Example of Recent Success in Drug Treatment (1) (Preface )

Abdallah A. Abbadi

Abstract


Just 10 years ago, Chronic Myeloid Leukemia (CML) was a universal killer. All forms of treatments made little effect on the cause of disease. They only controlled the white cell count, platelet count and the size of the spleen, but had little effect on the natural evolution or the course of the disease. Most patients survived few years and almost none was alive at 9 years. The only known "curative" treatment at the time was allogeneic Bone Marrow Transplantation (BMT). Even in the best centers, the long term survival results were in the range of 50-60%. Given the limitation in the availability of donors and the age of patients, only 20% of patients could be offered this form of therapy.

When imatinib (glivec) was discovered to inhibit the tyrosine kinase domain of the BCR-ABL (the abnormal fusion gene which causes the disease), clinical trials followed (such as the IRIS trial), comparing the results of imatinib with those of the best treatment available at the time which was alfa interferon with cytosine arabinoside.

It was soon found that major improvements were documented in all aspects of the disease, especially the effect on the abnormal Philadelphia chromosome and the gene causing the disease. For the first time, a new treatment made major impact on the BCR-ABL gene and caused the disappearance of the fusion gene in the great majority of patients. As a result, patients with CML can now live normal life for many years without evidence of disease.

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