Although chemotherapeutic drugs are often the mainstay of many forms of AML, newer targeted therapies are constantly being developed. Targeted therapy refers to a drug that targets or interacts specifically with a protein, enzyme, or receptor that is being expressed as a result of the mutation that is causing the AML.
An early targeted therapy that was developed was actually to CML in which the translocation known as the Philadelphia Chromosome caused the fusion of the bcr-abl genes which triggered the overexpression of an enzyme known as a tyrosine kinase. This enzyme is pivotal in the unchecked replication of the cells. By inhibiting this tyrosine kinase, the cells can be induced to stop replicating and to differentiate.
Newer research shows that some of the mutations in AML can also involve tyrosine kinases, and thus newer generations of these inhibitors may be useful in this disease as well.
