Diagnosing and treating early cancers is a priority for both physicians and researchers because treating late stage cancers is often difficult or impossible. Cancers with a genetic link can allow for the identification of high-risk individuals and about 5-10% of cancers are inherited. If a person is diagnosed with a cancer with a potential genetic link, family members can be tested to see if they also have a mutated gene and are at increased risk to develop cancer. 

Now, more genetic mutations with a potential link to cancer are under investigation, including genetic mutations leading to acute myeloid leukemia (AML). Acute myeloid leukemia is a type of blood cancer found in both children and adults. According to new research on AML by Dana-Farber Cancer Institute, blood cells need more than one genetic mutation or “hit” to turn cancerous. That window between the first genetic “hit” and the second mutation is a potential treatment window to prevent leukemia from developing in the first place.

In one animal study conducted at Dana-Farber, mice who experienced the first genetic mutation, called clonal hematopoiesis of indeterminate potential (CHIP), and then a second mutation ultimately developed leukemia. However, when treated after the first mutation with a compound called VTP-50469, the problematic mutated blood cells stopped reproducing and leukemia never developed. If doctors can treat the mutations that cause AML rather than treating the resulting leukemia with traditional cancer therapies, this could potentially improve patient outcomes. Scientists still need to prove that the compound has a high level of safety and efficacy in patients, but this research could lead to the early treatment of patients with AML.