Where chronic myeloid leukemia treatment is heading

About 9,000 people in the US are diagnosed with chronic myeloid leukemia each year. Daily pills that block the BCR::ABL1 protein have turned it into a manageable condition: most people now have a near-normal life expectancy. The focus has shifted to pills with fewer side effects, options when resistance develops, and safely stopping treatment altogether.

• 128studies open or running
• 8phase 3 trials
• 716papers published this year
• 40NIH-funded projects

1. Now

   Nov 2026

   A better-tolerated first pillHappened 74.1% major molecular response

   Asciminib: 74.1% major molecular response at 2 years vs 52.0% with older pills, with fewer dose cuts.

   74.1%: major molecular response at 96 weeks, vs 52.0% with other pills.

   • ASC4FIRST trial, 2-year results

   Asciminib after earlier pills failHappened 33.8% major molecular response

   Three years in, 33.8% had a major molecular response vs 10.5% with bosutinib.

   33.8%: major molecular response at 3 years, vs 10.5% with bosutinib.

   • ASCEMBL trial, 4-year results

   Many people can stop their pillHappened 66.4% treatment-free remission

   After a year at half dose, 66.4% were off nilotinib and still in remission at 96 weeks.

   66.4%: in full treatment-free remission at 96 weeks.

   • Nilotinib de-escalation study

2. 6 months

   by May 2027

   Asciminib vs nilotinib on side effectsLikely 74.1% response (ASC4FIRST)

   ASC4START (568 people) compared how often people stopped asciminib or nilotinib because of side effects; results are expected.

   74.1%: major molecular response at 96 weeks, vs 52.0% with other pills.

   • ASC4FIRST trial, 2-year results
   • ASC4START

3. 12 months

   by Nov 2027

   A third-generation pill vs bosutinibPossible 61% cytogenetic response (early study)

   POLARIS-2 (333 people) compares olverembatinib with bosutinib after earlier pills; due June 2027.

   61%: complete cytogenetic response (chronic phase).

   • Olverembatinib US study
   • POLARIS-2

4. 3 years

   by late 2029

   Next-generation allosteric pillsPossible

   Enliven's ENABLE-2 (450 people) compares ELVN-001 with standard pills (due December 2028), and Merck's MK-4208 is in a 330-person study.

   • TERN-701 (MK-4208) phase 1 study
   • ENABLE-2
   • MK-4208 study

   Who can safely stop treatmentPossible

   A French trial (170 people) studies stopping tyrosine kinase inhibitors; due December 2028.

   • Nilotinib de-escalation study
   • Stopping trial

5. 5 years

   by late 2031

   Starting with asciminib to reach remission fasterLong shot

   The Italian PEARL trial (160 people) tests whether early asciminib gets more people to the deep response needed to stop treatment; due June 2029.

   • PEARL

The research paths

Open to join now

• Chronic phase, after earlier pills

  Olverembatinib vs bosutinib

  Phase 3 · 163 sites

  Locations and eligibility →

• Chronic phase, after earlier pills

  MK-4208 (TERN-701)

  Phase 1/2 · 54 sites

  Locations and eligibility →

• Newly diagnosed

  Asciminib vs nilotinib (PEARL)

  Phase 2 · 11 sites

  Locations and eligibility →

See every recruiting CML trial →

When the trials finish

Completion dates the trials registered. Results usually follow 3 to 12 months later.

What just happened

Standard treatment today

• Newly diagnosed, chronic phaseA daily pill: asciminib, imatinib, dasatinib, nilotinib or bosutinib.
• Deep remission for 2+ yearsTrying to stop treatment, with close molecular monitoring.
• Resistance or side effectsSwitch pill; asciminib or ponatinib after two pills; ponatinib or asciminib for the T315I mutation.
• Accelerated or blast phaseIntensive treatment, often followed by stem-cell transplant.

See CML trials recruiting now →