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Published on: October 27, 2014
ON-01, an engineered recombinant oncolytic herpes simplex virus type-1, in recurrent glioma: A single-arm, phase 1/2
Wei Zeng1,2,3, Peiwen Wang1,4,3, Sheng Fang1,4,3
1Brain Tumor Research Center, Beijing Neurosurgical Institute, Capital Medical University, Beijing, People's Republic of China.
Background:
The prognosis of patients with recurrent WHO grade 4 glioma is poor, particularly in glioblastoma, which has a median survival of approximately 6 months and no effective treatment options. We evaluated the short-term (28-day) safety and efficacy of ON-01, an engineered recombinant oncolytic herpes simplex virus type-1, in patients with recurrent WHO grade 4 glioma.
Methods:
In this single-arm, phase 1/2 clinical trial, eligible patients received intratumoral injections of ON-01 under stereotactic guidance. The primary endpoint was to assess the short-term safety profile of ON-01 treatment. Secondary endpoints included progression-free survival (PFS), overall survival (OS), and the 2-year OS rate. An exploratory objective was to identify tumor-related biomarkers predictive of treatment efficacy.
Results:
Of the 30 patients treated with ON-01, 13 (43.3%) were male, and the median age was 50.0 years (range, 22-75). A total of 36 grade 1, 12 grade 2, and 2 grade 3 adverse events were reported. Among all treated patients, the median OS was 12.0 months (95% CI, 10.1-13.9), median PFS was 3.0 months (95% CI, 1.7-4.3), and 2-year OS rate was 27.7% (95% CI, 12.6%-45.0%). Seven patients with recurrent multifocal gliomas demonstrated regression of non-injection site lesions following ON-01 therapy. Furthermore, patients with elevated expression of herpesvirus entry mediator exhibited significantly prolonged survival (P = .015).
Conclusions:
Intratumoral infusion of ON-01 appeared safe and demonstrated efficacy in patients with recurrent malignant glioma, with no evidence of neurotoxicity. The therapeutic response to ON-01 may be associated with HVEM expression levels.
Insights
ON-01, an oncolytic herpes simplex virus, shows promise for recurrent malignant glioma. This therapy is safe and effective, potentially linked to herpesvirus entry mediator expression.
Area of Science:
- Oncology
- Virology
- Gene Therapy
Background:
- Recurrent WHO grade 4 glioma, including glioblastoma (GBM), has a poor prognosis with limited treatment options.
- ON-01 is an engineered recombinant oncolytic herpes simplex virus type-1 designed for cancer therapy.
Purpose of the Study:
- To evaluate the short-term safety and efficacy of ON-01 in patients with recurrent WHO grade 4 glioma.
- To explore biomarkers predictive of ON-01 treatment efficacy.
Main Methods:
- A single-arm, phase 1/2 clinical trial involving intratumoral injections of ON-01 under stereotactic guidance.
- Primary endpoint: short-term safety profile. Secondary endpoints: progression-free survival (PFS), overall survival (OS), and 2-year OS rate.
Main Results:
- Median OS was 12.0 months, median PFS was 3.0 months, and the 2-year OS rate was 27.7% in 30 treated patients.
- Seven patients with recurrent multifocal gliomas showed regression of non-injection site lesions.
- Elevated herpesvirus entry mediator (HVEM) expression correlated with significantly prolonged survival (p=0.015).
Conclusions:
- Intratumoral ON-01 infusion is safe and effective in recurrent malignant glioma patients, with no observed neurotoxicity.
- Therapeutic response to ON-01 may be associated with HVEM expression levels.
