Table 1 FDA-approved drugs for neuroblastoma

From: Molecular principles underlying aggressive cancers

Drug name (brand name)

Drug type (PubChem CID)

Cancer treatment

Mechanism of action

Route of administration

Cyclophosphamide (Cytoxan, Procytox)

Small molecule (2907)

Neuroblastoma, ALL, AMoL, AML, CLL, CML, breast cancer, HL, MM, MF, neuroblastoma, NHL, ovarian cancer, retinoblastoma

Precursor of alkylating agents of metabolite PM, leading to DNA cross-linking and cell apoptosis

Oral or intravenous

Dinutuximab (Unituxin)

Monoclonal antibody

Neuroblastoma

Binds to GD2 on the surface of cancer cells, triggering an immune response

Intravenous

Doxorubicin hydrochloride (Adriamycin, Doxil, Caelyx, Rubex, Myocet)

Small molecule (443939)

Neuroblastoma, ALL, AML, breast cancer, gastric cancer, HL, NHL, NSCLC, soft tissue and bone sarcomas, thyroid cancer, transitional cell bladder cancer, Wilms tumor

Interacting with DNA interferes with Topo II activity and prevents relegation of Topo-mediated DNA breaks, leading to inhibition of replication and transcription and induction of apoptosis.

Intravenous

Eflornithine hydrochloride or DFMO (Iwilfin, Vaniqa)

Small molecule (57004)

Neuroblastoma

Inhibits ODC, an enzyme that synthesizes a polyamine required for tumor formation and growth

Intravenous

Naxitamab-gqgk (Danyelza)

Monoclonal antibody

Neuroblastoma

Binds to GD2 on the surface of cancer cells, triggering an immune response

Intravenous

Vincristine sulfate (Oncovin, Vincasar, Marqibo, Vincrex)

Small molecule (249332)

Neuroblastoma, AL, HL, NHL, RMS, Wilms tumor

Inhibits microtubule formation in the mitotic spindle, resulting in cell division arrest at the metaphase stage

Intravenous

  1. AMoL acute monocytic leukemia, DFMO difluoromethylornithine, GD2 glycolipid GD2, HL Hodgkin lymphoma, MF mycosis fungoides, MM multiple myeloma, NHL non-Hodgkin lymphoma, ODC ornithine decarboxylase, PM phosphoramide mustard, RMS rhabdomyosarcoma, Topo II topoisomerase II