Although chemotherapeutic drugs are often the mainstay of many forms of AML, newer targeted therapies are constantly being developed. A targeted therapy is a drug that targets or interacts specifically with a protein, enzyme, or receptor being expressed due to the mutation causing the AML.
An early targeted therapy developed was for 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; thus, newer generations of these inhibitors may also be helpful in this disease.
