Table 1.
Summary of molecularly targeted agents in clinical development for childhood acute leukemia.
| Class | Molecular target | Agent(s) | Disease | Subset | Phase |
|---|---|---|---|---|---|
| Monoclonals | CD33 | Gemtuzumab ozogamicin | AML | III | |
| CD22 | Epratuzumab | ALL | CD22+ | II | |
| TKIs | BCR–ABL | Imatinib, dasatinib | ALL | Ph+ | II |
| FLT3 | Lestaurtinib | AML | FLT3 mutated | II | |
| ALL | MLL rearranged | II | |||
| Proteasome inhibitors | NF-κB | Bortezomib | AML, ALL | I | |
| mTOR inhibitors | mTOR | Sirolimus, temsirolimus | AML, ALL | I | |
| Epigenetic modulators | HDAC | Vorinostat, entinostat | AML, ALL | I | |
| DNA methyltransferase | Azacitidine, decitabine | AML, ALL | I | ||
| FTI | Farnesyltransferase | Tipifarnib | AML, ALL | RAS (or upstream) mutated | I |
| Notch inhibitors | γ-secretase | MK0752, LY450139 | ALL | T-cell, Notch-mutant | I |
ALL: Acute lymphoblastic leukemia; AML: Acute myeloid leukemia; FTI: Farnesyltransferase inhibitor; HDAC: Histone deacetylase; NF: Nuclear factor; Ph: Philadelphia chromosome; TKI: Tyrosine kinase inhibitor.