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An RNAi therapeutic, DFP-10825, for intraperitoneal and intrapleural malignant cancers
Hidenori Ando1, Tatsuhiro Ishida1
1Department of Pharmacokinetics and Biopharmaceutics, Institute of Biomedical Sciences, Tokushima University, Tokushima, Japan.
Abstract:
RNA interference (RNAi), a potent post-transcriptional gene-silencing action, has received considerable attentions as a novel therapeutic tool to treat intractable cancers. In recent days, we have developed a novel RNAi-based therapeutic formulation, DFP-10825, for the treatment of intractable advanced cancers developed in coelomic cavities. DFP-10825 was composed of chemically synthesized short hairpin RNA (shRNA) against thymidylate synthase (TS), a key enzyme for cancer proliferation, and cationic liposomes, and achieved high therapeutic effect on the mouse models of peritoneally disseminated gastric and ovarian cancers and malignant pleural mesothelioma without severe side effects by intracoelomic direct treatment. We further designed a freeze-dried DFP-10825 formulation for mass industrial production. DFP-10825 is undergoing in pre-clinical phase and goes to clinical trials. This review introduces a DFP-10825 formulation, a potent novel RNAi-based therapeutic maximizing the benefit of RNAi molecule (shRNA).
Insights
A novel RNAi therapeutic, DFP-10825, effectively treats advanced cancers by silencing thymidylate synthase. This formulation shows promise for clinical trials, offering a new treatment for difficult-to-treat cancers.
Area of Science:
- Biotechnology
- Molecular Biology
- Oncology
Background:
- RNA interference (RNAi) is a gene-silencing mechanism with therapeutic potential for cancer.
- Intractable cancers, particularly those in coelomic cavities, require novel treatment strategies.
Purpose of the Study:
- To introduce DFP-10825, a novel RNAi-based therapeutic formulation for advanced cancers.
- To highlight the efficacy and formulation development of DFP-10825 for industrial production.
Main Methods:
- Development of DFP-10825 using chemically synthesized short hairpin RNA (shRNA) targeting thymidylate synthase (TS).
- Formulation of shRNA with cationic liposomes for targeted delivery.
- Evaluation of therapeutic effects in mouse models of peritoneally disseminated gastric and ovarian cancers and malignant pleural mesothelioma.
Main Results:
- DFP-10825 demonstrated high therapeutic efficacy in preclinical mouse models.
- Treatment was administered via intracoelomic injection, showing effectiveness without severe side effects.
- A freeze-dried formulation was developed for scalable industrial production.
Conclusions:
- DFP-10825 represents a potent RNAi-based therapeutic for intractable cancers.
- The formulation is advancing to clinical trials, indicating significant therapeutic promise.
- DFP-10825 maximizes the benefits of shRNA technology for cancer treatment.
