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Future Treatments for CML

December, 2006

CR&T has emphasized the treatment of chronic myeloid leukemia (CML) and research activities on the JAK2 gene which has caused an investigative explosion in the myeloproliferative diseases. These activities have been well directed, for the 2006 Annual Meeting of the American Society of Hematology (ASH) was once again filled with research reports and updates pertaining to these two areas of investigation.

The treatment of CML with the drug Imatinib (Gleevec) has pushed the success of targeted treatments to the forefront of new research into cancer therapies. Clinical-scientists associated with the Cancer Research and Treatment Fund including Dr. Richard Silver and Dr. Eric Feldman played active and leading roles in the early clinical trials for this drug at Weill Cornell Medical College.

At the December meeting of the ASH in Orlando, it was reported that in a six-year follow-up study, 93% of patients who received Imatinib and achieved a major cytogenetic response by 12 months have remained in remission. However, as is usually the case with disease treatments, some new drug resistant mutations to the BCR-ABL “gatekeeper” gene have been noted. New trials are getting underway to overcome this resistance. One such drug is MK-0457, a small molecular inhibitor. Early tests have shown that this is the first compound to show positive activity against a very specific but somewhat common Gleevec resistant mutation.

This resistance to Gleevec occurs in only 2% of patients in first treatments and 8-13% of patients in end stage disease. As the number of patients who develop resistance grows, new targeted drugs such as MK-0457 will be an important addition to the available treatments.

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