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New TKI Therapy Shows Promise for Patients with Recurrent CML

New TKI Therapy Shows Promise for Patients with Recurrent CML

Published on December 14, 2020

Clinical Trial Sees Promising Results for TKI Therapy in CML

Patients with recurring chronic myeloid leukemia (CML) may need to try several therapies to achieve a good long-term response. A clinical trial announced during the 62nd American Society of Hematology (ASH) Annual Meeting and Expo had promising news for patients who have been through several lines of therapy and need new options. This year’s meeting was held online only due to the continued challenges caused by the novel coronavirus (COVID-19).

In a phase III trial called ASCEMBL, the drug asciminib was nearly twice as effective as a standard treatment regimen for patients with recurrent CML who have already gone through two courses of tyrosine kinase inhibitors (TKIs). TKIs directly block the BCR-ABL protein that causes this disease. TKIs currently used to treat CML include:

  • imatinib (Gleevec)
  • dasatinib (Sprycel)
  • nilotinib (Tasigna)
  • bosutinib (Bosulif)
  • ponatinib (Iclusig)

“Asciminib may provide a good opportunity for a third-line treatment in CML patients,” said senior study author Dr. Andreas Hochhaus of University Hospital in Jena, Germany. “The ASCEMBL data shows that asciminib has a reduced rate of side effects that lead to discontinuation or dose adjustment compared to the TKI drug bosutinib while improving the response rate and speed of response,” Dr. Hochhaus said in a news release.


The study accrued 233 patients who were randomly selected to receive asciminib (157 patients) or bosutinib (76 patients). Patients who had already been through three or more lines of therapy were placed in the asciminib arm of the study.

The research team followed up with patients at 24 weeks to evaluate how they were doing. Nearly 26 percent of patients in the asciminib arm met the primary endpoint of the study and experienced major molecular response (MMR), which means that the cancer cells had been reduced to very low levels. Only 13.2 percent of those in the bosutinib arm experienced MMR. Patients who did not do well in the bosutinib arm either discontinued treatment or were moved to the asciminib arm.

It appears that those taking asciminib were also twice as likely to achieve a deep molecular response, an indicator of more thorough eradication of cancer cells.

Side Effects of Asciminib

Gastrointestinal complications such as diarrhea were the leading side effect of asciminib, followed by nausea, vomiting and rash. Fewer patients experienced thrombocytopenia (low platelet levels) and neutropenia (low level of white blood cells). A handful of patients experienced heart problems. If at any point patients felt that the side effects were too much to handle, they had the option to discontinue treatment.

During his presentation at the online conference, Dr. Hochhaus concluded that “The ASCEMBL trial data supports the use of asciminib as a new treatment option in CML, particularly in patients with resistance or intolerance to at least two TKIs.”

Of course, none of this would be possible without the generosity of the CML patients who are willing to take part in practice-changing research. “We thank the patients participating in this study,” Dr. Hochhaus said.

The survival rate for CML has improved significantly in recent years — patients and their care partners have reason to be hopeful for the future.

~Lauren Evoy Davis


1Asciminib Found Safe and Effective for Hard-to-Treat Chronic Myeloid Leukemia. American Society of Hematology.

2Late-Breaking Abstracts Session. ASH 62nd Annual Meeting and Expo. December 8, 2020.

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