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Clinical Trials

Taking part in a clinical trial may be the best option for some chronic myeloid leukemia (CML) patients. Clinical trials are designed to be accurate and very safe. There are clinical trials for newly diagnosed patients, for patients with advanced disease, and for patients who are either intolerant to or resistant to their current medications. The Leukemia & Lymphoma Society continues to invest funds in CML research.

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Current CML Research and Clinical Trials

Current goals of CML research are to

  • Develop truly curative therapies
  • Develop therapies that can work when others have failed
  • Decrease the side effects of treatment

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The following approaches are under study in clinical trials for the treatment of patients with CML.

Improving Current Treatments. Based on the positive results of tyrosine kinase inhibitor (TKI) therapy in chronic phase CML, many trials are looking at ways to further optimize the use of these drugs. This research includes

  • Determining which chronic-phase patients should receive which TKI as initial therapy
  • Establishing the best time to switch patients to second-line therapy
  • Finding out whether deeper responses are achieved when other agents are added to TKIs
  • Preventing and/or predicting long-term side effects of TKIs
  • Determining which patients can successfully discontinue TKI therapy 

New Drug Therapies and Drug Combinations. 

  • Asciminib (ABL001) is an investigational tyrosine kinase inhibitor (TKI) that binds to the ABL1 portion of the BCR-ABL1 fusion protein at a location that is distinct from the ATP-binding domain.
  • Ruxolitinib (Jakafi®) is a pan-Janus kinase inhibitor. It is already approved to treat patients who have been diagnosed with myelofibrosis or polycythemia vera.
  • Ipilimumab (Yervoy®) is a monoclonal antibody and also an immune checkpoint inhibitor that is currently being evaluated.
  • A tyrosine kinase inhibitor in combination with interferon alpha. Several studies have shown improved molecular response rates in CML patients using this combination.

TKI Discontinuation Studies. Treatment of CML with TKIs has advanced to a point where many patients are able to achieve deep and durable remissions. The feasibility and safety of discontinuing TKI therapy, along with close monitoring of carefully selected patients who have achieved and maintained a deep molecular response (DMR) for at least two years, continues to be evaluated in several long-term studies. (TKI discontinuation can also occur outside of a clinical trial, under certain circumstances.) 
                                         
Reduced-intensity Stem Cell Transplantation. This modified form of allogeneic transplantation (also known as nonmyeloablative allogeneic stem cell transplantation), may be an option for CML patients who do not respond to other treatments. Patients being prepared for a reduced-intensity transplant receive lower doses of chemotherapy drugs and/or radiation therapy in preparation for the transplant, compared to the doses given to patients receiving a traditional allogeneic stem cell transplant. The theory being tested with a Chronic Myeloid Leukemia I 41 reduced-intensity transplant is that: 1) by undergoing less toxic procedures prior to the transplant, the body is better able to withstand the transplant; and 2) full donor engraftment and the desired graft-versus-leukemia effect would still occur. Ongoing clinical trials are evaluating the use of this type of stem cell transplantation in adult and pediatric patients. For more information about all types of stem cell transplantation, see the free LLS booklet, Blood and Marrow Stem Cell Transplantation.


For information about the drugs listed on this page, visit Drug Listings.


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