AML is a fast-growing cancer of myeloid progenitor cells. As a results, there is a buildup of blasts in the marrow and blood. In turn, there are not enough red blood cells, platelets, and mature granulocytes. This cause serious health problems. If left untreated, AML is fatal.
Figure 1. The process of hematopoietic stem cells differentiate into mature cells. In AML, the process of differentiation and proliferation of myeloblasts into leukocytes is disrupted. (Source: Terese Winslow U.S. Govt).
Chemotherapy
Chemotherapy kills fast-growing cells throughout the body, including cancer cells and normal cells. Chemotherapy drugs used for the treatment of AML affect the instructions (genes) that tell cancer cells how and when to grow and divide. This disrupts the life cycle of cancer cells.
There are some types of chemotherapy used to treat AML:
- Anthracyclines: damage and disrupt the making of DNA causing cell death of both cancercous and non-cancerous cells. Some of the anthracyclines used to treat AML include daunorubicin, idarubicin, and mitoxantrone. These drugs can cause heart problems.
- Anti-metabolites: prevent the “building blocks” of DNA from being used. Some of the anti-metabolites used to treat AML include cladribine, clofarabine, cyratabine, fludarabine, methotrexate.
- Hypomethylating agents (HMAs): block methyl groups from binding to DNA. Some of the HMAs used to treat AML include decitabine, azacitidine. HMAs may be a good option for older patients or patients having serious health issues.
There are dosage forms that combine drugs belonging to the above drug groups. For instant, dual-drug liposome of cytarabine and daunorubicin (VyxeosTM, CPX-351) includes an anti-metabolite and an anthracycline.
Targeted therapy
Targeted therapy works throughout the body. It is drug therapy that focuses on specific or unique features of cancer cells. Targeted therapy seeks out how cancer cells grow, divide, and move in the body. These drugs stop the action of molecules that help cancer cells grow and/or survive. Targeted therapy might be used alone or in combination with chemotherapy.
Here is a list of some targeted therapies used in treating AML, classified by their targets:
- CD33 surface protein: Gemtuzumab ozogamicin (GO) is a type of targeted therapy that is linked to a chemotherapy drug. Gemtuzumab attaches to CD33 in, then GO enters the cells. Once inside, ozogamicin is released and kills cancer cells. Many leukemic blasts have CD33 proteins. Mature blood cells do not have CD33 and are not affected. GO may cause liver issues.
- FLT3: Gilteritinib or midostaurin is used to treat AML with FLT3-ITD and FLT3-TKD gene mutations. Sorafenid with azacitidine or decitabine may be used to treat AML with FLT3-ITD mutation.
- IDH1 and IDH2: Ivosidenib is used to treat AML with IDH1 mutation. Enasidenib is used to treat AML with IDH2 mutation.
Source: National Comprehensive Cancer Network (NCCN) guideline for patient, AML, 2022.
