Chronic Phase of CML
Our Transplant Program Information about our transplant program 
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Imatinib controls the disease in most patients, but because the drug was licensed by the FDA somewhat recently, in 2001, doctors do not yet know how long patients will respond to the drug. As noted above, there are alternative drugs to imatinib that have also been shown to be highly effective. For those patients whose disease "breaks through" these drugs, another option called stem cell transplantation may be available. In this procedure, stem cells -- blood-forming cells -- are removed from an appropriate donor. The patient then receives a high dose of chemotherapy, sometimes in conjunction with radiation therapy, which destroys tumor cells but also damages the stem cells in the patient's bone marrow. The harvested stem cells are then administered (transplanted) to help rebuild the patient's immune system.
Accelerated Phase
For patients in the accelerated phase, treatment may include the options outlined above - imatinib, dasatinib, nilotinib, bone marrow transplantation, immunotherapy, or chemotherapy. Patients may also receive transfusions of blood or blood products to relieve symptoms.
Blast Phase
For patients in the blast phase, treatment generally includes chemotherapy in addition to imatinib (or dasatinib or nilotinib). This is a difficult phase of the disease to treat. Often, stem cell transplantation is recommended if an appropriate donor is identified and if the patient is healthy enough to undergo the procedure. Because the best results with stem cell transplantation are when the disease is in chronic or early accelerated phase disease, the aim of treatment in this phase is to get the patient back to the earlier phase of the disease.
Investigational Treatments
Find a Clinical Trial Find out about new clinical trials for leukemia
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Researchers are now developing new agents for cancer treatment at a faster rate than at any time since chemotherapeutic drugs were introduced in the late 1940s. Relying, in part, on the information that is emerging about the genetic basis of leukemia, investigators are pursuing a variety of strategies to control the disease -- approaches that can kill tumor cells directly, inhibit the body's production of substances that promote their growth, or enhance the immune response against leukemic cells.
Below are some of the therapies now being tested for CML. Studies of these and other promising new treatments are available to patients through clinical trials. For patients whose disease has recurred or is refractory, or who are not candidates for stem cell transplantation, clinical trials provide additional treatment options.
Targeted Therapies
Researchers are studying the most effective ways, including timing and dose, to use the targeted therapies now available (imatinib, dasatinib, nilotinib) and are working to create and test additional therapies that will block the aberrant gene and its variants.
Vaccines
Many leukemias, including CML and some solid tumors, are associated with chromosome translocations that create fusion genes -- new genes that include pieces of both the original chromosomes. These new genes contain instructions for new proteins, called fusion gene products. Researchers are using the amino acid sequences of these fusion proteins or mutated sequences to develop tumor-specific vaccines (called oncogene fusion point vaccines) for treating patients with cancer.
The first such vaccine for treating CML was developed by investigators at Memorial Sloan-Kettering and has been used in clinical trials to treat patients. The vaccine consists of a piece of the protein made by the Philadelphia chromosome gene and uses a portion of the fusion gene, called the bcr/abl fusion point, as its target. The goal of this therapeutic vaccine is to make the patients' immune system recognize and destroy the leukemia cells.
Cellular Therapy After Stem Cell Transplantation
In some patients who undergo stem cell transplantation, the disease recurs following the procedure. A strategy developed here at Memorial Sloan-Kettering and at other institutions -- a reinfusion of a particular type of white blood cell called lymphocytes from the original donor -- may boost these patients' ability to fight the disease. The effectiveness of this approach is now under study.
