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Cancer gene therapy: connecting basic research with clinical inquiry
1Dana-Farber Cancer Institute and Harvard Medical School, Boston, MA 02115, USA. glenn_dranoff@dfci.harvard.edu
Abstract:
Molecular genetics has spawned an impressive outpouring of insights into the biology of neoplastic transformation and the host-tumor relationship. This deeper understanding of cancer pathogenesis presents a rich opportunity to develop novel therapeutic agents with improved selectivity for cancer cells. One promising approach involves gene therapy, which is the introduction of genetic material into a patient's tissues with the intent to achieve therapeutic benefit. A number of gene transfer systems have been designed that enable the genetic modification of relevant target cells, albeit with varying strengths and limitations. Several strategies to exploit gene transfer as a tool to target specific molecular defects intrinsic to cancer cells, enhance tumor chemosensitivity, and augment tumor immunogenicity are under intensive investigation. A number of these approaches have entered initial clinical testing and already provide intriguing new information about the biology of cancer in patients. In this review, I will highlight the critical issues and controversies that underscore preclinical experiments in cancer gene therapy, discuss some of the preliminary findings from the first wave of clinical trials, and speculate about the prospects that cancer gene therapy will change the way that cancer medicine is practiced.
Insights
Gene therapy offers a promising new avenue for cancer treatment by introducing genetic material to target cancer cells. Early clinical trials are yielding valuable insights into cancer biology and the potential of gene therapy in clinical practice.
Area of Science:
- Molecular genetics
- Cancer biology
- Gene therapy
Background:
- Advances in molecular genetics have deepened our understanding of cancer development and the host-tumor relationship.
- This knowledge creates opportunities for developing targeted cancer therapies with improved selectivity.
Purpose of the Study:
- To review critical issues and controversies in preclinical cancer gene therapy research.
- To discuss preliminary findings from early-phase clinical trials of gene therapy for cancer.
- To explore the future prospects of gene therapy in clinical cancer medicine.
Main Methods:
- Review of preclinical gene therapy experiments.
- Analysis of preliminary data from initial clinical trials.
- Discussion of gene transfer systems and their applications in targeting cancer cells.
Main Results:
- Various gene transfer systems exist with different strengths and limitations for modifying target cells.
- Strategies are being investigated to target cancer-specific molecular defects, enhance chemosensitivity, and boost immunogenicity.
- Initial clinical trials are providing novel biological insights into cancer in patients.
Conclusions:
- Cancer gene therapy is an intensively investigated field with approaches entering clinical testing.
- Preliminary clinical data offer intriguing insights into cancer biology.
- Gene therapy holds the potential to significantly alter the practice of cancer medicine.