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

ABSTRACT

Introduction

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.

Areas covered

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.

Expert opinion

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.

KEYWORDS:

• Myelodysplastic syndrome
• acute myeloid leukemia
• immunotherapy
• bispecific antibody
• immune checkpoint inhibitor
• chimeric antigen receptor
• natural killer cell
• adoptive cell therapy

Article highlights

• Immune checkpoint inhibitors for MDS and AML

• Bispecific antibodies for MDS and AML

• CAR T cells for MDS and AML

• Predictive markers of response to immunotherapy in MDS and AML

Declaration of Interest

S Assouline is a consultant for AstraZeneca, Beigene, Genentech Roche, Janssen, Amgen, and Abbvie. She receives research funding from Novartis.

The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial relationships or otherwise to disclose.

Additional information

Funding

This paper was not funded.