For patients with acute myeloid leukemia — and for the physicians who treat them — 2017 has been a milestone year so far. And, as patients and advocates observe Leukemia and Lymphoma awareness month throughout September, there is plenty of reason for optimism and encouragement.
What is acute myeloid leukemia?
Acute myeloid leukemia (AML) is an aggressive, rapidly advancing blood cancer that forms in bone marrow and causes a sharp decline in the number of white blood cells in the bloodstream. The National Cancer Institute estimates that more than 21,000 people in the United States will be diagnosed with AML this year.
After decades without new treatment for AML, an aggressive blood cancer, has seen approval this year of three new therapies for the disease.
“There have been few significant drugs developed for acute myeloid leukemia in 30 years, so these treatments are very exciting advances,” says Rocky Mountain Cancer Centers Oncologist and Hematologist John M. Burke, MD.
- In April, the U.S. Food and Drug Administration approved the first targeted therapy for AML, a drug called Rydapt (its generic name is midostaurin) for adults with newly diagnosed disease who have a genetic mutation called FLT3. The drug is approved for use along with a diagnostic tool that is used to detect the FLT3 mutation, and in combination with chemotherapy. Rydapt is a kinase inhibitor that works by blocking enzymes that promote cell growth.
- In August, the U.S. Food and Drug Administration (FDA) approved a drug called Vyxeos for adults with two types of aggressive acute myeloid leukemia (AML). Vyxeos is a combination of two chemotherapy drugs, daunorubicin and cytarabine, encapsulated in a liposome — a tiny fatlike particle. Because cancer cells often absorb liposomes more efficiently than other cells do, a higher dose of the drugs is delivered to leukemia cells than to normal cells, which may mean the drug can be more effective without causing increased toxicity. Vyxeos is approved for use in patients who developed AML after treatment for another cancer and for patients who have AML with myelodysplasia-related changes (AML-MRC)
- Also in August, the U.S. Food and Drug Administration (FDA) approved a targeted therapy, Idhifa (its generic name is enasidenib) to treat adults who have a specific type of AML. The drug is for AML patients who have a mutation in a gene called IDH2, and the FDA approved a test to check for the mutation. Idhifa is intended to treat AML patients whose cancer has either not responded to or has come back after previous treatment. The drug is the first therapy approved for recurring AML with the IDH2 mutation. In clinical trials, nearly 20 percent of patients achieved complete remission with the drug.
- Finally, in September, the U.S. Food and Drug Administration (FDA) approved gemtuzumab ozogamicin (Mylotarg) for patients with both newly diagnosed and relapsed/refractory AML. Gemtuzumab ozogamicin had been approved years ago but later was withdrawn from the market because of concern about toxicities and uncertain benefit. However, recent studies have demonstrated that, when combined with standard chemotherapy drugs, gemtuzumab ozogamicin improves outcomes in patients with newly diagnosed AML. In addition, separate studies of single-agent gemtuzumab ozogamicin in patients with relapsed disease confirmed a benefit. The dose that is now recommended for gemtuzumab ozogamicin is lower than the dose that was approved years ago.
These advances demonstrate that, even in a disease that has been extremely difficult to find new and effective treatments for, modern scientific research is starting to make considerable progress in developing new therapies.
Learn more about how Rocky Mountain Cancer Centers is helping patients fight leukemia through advanced treatment, research, and clinical trials.