Acute myeloid leukemia (AML) is the second most common type of leukemia. In 2016 in the US alone 19,950 people of all ages were diagnosed with AML. Despite chemotherapy, only 27% of patients survive 5 years after diagnosis. One-third of patients with AML carry the mutation in the FLT3 gene. In patients who received the FLT3 targeting therapy midostaurin the median survival improved to 75 month among midosaurin in combination with chemotherapy treated patients in comparison to only 26 months among patients administered placebo with chemotherapy.
This is just one example how a new targeted therapy can benefit cancer patients significantly.
Targeted cancer therapies are therapeutics that block the growth and spread of cancer by interfering with specific molecules in cancer cells that are involved in the growth, progression, and spread of the cancer.
The cornerstone of this new approach is the identification of clinically relevant targets. These are targets that play a key role in cancer cell growth and survival. In the first step we select proteins that are present in cancer cells but not normal cells or that are more abundant in cancer cells. Then we have to demonstrate that altering these proteins kills cancer cells.
One of the major downsides of gene-targeted cancer drugs has been that their effects don’t last and the cancer reappears after some time.