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Updated: Aug 16, 2026
Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
Published on: August 25, 2021
Cancer gene therapy
Masato Yamamoto1, David T Curiel
1BMR2-410, 901 19th Street South, Birmingham, AL 35294-2172, USA.
Abstract:
The prognosis of patients with some kinds of cancers whose patients are often found unresectable upon diagnosis is still dismal. In these fields, development of a new therapeutic modality is needed and gene therapy represents one promising strategy. So far, numerous cancer gene therapy clinical trials based on these principles have been carried out and have shown the safety of such modalities, but have fallen short of the initial expectations to cure cancers. In this review, we would like to make a problem-oriented discussion of current status of cancer gene therapy research by using mainly gastrointestinal cancers as an example. In order to overcome obstacles for full realization of cancer gene therapy, numerous researches have been conducted by many researchers. Various cancer-selective and non-selective genes, as well as lytic viruses themselves have been employed for gene therapy. In the context of gene delivery method, different kinds of viral and non-viral strategies have been utilized. In addition, surrogate assays, such as soluble markers and imaging, have been developed for safer and more informative clinical trials. Many experiments and clinical trials to date have figured out current obstacles for the realization of an effective cancer gene therapy modality. Tireless efforts to overcome such hurdles and continuous infusion of novel concepts into this field should lead to break through technologies and the cure of the patients.
Insights
Cancer gene therapy shows promise for unresectable cancers, but faces challenges. Ongoing research into novel genes, delivery methods, and surrogate assays aims to overcome obstacles and improve patient outcomes.
Area of Science:
- Oncology
- Gene Therapy
- Cancer Research
Background:
- Prognosis for unresectable cancers remains poor, necessitating novel therapeutic strategies.
- Gene therapy is a promising approach, with numerous clinical trials demonstrating safety but limited efficacy.
- Gastrointestinal cancers serve as a key example in evaluating current gene therapy research.
Purpose of the Study:
- To provide a problem-oriented discussion on the current status of cancer gene therapy.
- To identify and analyze obstacles hindering the full realization of cancer gene therapy.
- To highlight ongoing research efforts and future directions in the field.
Main Methods:
- Review of existing cancer gene therapy research, focusing on gastrointestinal cancers.
- Analysis of various therapeutic agents, including cancer-selective/non-selective genes and lytic viruses.
- Examination of diverse gene delivery strategies, encompassing viral and non-viral methods.
- Evaluation of surrogate assays, such as soluble markers and imaging, for clinical trial enhancement.
Main Results:
- Cancer gene therapy trials have established safety but have not met initial curative expectations.
- Numerous strategies involving different genes, viruses, and delivery systems have been explored.
- Development of surrogate assays has improved the safety and informativeness of clinical trials.
- Current research has identified significant obstacles to effective cancer gene therapy.
Conclusions:
- Overcoming obstacles in cancer gene therapy requires continued research and novel concepts.
- Efforts to refine gene delivery and therapeutic agents are crucial for clinical success.
- Advancements in gene therapy hold potential for breakthrough treatments and cures for cancer patients.
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