Novel therapies in advanced cml under investigation
| Drug class . | Clinical trials . | N . | Response . |
|---|---|---|---|
| Asciminib (BCR-ABL TKI) | Hughes et al41 (phase 1—asciminib in CP/AP CML after TKI failure) NCT02081378 (phase 1—asciminib ± TKI in CP/AP/BP CML after TKI failure) NCT03595917 (phase 1—asciminib + dasatinib + prednisone in Ph+ ALL/CML lymphoid BP) | N = 9 (AP) | CHR 8/9 (AP) MMR 1/9 (AP) |
| HQP1351(BCR-ABL TKI) | Jiang et al43 (phase 2—HQP1351 in T315I-mutated CP/AP CML) | N = 23 (AP) | MHR 78% (AP) MCyR 52% (AP) |
| K0706 (BCR-ABL TKI) | NCT02629692 (phase 1/2—K0706 in AP/CP/BP CML after TKI failure) | ||
| PF-114 (BCR-ABL TKI) | Turkina et al44 (phase 1—PF-114 CP/AP CML after TKI failure or T315I) | N = 51 (CP/AP) | MHR 42% (CP/AP) MCyR 29% (CP/AP) |
| PHA-739358 (aurora kinase inhibitor) | Borthakur et al45 (phase 1—PHA-739358 in AP/BP CML) | N = 29 (AP/BP) | HR 14% (AP/BP) |
| SCH 6636 (farnesyl transferase inhibitor) | Cortes et al46 (phase 1—SCH 6636 CP/AP/BP CML after imatinib failure) | N = 15 (AP/BP) | CHR 14% (AP/BP) |
| Venetoclax (BCL2 inhibitor) | Maiti et al42 (retrospective—venetoclax + TKI) | N = 9 (BP) | ORR 75% (BP) |
| BP1001 (liposomal Grb-2 antisense oligonucleotide) | Ohanian et al47 (phase 1/1b—BP1001 ± low-dose cytarabine in advanced myeloid malignancies) | N = 5 (BP) | ORR 1/5 (BP) |
| Nivolumab (anti–PD-1) | NCT02011945 (phase 1b—nivolumab + dasatinib in CP/AP CML) | ||
| Inotuzumab (anti-CD22) | Jain et al48 (phase 1/2—inotuzumab+bosutinib in Ph+ ALL/CML lymphoid BP) | N = 2 (BP) | ORR 1/2 (BP) |
| Drug class . | Clinical trials . | N . | Response . |
|---|---|---|---|
| Asciminib (BCR-ABL TKI) | Hughes et al41 (phase 1—asciminib in CP/AP CML after TKI failure) NCT02081378 (phase 1—asciminib ± TKI in CP/AP/BP CML after TKI failure) NCT03595917 (phase 1—asciminib + dasatinib + prednisone in Ph+ ALL/CML lymphoid BP) | N = 9 (AP) | CHR 8/9 (AP) MMR 1/9 (AP) |
| HQP1351(BCR-ABL TKI) | Jiang et al43 (phase 2—HQP1351 in T315I-mutated CP/AP CML) | N = 23 (AP) | MHR 78% (AP) MCyR 52% (AP) |
| K0706 (BCR-ABL TKI) | NCT02629692 (phase 1/2—K0706 in AP/CP/BP CML after TKI failure) | ||
| PF-114 (BCR-ABL TKI) | Turkina et al44 (phase 1—PF-114 CP/AP CML after TKI failure or T315I) | N = 51 (CP/AP) | MHR 42% (CP/AP) MCyR 29% (CP/AP) |
| PHA-739358 (aurora kinase inhibitor) | Borthakur et al45 (phase 1—PHA-739358 in AP/BP CML) | N = 29 (AP/BP) | HR 14% (AP/BP) |
| SCH 6636 (farnesyl transferase inhibitor) | Cortes et al46 (phase 1—SCH 6636 CP/AP/BP CML after imatinib failure) | N = 15 (AP/BP) | CHR 14% (AP/BP) |
| Venetoclax (BCL2 inhibitor) | Maiti et al42 (retrospective—venetoclax + TKI) | N = 9 (BP) | ORR 75% (BP) |
| BP1001 (liposomal Grb-2 antisense oligonucleotide) | Ohanian et al47 (phase 1/1b—BP1001 ± low-dose cytarabine in advanced myeloid malignancies) | N = 5 (BP) | ORR 1/5 (BP) |
| Nivolumab (anti–PD-1) | NCT02011945 (phase 1b—nivolumab + dasatinib in CP/AP CML) | ||
| Inotuzumab (anti-CD22) | Jain et al48 (phase 1/2—inotuzumab+bosutinib in Ph+ ALL/CML lymphoid BP) | N = 2 (BP) | ORR 1/2 (BP) |
BCL2, B-cell lymphoma 2; HR, hematologic response; MCyR, major cytogenetic response; MHR, major hematologic response; ORR, overall response rate.