Immunotherapy for myelodysplastic syndrome and acute myeloid leukemia: where do we stand?

Myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) are generally characterized by a poor prognosis with currently available therapies. Immunotherapies have already seen success in treating a variety of malignant disorders, and their role in managing myeloid cancers is evolving rapidly.

This is a review of the immunotherapies tested in MDS and AML, including immune checkpoint inhibitors, bispecific antibodies, and cell therapies such as chimeric antigen receptor (CAR) T cell therapy, T cell receptor (TCR) engineered T cells, and natural killer (NK) cells, with a focus on clinical trials conducted to date and future directions.

Initial clinical trials exploring checkpoint inhibitors in MDS and AML have demonstrated high toxicity and disappointing efficacy. However, ongoing trials adding novel checkpoint inhibitors to standard therapy are more promising. Technological advances are improving the outlook for bispecific antibodies, and cellular therapies like adoptive NK cell infusion have favorable efficacy and tolerability in early trials. As our understanding of the immune microenvironment in MDS and AML improves, the role for immunotherapy in the treatment of these diseases will become clearer.