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|Therapy Name||Cyclophosphamide + Fludarabine + Melphalan + Sirolimus|
|Drug Name||Trade Name||Synonyms||Drug Classes||Drug Description|
|Cyclophosphamide||Cytoxan||CPM||Chemotherapy - Alkylating 14||Cytoxan (cyclophosphamide) is an alkylating agent, which inhibits DNA replication (NCI Drug Dictionary). Cytoxan (cyclophosphamide) is FDA approved in multiple hematological malignancies, breast cancer, neuroblastoma, ovarian cancer, and retinoblastoma (NCI Drug Dictionary).|
|Fludarabine||Fludara||FAMP|Fludarabine phosphate||Flurdara (fludarabine) is converted to 2-fluoro-ara-ATP intracellularly, which potentially inhibits DNA polymerase alpha, ribonucleotide reductase and DNA primase, leading to decreased DNA synthesis and reduced tumor growth (NCI Drug Dictionary)|
|Melphalan||Alkeran||Chemotherapy - Alkylating 14||Alkeran (melphalan) is an antineoplastic alkylating agent, which cross-links DNA and induces cell toxicity and is FDA approved for multiple myeloma and epithelial ovarian carcinoma (FDA.gov).|
|Sirolimus||Rapamune||Rapamycin||mTORC1 Inhibitor 8||Rapamune (sirolimus) binds to the FKBP-12 to generate an immunosuppressive complex that binds and allosterically inhibits mTOR (PMID: 25261369). Rapamune (sirolimus) is FDA approved for the prevention of renal transplant rejection and for patients with lymphangioleiomyomatosis (FDA.gov).|
|Molecular Profile||Indication/Tumor Type||Response Type||Therapy Name||Approval Status||Evidence Type||Efficacy Evidence||References|
|Clinical Trial||Phase||Therapies||Title||Recruitment Status|
|NCT01804634||Phase II||Cyclophosphamide + Fludarabine + Melphalan + Sirolimus||A Phase II Trial of Reduced Intensity Conditioning and Haploidentical BMT for High-risk Solid Tumors||Recruiting|