Gene Therapy for High Grade Glioma: The Clinical Experience

Area of Science:

• Neuro-oncology
• Gene Therapy
• Oncology

Background:

• High-grade gliomas (HGG) are aggressive primary brain tumors with limited treatment options.
• The current standard of care has remained unchanged since 2005, highlighting the need for novel therapeutic strategies.
• Gene therapy presents a promising approach to overcome the limitations of existing treatments for HGG.

Purpose of the Study:

• To review completed and ongoing clinical trials of gene therapy for adult and pediatric HGG.
• To evaluate the preclinical data supporting gene therapy applications in HGG treatment.
• To discuss the safety and efficacy of viral and non-viral gene therapy vectors in HGG.

Main Methods:

• Review of completed and ongoing clinical trials involving viral and non-viral gene therapy vectors for HGG.
• Analysis of preclinical data supporting gene therapy in HGG models.
• Examination of safety profiles and efficacy markers from clinical studies.

Main Results:

• Gene therapies reviewed have demonstrated safety in clinical trials.
• Tumor cell lysis, increased immune cell infiltration, and enhanced systemic immune function were observed.
• Oncolytic HSV vectors and PVSRIPO showed promising results, with some studies indicating survival improvements in recurrent HGG.

Conclusions:

• Gene therapy is a safe and promising approach for HGG treatment.
• While significant survival benefits are yet to be realized, ongoing trials with oncolytic viruses show potential.
• Future research focusing on novel administration protocols and combination therapies is expected to improve HGG prognosis.