Rationale of disease
AML is a rare type of cancer of the blood and bone marrow with excess immature white cells. These abnormal white blood cells leave less room for healthy cells (red blood cells, white blood cells, and platelets), resulting in easy bleeding, infections, and anemia. AML is a liquid cancer so it does not produce tumors. However, AML is aggressive and can spread quickly to the lymph nodes, spine, brain, liver, spleen, and other organs. If left untreated, AML can be life threatening. The 5-year survival rate of AML is 28.3%.
AML is most common among men and older adults, with the average age of diagnosis being 68. Genetic disorders, blood disorders, and exposure to hazardous chemicals through smoking or other means are common causes of AML. AML can be diagnosed through blood tests, a bone marrow aspiration or biopsy, a spinal tap, and genetic testing. The most common treatment for AML is chemotherapy drugs. Other treatment options include bone marrow transplants and clinical trials.
Although AML is a rare form of cancer, it is the second most common form of leukemia diagnosed in children and adults. Each year in the United States, over 20,000 people of all ages are diagnosed with AML. The proportion of leukemia cases that are AML has increased by over 5% from 1990 to 2017, increasing the need for new AML treatments.
While AML was uniformly fatal half a century ago, intensive chemotherapy now cures 40% of adults. This is a substantial improvement, but there remains a significant unmet clinical need for older and relapsed and refractory patients where fewer than 10% of patients are cured. AML represents a unique burden because it affects people of various ages, involves extensive time in the hospital, is associated with high rates of infection and complications, and requires the need for stem cell transplants (bone marrow).
Many AML patients are not eligible for intensive chemotherapy. There is an unmet need for more effective and less toxic treatments that improve quality of life from disease. AML patients also have a high rate of relapse so treatments that reduce relapse are in high demand.