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Updated: Jun 11, 2025
High Throughput Characterization of Adult Stem Cells Engineered for Delivery of Therapeutic Factors for Neuroprotective Strategies
Published on: January 4, 2015
Cell and gene therapy in neuro-oncology
Maria Luisa Varela1, Andrea Comba1, Syed M Faisal1
1Department of Neurosurgery, University of Michigan Medical School, Ann Arbor, MI, United States; Department of Cell and Developmental Biology, University of Michigan Medical School, Ann Arbor, MI, United States; Rogel Cancer Center, University of Michigan Medical School, Ann Arbor, MI, United States.
Abstract:
The majority of primary brain tumors are gliomas, among which glioblastoma multiforme (GBM) is the most common malignant brain tumor in adults. GBM has a median survival of 18-24 months, and despite extensive research it remains incurable, thus novel therapies are urgently needed. The current standard of care is a combination of surgery, radiation, and chemotherapy, but still remains ineffective due to the invasive nature and high recurrence of gliomas. Gene therapy is a versatile treatment strategy investigated for multiple tumor types including GBM. In gene therapy, a variety of vectors are employed to deliver genes designed for different antitumoral effects. Also, over the past decades, stem cell biology has provided a new approach to cancer therapies. Stem cells can be used as regenerative medicine, therapeutic carriers, drug targeting, and generation of immune cells. Stem cell-based therapy allows targeted therapy that spares healthy brain tissue as well as establishes a long-term antitumor response by stimulating the immune system and delivering prodrug, metabolizing genes, or even oncolytic viruses. This chapter describes the latest developments and the current trends in gene and cell-based therapy against GBM from both preclinical and clinical perspectives, including different gene therapy delivery systems, molecular targets, and stem cell therapies.
Insights
Novel gene and stem cell therapies offer new hope for glioblastoma multiforme (GBM), a deadly brain cancer. These advanced treatments aim for targeted delivery and long-term immune response against GBM, improving patient outcomes.
Area of Science:
- Neuro-oncology
- Cancer Gene Therapy
- Stem Cell Biology
Background:
- Glioblastoma multiforme (GBM) is the most prevalent and aggressive adult primary brain tumor.
- Current treatments (surgery, radiation, chemotherapy) are insufficient due to tumor invasiveness and recurrence, leading to a poor prognosis.
- Novel therapeutic strategies are essential for improving outcomes in GBM patients.
Purpose of the Study:
- To review recent advancements in gene and stem cell-based therapies for glioblastoma multiforme.
- To explore various gene therapy delivery systems and molecular targets for GBM treatment.
- To discuss the potential of stem cell-based approaches in preclinical and clinical settings for GBM.
Main Methods:
- Review of preclinical and clinical studies on gene therapy for GBM.
- Analysis of different gene delivery vectors and their antitumoral mechanisms.
- Examination of stem cell applications, including regenerative medicine and therapeutic delivery systems.
- Investigation of stem cell-mediated immune stimulation and targeted drug delivery for GBM.
Main Results:
- Gene therapy utilizes diverse vectors for targeted gene delivery to combat GBM.
- Stem cell-based therapies offer targeted treatment, sparing healthy tissue and potentially inducing long-term antitumor immunity.
- These approaches include delivering genes, prodrugs, or oncolytic viruses, and stimulating immune responses.
Conclusions:
- Gene and stem cell-based therapies represent promising avenues for treating glioblastoma multiforme.
- These innovative strategies hold potential for more effective and targeted treatment of GBM.
- Continued research in preclinical and clinical settings is crucial for realizing the full potential of these therapies.
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