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Taking part in a clinical trial may be a treatment choice for some chronic myeloid leukemia (CML) patients. Clinical trials are under way to increase the remission rate of CML or cure the disease. Today's standard treatments for cancer are based on earlier clinical trials. The Leukemia & Lymphoma Society continues to invest funds in CML research.

Clinical trials can involve new drugs, new combinations of drugs or approved drugs being studied to treat patients in new ways such as new drug doses or new schedules to administer the drugs. Clinical trials are conducted worldwide under rigorous guidelines to help doctors find out whether new cancer treatments are safe and effective or better than the standard treatment.

Current CML Research and Clinical Trials

Scientists are conducting research strategies and clinical trials that hold the promise of increasing remission and cure rates of CML patients. These are some of the types of research and trials under way:

  • Disease eradication strategies. A number of laboratory studies have identified potential treatments that may help eradicate the few remaining CML cells in most patients treated with TKIs, and thus hopefully cure patients so that they may discontinue medical therapies altogether. One such area of interest involves inhibitors of a protein called "smoothened" (SMO) in combination with Bcr-Abl TKIs. As with all clinical advances, the participation of patients in clinical trials is crucial to determining whether such strategies are of clinical promise. Given that chronic phase CML is generally a slowly progressive disease even in the absence of effective therapy, it will likely be many years before it is known whether strategies aimed at disease eradication truly achieve disease cure.
  • Drug resistance. Drugs used to kill CML cells may work in some people but not in others. When the drugs don't work, it's called drug resistance. Scientists are trying to understand why some patients don't respond to Gleevec, Sprycel or Tasigna.
  • Treatment cessation. Careful clinical trials have begun to examine whether individuals who have deep remissions while taking therapy are able to sustain stable remissions after they stop therapy. Some patients with undetectable disease have interrupted treatment without evidence of disease recurrence during the subsequent two to three years, but it is not known if any of these patients are cured of their CML. More research is required in this area before any change can be confidently made to the current recommendation to maintain therapy indefinitely.
  • Third-generation TKIs and T3151-specific inhibitors. Scientists are studying several drugs to follow in the successful footsteps of, and improve upon, first-generation tyrosine kinase inhibitor (TKI) Gleevec and second-generation TKIs Sprycel and Tasigna. Doctors hope that third-generation drugs may succeed in overcoming the resistance to the earlier drugs that some patients experience. They're also looking for a treatment for a gene mutation called T3151, which doesn't respond well to the earlier drugs.
  • Vaccine therapy. Scientists are studying new methods to encourage a patient's own immune system cells to attack CML cells.
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last updated on Friday, December 12, 2014

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