Temozolomide is marketed as TEMODAR, an alkylating antineoplastic drug used in adults with newly diagnosed glioblastoma (with radiotherapy then maintenance) and anaplastic astrocytoma settings. It is a prodrug that converts nonenzymatically to MTIC, which alkylates DNA and induces tumor cell death. U.S. initial approval was in 1999.
A phase I trial and a preclinical study found viral immunotherapy triggered T cell responses in glioblastoma. The clinical study enrolled 41 patients; the mouse study showed prolonged survival and immunologic memory.
WHO in January 2024 launched its first list of priority childhood cancer drug formulations, naming six medicines that still lack optimal child-friendly versions. The agency says about 400,000 children and adolescents develop cancer each year and is working to expand access to safe, effective paediatric formulations.
Cabozantinib plus metronomic temozolomide showed a 15% response rate and 28.5-month median progression-free survival in a phase 2 NET study. A separate analysis found cabozantinib cost-effective for pancreatic NETs, but not extrapancreatic NETs, in China and the U.S.
Researchers have developed new immunotherapy approaches including internal immune cell reprogramming and CD40-based therapies showing promising results in early trials. Meanwhile, glioblastoma remains resistant to current immunotherapies due to blood-brain barrier constraints, though advances in antibody engineering offer new potential strategies.
The blood-brain barrier continues to block most treatments for glioblastoma and central nervous system diseases, with only 0.01% of biologic drugs reaching the brain. New intellectual property coverage and synthetic methods aim to address delivery challenges.
Researchers have developed a remote-controlled CAR T-cell therapy that can be switched off on demand using venetoclax, while new engineering strategies aim to overcome barriers in treating glioblastoma and other solid tumors.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07569042 |
A Rollover Study of NBM-BMX in Combination With Temozolomide in Patients With Newly Diagnosed Glioblastoma |
RECRUITING | PHASE1/PHASE2 |
| NCT07492680 |
A Study of BMS-986504 Monotherapy and in Combination With Other Agents in Participants With Advanced and/or Metastatic Solid Tumors With Homozygous MTAP Deletion (MountainTAP-5) |
NOT_YET_RECRUITING | PHASE2 |
| NCT07457307 |
Integrating Biology for Safe Intensification of Glioblastoma Hypofractionated Treatment on the MR-Linac |
NOT_YET_RECRUITING | PHASE1 |
| NCT07452458 |
Temporally-Modulated Pulsed Radiation Therapy Versus Standard Radiation Therapy for the Treatment of Newly Diagnosed, IDH Wildtype, MGMT-Unmethylated Glioblastoma |
NOT_YET_RECRUITING | PHASE3 |
| NCT07347210 |
Evaluation of UCPVax Vaccine +/- Pembrolizumab Combined With Standard Treatment as Adjuvant Therapy in Patients With Unmethylated MGMT Glioblastoma |
NOT_YET_RECRUITING | PHASE2 |
| NCT07297212 |
A Clinical Trial Testing the Safety of the Investigational Drug Pumitamig (BNT327) and How Well it Works in Patients With Recurrent Glioblastoma |
RECRUITING | PHASE2 |
| NCT07223034 |
A Study of 177Lu-PSMA-617 in People With Gliomas |
RECRUITING | PHASE1 |
| NCT07215910 |
Testing Addition of an Anti-cancer Drug, Vorasidenib to Temozolomide, After Radiation for Advanced Brain Cancer |
NOT_YET_RECRUITING | PHASE3 |
| NCT07195591 |
Beginning Radiation Immediately With GammaTile at GBM Excision Versus Standard of Care |
RECRUITING | PHASE3 |
| NCT07194044 |
Metastatic Ewing's Trial Testing Schedule Enhancement to Improve Outcomes |
RECRUITING | PHASE1 |
