New research confirmed that a revolutionary drug has transformed a previously fatal leukemia into a manageable chronic disease for many patients. The drug, imatinib (brand name: Gleevec), was approved by the United States Food and Drug Administration in 2001 for patients with chronic myelogenous leukemia (CML), a cancer of the white blood cells.

Researchers found patients taking imatinib for CML, who have been in cytogenic remission after two years of treatment, have a mortality rate similar to that of the general population. For patients this provides the first clear evidence that the drug is able to control their cancer in a way that gives them a normal life expectancy. The Long Term Imatinib Effects (ILTE) study was published online March 22, 2011 in the Journal of the National Cancer Institute. Data came from patients from 27 centers in Europe, North and South America, Africa, the Middle East and Asia.

According to the National Cancer Institute, “Imatinib represents a new class of cancer drugs and a new way of thinking about cancer. These molecularly targeted drugs are different because they target abnormal proteins that are fundamental to the cancer itself.”

Multiple studies of imatinib have taken place during industry-sponsored clinical trials at medical centers in the United States and other countries. This research was set up to study patients taking the drug as part of their daily lives instead of during clinical trials. Led by Carlo Gambacorti-Passerini, M.D. of the University of Milano Bicocca/San Gerardo Hospital in Monza, Italy, the study followed some 800 patients who were in complete cytogenic remission after two years of taking the drug. The mortality rate for those studied was 4.8 percent, which is similar to what would normally be expected in the general population.

The authors concluded that patients on imatinib “frequently suffer from side effects that are non-serious but can nonetheless reduce their quality of life.” They noted that the findings highlight the “importance of a good relationship between health care providers and patients, where side effects are easily communicated and addressed to reduce/avoid non-compliance.”

In an editorial comment about the study, a leukemia expert from Johns Hopkins University in Baltimore said the use of imatinib and similar agents "create a platform from which we can entertain the possibility of curing" the disease without a stem cell transplant. B. Douglas Smith, MD, of the Sidney Kimmel Comprehensive Cancer Center, called for research on ways to completely eliminate the small amount of residual disease after treatment with imatinib. “It is now time for clinical and laboratory investigators to build on this platform and work to turn good and great responses into cures," he added.

Resources:

Gambacorti-Passerini C, Multicenter independent assessment of outcomes in chronic myeloid leukemia patients treated with imatinib. Journal of the National Cancer Institute, 2011;103(7):553-561. http://jnci.oxfordjournals.org/content/103/7/553.short?rss=1

Smith, B. Douglas, MD, Division of Hematologic Malignancies, Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Correspondence, Journal of the National Cancer Institute, 201,103 (7): NP. doi: 10.1093/jnci/djr127 http://jnci.oxfordjournals.org/content/103/7/527.full

National Cancer Institute: Gleevec Questions and Answers: http://www.cancer.gov/newscenter/qa/2001/gleevecqa

National CML Society: CML Therapies: http://www.nationalcmlsociety.org/living-cml/therapies