The Food and Drug Administration announced that it has sanctioned the combination of Daurismo (glasdegib) tablets and low-dose cytarabine (LDAC), a type of chemotherapy, for the treatment of newly-diagnosed acute myeloid leukemia (AML). The therapy is meant for senior patients, 75 or older, and those who may preclude the use of intensive chemotherapy.
“Intensive chemotherapy is usually used to control AML, but many adults with AML are unable to have intensive chemotherapy because of its toxicities. Today’s approval gives health care providers another tool to use in the treatment of AML patients with various, unique needs”, said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence.
At the moment, Daurismo is the first and only Hedgehog pathway inhibitor for AML to be endorsed by the agency. The Hedgehog signaling pathway plays an essential role in embryogenesis, the process by which human embryos are developed. Later in life, however, abnormal activation of the pathway can lead to the development and persistence of cancer stem cells.
In the press release issued by Pfizer, Jorge Cortes, M.D., deputy chair and professor of medicine in the Department of Leukemia at MD Anderson Cancer Center noted that: “In the trial, Daurismo plus low-dose chemotherapy reduced the risk of death during the study period by 54 percent compared to chemotherapy alone. This provides a much-needed treatment for those patients for whom intensive chemotherapy is not an option”.
Even though it received a green light, the treatment does come with warnings for toxicity. It also lists a number of possible side effects, including anemia, fatigue, hemorrhage, febrile neutropenia, musculoskeletal pain, and others.
The American Cancer Society estimates about 19.500 new cases of acute myeloid leukemia for 2018 in the United States. The disease has a rapid progression and a particularly low survival rate. This new treatment could save countless lives.