Skip to main content
. Author manuscript; available in PMC: 2023 Jul 21.
Published in final edited form as: Nat Rev Neurol. 2022 Mar 11;18(4):221–236. doi: 10.1038/s41582-022-00621-0

Table 3 |.

Clinical trials in high-g rade glioma: localized immunotherapy

Study Treatment Study type Patient cohort Results Adverse events (number of patients)
Viral therapy
Wheeler et al. (2016)87 AdvHSV-tk injection in resection cavity wall + ganciclovir with standard of care Phase II, with historical controls Primary glioblastoma (n = 48) 3 × 1011 particles well tolerated; median survival, 17.1 months vs 13.5 months in historical controls; median OS after gross total resection, 25.0 vs 16.9 months Worsening of existent hemiparesis (1)
Ji et al. (2016)88 Repeated dosing of intra-arterial AdvHSV-tk with BBB disruption and standard of care vs standard of care Phase II Recurrent WHO grade III or IV glioma (n = 53) Median OS, 10.4 months in AdvHSV-tk group vs 3.3 months in controls; median PFS, 6.8 vs 1.9 months; treatment of controls (surgery, chemotherapy or palliative care) not stated No increased risk of adverse events
Chiocca et al. (2019)89 Ad-RTS-hIL-12 in resection cavity wall + systemic veledimex Phase I Recurrent glioblastoma (n = 31) Maximum tolerated dose of veledimex, 20 mg; median survival, 12 months Reversible cytokine release syndrome (10 grade 2, 6 grade 3), brain oedema (1), confusion (1), aseptic meningitis (1)
Desjardins et al. (2018)96 Single dose of PVSRIPO via CED Phase I Recurrent glioblastoma (n = 61) Median OS, 12.5 months vs. 11.3 months in historical controls Haemorrhage leading to hemiparesis (1), peritumoural inflammation requiring bevacizumab (32), requiring surgery (4)
Lang et al. (2018)98 DNX-2401 in resection cavity wall Phase I Recurrent glioblastoma (n = 37) 20% of patients survived >3 years, three showed >95% tumour reduction; OS, 13.0 months Transient fever, headache and malaise (2)
Markert et al. (2014)102 Single stereotactic injection of G207 combined with radiotherapy Phase I Recurrent glioblastoma (n = 9) Six patients showed stabilization of disease or partial response; median OS, 7.5 months; median PFS, 2.5 months Seizures and hemiparesis (number of patients unclear)
Friedman et al. (2021)104 Single injection of G207 via CED combined with radiotherapy Phase I Paediatric recurrent high-grade glioma (n = 12) Radiological and/or clinical response in 11 patients; median OS, 12.2 months No dose-l imiting adverse effects
Cytokine and antibody therapy
Kunwar et al. Single injection of (2010)50 IL13-Pe38QQR via CED vs carmustine wafers Phase III Recurrent glioblastoma (n = 296) Median OS, 9.1 months in IL13-Pe38QQR group vs 8.8 months in controls Increased incidence of pulmonary embolism in IL13-Pe38QQR group (16 vs 2 patients)
Immunostimulating oligodeoxynucleotides
Carpentier et al. (2010)111 Single stereotactic injection of CpG-O DN Phase II Recurrent glioblastoma (n = 31) One partial response and three minor responses; median OS, 6.4 months; median PFS, 2.1 months Transient worsening of neurological deficits (22), death owing to haemorrhage (1)
Ursu et al. (2017)112 CpG-ODN injected into resection cavity wall followed by standard of care vs standard of care only Phase II Primary glioblastoma (n = 81) Median OS, 17 months in CpG-ODN group vs 18 months in controls; median PFS, 9 months in both groups Increased occurrence of fever and postoperative haemorrhage

BBB, blood–brain barrier; CED, convection-enhanced delivery; CpG-ODN, oligodeoxynucleotides containing unmethylated cytosine–guanosine motifs; OS, overall survival; PFS, progression-free survival; PVSRIPO, recombinant non-pathogenic polio–rhinovirus chimaera.