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intrathecal chemotherapy
Intrathecal chemotherapy delivers chemotherapeutic agents directly into the cerebrospinal fluid to treat central nervous system malignancies and prevent leptomeningeal disease.
Intrathecal chemotherapy delivers chemotherapeutic agents directly into the cerebrospinal fluid to treat central nervous system malignancies and prevent leptomeningeal disease. Used for Prophylaxis and treatment of leptomeningeal disease in pediatric acute lymphoblastic leukemia, CNS involvement in pediatric lymphomas and other hematologic malignancies, Primary CNS tumors in pediatric patients.
At a glance
| Generic name | intrathecal chemotherapy |
|---|---|
| Also known as | Cytosine Arabinoside, Ara-C, Cytosar®, NSC #63878 (102004) |
| Sponsor | Princess Maxima Center for Pediatric Oncology |
| Drug class | Chemotherapy administration route (intrathecal) |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
Mechanism of action
This is an administration route rather than a single drug entity; chemotherapy agents (commonly methotrexate, cytarabine, or hydrocortisone) are injected directly into the intrathecal space via lumbar puncture or ventricular reservoir. This approach achieves high local concentrations in the cerebrospinal fluid while minimizing systemic exposure, allowing treatment of CNS-penetrating cancers and prophylaxis of meningeal involvement in hematologic malignancies.
Approved indications
- Prophylaxis and treatment of leptomeningeal disease in pediatric acute lymphoblastic leukemia
- CNS involvement in pediatric lymphomas and other hematologic malignancies
- Primary CNS tumors in pediatric patients
Common side effects
- Chemical arachnoiditis
- Neurotoxicity
- Headache
- Myelosuppression
- Infection at injection site
Key clinical trials
- Pediatric-Inspired Regimen Combined With Venetoclax and Immunotherapy for Adult Ph-Negative Acute Lymphoblastic Leukemia (NA)
- Lymphodepletion Plus Adoptive Cell Transfer With or Without Dendritic Cell Immunization in Patients With Metastatic Melanoma (PHASE2)
- BNHL-2015 for Children or Adolescents in China (PHASE2, PHASE3)
- Evaluation of Neuroinduced Mesenchymal Stem Cells and Mesenchymal Stem Cell-Derived Exosomes in Patients With Parkinsonism (PHASE2)
- Study of Revumenib, Azacitidine, and Venetoclax in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid Leukemia (PHASE1)
- ESP Block VS Intrathecal Opioid After Laparoscopic Colorectal Surgery (PHASE1, PHASE2)
- Cognitive Impairment in Patients With Diffuse Large B-Cell Lymphoma (NA)
- Pilot Study of IT Topotecan and Maintenance Chemotherapy for HR-EBTs in Children < 6 Years, Post Consolidation (EARLY_PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- intrathecal chemotherapy CI brief — competitive landscape report
- intrathecal chemotherapy updates RSS · CI watch RSS
- Princess Maxima Center for Pediatric Oncology portfolio CI