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. This medication is currently under study in clinical trials in combination with other TKIs, such as imatinib and dasatinib.
• Ruxolitinib (Jakafi®) is a pan-Janus kinase inhibitor that is already approved to treat patients who have been diagnosed with myelofibrosis or polycythemia vera. This drug is being studied in clinical trials in combination with TKIs.
• Ipilimumab (Yervoy®) is a monoclonal antibody and also an immune checkpoint inhibitor that is currently being evaluated, in combination with dasatinib, in patients with chronic or accelerated phase CML.
• Tyrosine kinase inhibitor in combination with pioglitazone. Pioglitazone is a drug used to treat type 2 diabetes. In early studies, it has shown the ability to induce cell death in leukemic cells when combined with a TKI.
• Tyrosine kinase inhibitor in combination with interferon alpha. Several studies have shown improved molecular response rates when using this combination, compared with imatinib alone.

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.)
 
WT1 Peptide Vaccine. The Wilms Tumor 1 (WT1) protein is expressed in CML, acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) cells, making this protein a good target for immunotherapy. A WT1 peptide vaccine is currently under study in clinical trials for CML to test whether the vaccine can increase the number of immune cells responding to the cancer and thereby slow progression of the disease. See the free LLS booklet Immunotherapy Facts for information about the development of blood cancer vaccines. See the free LLS booklet, Immunotherapy Facts for information about the development of blood cancer vaccines.
                                           
Reduced-intensity Stem Cell Transplantation. A modified form of allogeneic transplantation known as “reduced-intensity” or “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 in preparation for the transplant, compared to the doses given to patients receiving a traditional allogeneic transplant. The theory being tested with a reduced-intensity transplant is that by undergoing less toxic procedures prior to the transplant, the body is better able to withstand the transplant. However, full donor engraftment would ideally still take place, and the desired graft-versus-leukemia effect would still occur. Ongoing clinical trials are evaluating the use of this type of 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.

Related Links

• Clinical  Trials
• Download or order The Leukemia & Lymphoma Society's free booklet, Chronic Myeloid Leukemia