxicities were mild, without serious adverse events. Pharmacokinetic analysis revealed a dose-dependent increase in CYL-02 DNA exposure in blood and tumors, while therapeutic RNAs were detected in tumors. No objective response was observed, but nine patients showed stable disease up to 6 months following treatment and two of these patients experienced long-term survival. Panels of plasmatic microRNAs and proteins were identified as predictive of gene therapy efficacy. We demonstrate that CYL-02 nonviral gene therapy has a favorable safety profile and is well tolerated in patients. We characterize CYL-02 biodistribution and demonstrate therapeutic gene expression in tumors. Treated patients experienced stability of disease and predictive biomarkers of response to treatment were identified. These promising results warrant further evaluation in phase 2 clinical trial.
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