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Chronic Myelogenous Leukemia Research Progress

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All forms of leukemia have similar symptoms, but the treatments differ. Chronic myelogenous leukemia (CML) is caused by a defect in one specific gene on chromosome 22. When this chromosome appears shorter than normal, it is called the Philadelphia chromosome. Chronic leukemia develops and progresses slowly at first, so that 20 to 40 percent of patients have no symptoms when they are first diagnosed based on a routine white blood cell count. However, the untreated disease typically progresses to an accelerated phase.

Dr. Jerry Avorn, a Harvard Medical School professor, describes research on treating CML as a milestone in pharmaceutical research. Most chemotherapy agents attack rapidly dividing cells in general, so that they kill a large number of healthy cells along with the malignant cells. Hair loss, nausea, and vulnerability to infection are well-known side effects of this class of drugs. “The holy grail of oncology research,” according to Avorn, is “blocking cancer cells preferentially without destroying normal ones, yielding a treatment that would cause many fewer side effects than conventional chemotherapy.”

Basic science researchers work to understand what specifically goes wrong with cancer cells to cause the aggressive and uncontrolled cell division. The Philadelphia chromosome was identified and linked with CML at the University of Pennsylvania. Further work at MIT, the University of California at San Francisco and at San Diego, the University of Chicago, and the Salk Institute produced important advances in understanding how the signaling protein BCR-ABL is involved in cancer. The Howard Hughes Medical Institute funded additional research in linking this protein to CML.

The result of these research efforts is a class of drugs called abl kinase inhibitors. The first-line treatment is Gleevec (imatinib), and has been successful in many patients. For patients who do not improve with this drug, alternatives include dasatinib (Sprycel) and nilotinib (Tasigna).

According to Reference 1, the only cure for CML is a transplant of stem cells, usually from bone marrow. Unfortunately, transplantation has its own risks and is considered a “fall back option”. With drug treatment, the 6 year-survival rate is reported as 88 percent. Reference 2 reports 5 year survival rates of 80 to 95 percent.

Research continues into further treatment options.


1. von Bubnoff N et al, “Chronic Myelogenous Leukemia”, Dtsch Arztebl Int 2010; 107(7): 114-21.

2. Vardiman JW, “Chronic Myelogenous Leukemia, BCR-ABL1+”, Am J Clin Pathol 2009 Aug; 132(2): 250-60.

3. Jerry Avorn, M.D., “Powerful Medicines”, Vintage Books, 2004.

4. Gleevec web site:

5. Other drug options:


Linda Fugate is a scientist and writer in Austin, Texas. She has a Ph.D. in Physics and an M.S. in Macromolecular Science and Engineering. Her background includes academic and industrial research in materials science. She currently writes song lyrics and health articles.

Add a Comment3 Comments

Expert HERWriter Guide Blogger

Indeed, Linda! It would make a real difference. There are additional drugs in trials for CML but the real goal is to find a cure. Far too many people continue to die although the improvements made possible by the TKIs are very, very impressive and encouraging. Thanks for covering this topic, and please continue to do so! Pat

September 16, 2010 - 7:57pm

Thanks, Pat! I wish we had a "magic bullet" drug effective enough to work for 100% of patients.

September 16, 2010 - 7:11pm
Expert HERWriter Guide Blogger

Dasatinib (Sprycel) and nilotinib (Tasigna) are now considered first line treatments for CML.Imatinib (Gleevec) remains a first line treatment. These options can be very confusing for patients. EmpowHER member and partner Andrew Schorr, of PatientPower.info, conducted an in-depth interview about this topic with Dr. Jorge Cortes, noted CML expert from M. D. Anderson Cancer Center.
You can find the interview here:

September 16, 2010 - 6:49pm
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We value and respect our HERWriters' experiences, but everyone is different. Many of our writers are speaking from personal experience, and what's worked for them may not work for you. Their articles are not a substitute for medical advice, although we hope you can gain knowledge from their insight.

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