Table 1.
Experimental and available LSC-directed therapies
| Target | Agents | Leukemic type | Phase of clinical development | Reference |
|---|---|---|---|---|
| CD33 | Gemtuzumab ozogamicin (Mylotarg)* | AML | In clinical use | (55, 54) |
| Telomeres and telomerase | Arsenic trioxide (Trisenox)* | APL | In clinical use | (17, 18) |
| GRN163L (hTERC inhibitor) | CLL | I/II | (19) Recruiting: NCT00124189 | |
| hTERT vaccine (TVAX) | Hematologic malignancies | I | (20, 21) | |
| Epigenetics (BMI-1) | Vorinostat (Zolinza)† | AML | II | Recruiting: NCT00305773 |
| Azacitidine (Vidaza)† | CML | II | (56) | |
| Decitabine (Dacogen)† | AML, ALL, CML | I | (57) | |
| FLT3 | CEP701 (Lestaurtinib) | AML | II | (45) |
| mTOR | PKC412 everolimus | AML hematologic malignancies | II | (58) |
| Temsirolimus | CML and CLL | I/II | (49) | |
| Rapamycin† | AML | I and II | Recruiting: NCT00101088, NCT00290472 | |
| Preclinical | (48) | |||
| P-Glycoprotein | Zosuquidar | Advance malignancies | I/II | Recruiting: NCT00129168, NCT00233909 |
| I | (53) | |||
| γ-Secretase (Notch) | MK-0752 | T-cell ALL and other leukemia | I | (26) |
| NF-κB | NPI-0052 (salinosporamides A) | Hematologic malignancies: NHL, CLL, and Ph+ ALL | I | (35, 59) |
| TDZD-8 (parthenolide) | Leukemia | Preclinical | (36) |
Abbreviations: APL, acute promyelocytic leukemia; CLL; chronic lymphocytic leukemia; ALL, acute lymphoblastic leukemia; CML, chronic myeloid leukemia; NHL, non-Hodgkin’s lymphoma.
*
Food and Drug Administration approved for treatment of leukemia.
†
Food and Drug Administration approved for different indication.