Target | Drug (s) | Trial design | Study population | Outcome | Ref |
---|---|---|---|---|---|
EGFR | Erlotinib | Randomised, controlled, phase II #26032 | 110 Recurrent GBM | 6-month PFS: 11.4% vs 24% control, Low akt borderline significance | [34] |
 | Erlotinib (+RT+TMZ) | Phase I/II cf historical controls #N0177 | 97 newly diagnosed GBM | Median survival 15.3 months, no benefit at OS | [41] |
 | Erlotinib (+RT +TMZ) | Phase II cf historical controls | 65 newly diagnosed GBM/gliosarcoma | Median survival 19.3 months vs 14.1 controls, positive correlation MGMT methylation with survival and MGMT methylation + PTEN positivity with improved survival | [40] |
 | Erlotinib (+RT +TMZ) | Phase II | 27 newly diagnosed GBM | OS 8.6 months median PFS 2.8 months | [42] |
 | Erlotinib (+RT+TMZ) | Phase II #NCT00187486 | Newly diagnosed GBM/gliosarcoma | Ongoing http://www.clinicaltrials.gov |  |
 | Erlotinib single-agent | Phase II open-label, multicenter #NCT00337883 | First Relapse GBM | Completed http://www.clinicaltrials.gov |  |
 | Gefitinib | Phase II | 53 Recurrent GBM | 31 patients had radiographic progressive disease within the first 2 months, 51 progressed eventually median EFS: 8.1 weeks | [53] |
 | Erlotinib +RT+TMZ) | Phase II #NCT00274833 | Newly diagnosed GBM | Ongoing http://www.clinicaltrials.gov |  |
Akt | Perifosine | Phase II #NCT00590954 | Recurrent/progressive Malignant Gliomas | Ongoing http://www.clinicaltrials.gov | Â |
 | Perifosine and Temsirolimus | Phase I/II #NCT01051557 | Recurrent/progressive Malignant Glioma | Planned, not yet recruiting http://www.clinicaltrials.gov |  |
 | Nelfinavir (+TMZ +RT) | Phase I/II #NCT00694837 | Newly diagnosed GBM | Recruiting http://www.clinicaltrials.gov |  |
PI3K/mTOR | XL765 +TMZ | Phase I #NCT00704080 | Adults Malignant Gliomas | Â | Â |
mTOR | Temsirolimus (CCI-779) | Phase II | 65 Recurrent GBM | 6-month PFS: 7.8% median OS 4.4 months, high levels phospho p70s6K appear to predict benefit of treatment | [60] |
 | Temsirolimus | Phase II | 43 Recurrent GBM | No evidence of efficacy, 1 patient PF at 6-month: 2 PR, 20 SD, median time to progression 9 weeks | [59] |
 | Temsirolimus (+TMZ+RT) | Phase I #NCT00316849 | newly diagnosed GBM | Recruiting http://www.clinicaltrials.gov |  |
 | Temsirolimus (+ Erlotinib+ Tipifarnib) | Phase I/II #NCT00335764 | recurrent GBM/gliosarcoma. | Recruiting http://www.clinicaltrials.gov |  |
 | Everolimus + gefitinib | Phase I/II #NCT00085566 | Progressive GBM | Ongoing http://www.clinicaltrials.gov |  |
 | Everolimus | Phase I/II Pilot, Multicenter #NCT00515086 | Recurrent GBM | Completed, Decemeber 2009 |  |
 | Everolimus +AEE788 | Phase IB/II multicenter, two-Arm, dose-escalation | Recurrent GBM | Ongoing http://www.clinicaltrials.gov |  |
Bcl-2 | Gossypol | Phase II #NCT00540722 | Recurrent GBM | Ongoing http://www.clinicaltrials.gov | Â |
 | Gossypol (AT-101) +RT +TMZ vs AT-101 +Adjuvant TMZ | Phase I non-randomised #NCT00390403 | Newly diagnosed GBM | Completed, June 2009 |  |