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Translational Approaches towards Cancer Gene Therapy: Hurdles and Hopes
1Ovarian Cancer Research Center, Translational Research Center, University of Pennsylvania, Philadelphia, PA, USA.
Introduction:
Of the cancer gene therapy approaches, gene silencing, suicide/apoptosis inducing gene therapy, immunogene therapy and targeted gene therapy are deemed to sub-stantially control the biological consequences of genomic changes in cancerous cells. Thus, a large number of clinical trials have been conducted against various malignancies. In this review, we will discuss recent translational progresses of gene and cell therapy of cancer.
Methods:
Essential information on gene therapy of cancer were reviewed and discussed towards their clinical translations.
Results:
Gene transfer has been rigorously studied in vitro and in vivo, in which some of these gene therapy endeavours have been carried on towards translational investigations and clinical applications. About 65% of gene therapy trials are related to cancer therapy. Some of these trials have been combined with cell therapy to produce personalized medicines such as Sipuleucel-T (Provenge®, marketed by Dendreon, USA) for the treatment of asymptomatic/minimally symptomatic metastatic hormone-refractory prostate cancer.
Conclusion:
Translational approach links two diverse boundaries of basic and clinical researches. For successful translation of geno-medicines into clinical applications, it is essential 1) to have the guidelines and standard operating procedures for development and application of the genomedicines specific to clinically relevant biomarker(s); 2) to conduct necessary animal experimental studies to show the "proof of concept" for the proposed genomedicines; 3) to perform an initial clinical investigation; and 4) to initiate extensive clinical trials to address all necessary requirements. In short, translational researches need to be refined to accelerate the geno-medicine development and clinical applications.
Insights
Cancer gene therapy, including gene silencing and immunogene therapy, shows promise in controlling cancer. Translational research is key to advancing these geno-medicines from the lab to clinical application.
Area of Science:
- Oncology
- Gene Therapy
- Translational Research
Background:
- Gene therapy approaches like gene silencing, suicide/apoptosis inducing gene therapy, immunogene therapy, and targeted gene therapy aim to control cancerous cell genomic changes.
- Numerous clinical trials have been conducted for various malignancies utilizing these gene therapy strategies.
Purpose of the Study:
- To review and discuss recent translational progress in gene and cell therapy for cancer.
- To highlight the clinical translation of gene therapy approaches in oncology.
Main Methods:
- A comprehensive review of existing literature on gene therapy for cancer was conducted.
- Information was analyzed to discuss clinical translation and applications.
Main Results:
- Gene transfer has been extensively studied in vitro and in vivo, with many efforts progressing to translational investigations and clinical applications.
- Approximately 65% of gene therapy trials focus on cancer treatment, with some combined with cell therapy for personalized medicine, such as Sipuleucel-T for prostate cancer.
Conclusions:
- The translational approach bridges basic and clinical research, essential for advancing geno-medicines.
- Successful clinical translation requires specific guidelines, proof-of-concept studies, initial clinical investigations, and extensive trials.
- Refining translational research is crucial to accelerate the development and clinical application of geno-medicines.
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