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Published on: September 1, 2018
Gene therapy as an adjuvant treatment for malignant gliomas: from bench to bedside
Isabelle M Germano1, Emanuela Binello
1Department of Neurosurgery, Mt. Sinai School of Medicine, New York, NY 10029, USA. isabelle.germano@mountsinai.org
Abstract:
Malignant brain tumors, including high-grade gliomas, are among the most lethal of all cancers. Despite considerable advances, including multi-modality treatments with surgery, radiotherapy, and chemotherapy, the overall prognosis for patients with this disease remains dismal. Currently available treatments necessitate the development of more effective tumor-selective therapies. The use of gene therapy for brain tumor therapy is promising as it can be delivered in situ and selectively targets brain tumor cells while sparing the adjacent normal brain tissue. In this article, we summarize the laboratory and clinical work using viral, cell-based, and synthetic vectors, as well as other strategies focused on potentiate gene delivery. Although tangible results on patients' survival remains to be further documented, significant advances in therapeutic gene transfer strategies have been made. The enthusiasm of this progress needs to be tempered by the realistic assessment of the challenges needed to be overcome. Finally, as the field of gene delivery progresses, advances must be made in identifying genes and proteins key to the treatment of malignant gliomas. Due to the great heterogeneity of malignant glioma cells, only approaches combining different strategies may be ultimately successful in defeating this disease.
Insights
Gene therapy offers a promising, targeted approach for malignant brain tumors. While challenges remain, advances in gene delivery strategies are crucial for developing effective treatments for high-grade gliomas.
Area of Science:
- Neuro-oncology
- Molecular Biology
- Biotechnology
Background:
- Malignant brain tumors, particularly high-grade gliomas, have a poor prognosis despite current multi-modality treatments.
- There is a critical need for more effective and tumor-selective therapies.
Purpose of the Study:
- To review laboratory and clinical advancements in gene therapy for brain tumors.
- To discuss strategies for enhancing gene delivery and overcoming challenges in treating malignant gliomas.
Main Methods:
- Summary of research involving viral, cell-based, and synthetic vectors for gene delivery.
- Exploration of strategies to potentiate therapeutic gene transfer.
Main Results:
- Significant progress has been made in therapeutic gene transfer strategies for brain tumors.
- Tangible improvements in patient survival require further documentation and validation.
Conclusions:
- Gene therapy shows promise for selective targeting of brain tumor cells.
- Overcoming challenges in gene delivery and addressing glioma cell heterogeneity are essential for successful treatment.
- Combination approaches may be necessary to effectively combat malignant gliomas.

