Table 2.
Investigational therapy | Pharmacologic classificatic | Indication | Stage of development | Overall response rate | Adverse effects |
---|---|---|---|---|---|
Clofarabine [36–38] | Nucleoside analogue | ALL/AML | FDA-approved in pediatric ALL; phase 2 (adults) |
13% | Hepatotoxicity, rash, drug fever, palmoplantar erythrodysesthesia |
Hyper-CVAD + dasatinib*
[49••] |
Chemotherapy + tyrosine kinase inhibitor |
Philadelphia chromo- some– positive ALL |
Phase 2 | 100% in combination with chemotherapy |
Bleeding, pleural and pericardial effusion, liver dysfunction |
Sphingosomal vincristine [40,41•,42] |
Liposome-encapsulated vinca alkaloid |
ALL | Phase 2 | 14% | Peripheral neuropathy, orthostasis, headache |
Nelarabine [33] | Nucleoside analogue | T-cell ALL | FDA-approved | 41% | Peripheral sensory neuropathy, seizure, fatigue, muscle weakness, gastrointestinal distress |
Pegaspargase* [44,45] | Pegylated asparaginase | ALL | FDA-approved in pediatric ALL |
96% in combination with chemotherapy |
Hypersensitivity reactions, liver dysfunction, hyperglycemia, coagulation factor abnormalities, pancreatitis, cerebral dysfunction |
Responses reported in combination with chemotherapy.
ALL—acute lymphoblastic leukemia; AML—acute myelogenous leukemia; FDA—US Food and Drug Administration; hyper-CVAD—vincristine, doxorubicin, and dexamethasone with hyper-cyclophosphamide.