Temozolomide

|drug_name=Temozolomide (TMZ) |FDA_approval=Yes |used_for=Glioblastoma Multiforme (GBM) |clinical_trial_phase=Approved; ongoing trials exploring new combinations |common_side_effects=Nausea, vomiting, constipation, loss of appetite, fatigue, headache, hair loss, low blood cell counts |OS_with=Median survival of 14.6 months; 2-year survival rate approximately 27% |usefulness_rating=4 |usefulness_explanation=Temozolomide is the standard chemotherapy for GBM and has been shown to extend survival when used in combination with radiotherapy. While not curative, it provides a significant survival benefit, particularly for patients with MGMT methylation. Its oral administration makes it more convenient than intravenous chemotherapies, and its side effects are generally manageable. However, its effectiveness is limited in patients with unmethylated MGMT, and resistance often develops over time. |treatment_category=Standard of Care (SOC) Chemotherapy |toxicity_level=3 |toxicity_explanation=While TMZ is generally well-tolerated, it can cause side effects such as nausea, vomiting, and hematological toxicities like neutropenia and thrombocytopenia. These side effects are typically manageable with supportive care. |notes=Temozolomide is the standard chemotherapy agent for GBM, often used in conjunction with radiotherapy. Its effectiveness is notably higher in patients with methylated MGMT promoters. Ongoing research is investigating alternative dosing schedules and combination therapies to enhance its efficacy. Temozolomide (TMZ) is an oral alkylating agent that has become the cornerstone of chemotherapy for glioblastoma multiforme (GBM). The landmark study by Stupp et al. demonstrated that the addition of TMZ to radiotherapy significantly improved median survival from 12.1 months to 14.6 months, with a 2-year survival rate increasing from 10.4% to 26.5%. The standard regimen involves daily TMZ administration during radiotherapy, followed by six cycles of maintenance therapy. Patients with methylation of the MGMT promoter gene derive the most benefit from TMZ treatment, as this epigenetic modification is associated with increased sensitivity to the drug. Ongoing research aims to optimize TMZ therapy through alternative dosing schedules and combination treatments to overcome resistance and improve patient outcomes. |overview=Temozolomide (TMZ) is an FDA-approved oral chemotherapy drug used primarily to treat glioblastoma multiforme (GBM). When combined with radiotherapy, TMZ has been shown to improve median survival to 14.6 months, with approximately 27% of patients surviving at two years. Its efficacy is enhanced in patients with methylated MGMT promoters. Common side effects include nausea, fatigue, and hematological toxicities, earning it a toxicity rating of 3. Research continues to explore new dosing regimens and combination therapies to further enhance its effectiveness.