Update on Immunotherapy in AML and MDS: Monoclonal Antibodies and Checkpoint Inhibitors Paving the Road for Clinical Practice

  • Lucia Masarova
  • Hagop Kantarjian
  • Farhad Ravandi
  • Padmanee Sharma
  • Guillermo Garcia-Manero
  • Naval DaverEmail author
Part of the Advances in Experimental Medicine and Biology book series (AEMB, volume 995)


In the past few years, our improved understanding of the pathogenesis of acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) has led to remarkable advances in the development of novel therapeutic approaches for these diseases. This chapter summarizes the available clinical data with immune-based therapeutic modalities in AML and MDS, focusing on monoclonal antibodies, T cell engager antibodies, chimeric antigen receptor (CAR)-T cells, and checkpoint blockade via blockade of PD-1/PD-L1 or CTLA4. Numerous clinical trials are currently ongoing in patients with AML and MDS, both in the frontline and relapsed refractory setting. Given the natural diversity of AML blasts, it became apparent that the best responses would be achieved with rationally designed combination strategies of immune therapy, molecular therapy, and chemotherapy. A number of such combinations are enrolling patients with AML in various clinical settings. Biomarkers to select the optimal combination regimen for individual patients are critical.


Acute myeloid leukemia Immunotherapy Monoclonal antibody Immune checkpoint blockade 


Funding Source

This review was supported in part by the MD Anderson Cancer Center Support Grant (CCSG) CA016672.

Conflict of Interest L.M. and G.G.M.—none. N.D.—research funding from BMS, Pfizer, Immunogen, AbbVie, Astellas, Servier, Daiichi-Sankyo, Nohla, Genentech; advisor/consultant to Novartis, BMS, Daiichi-Sankyo, Astellas, AbbVie, Pfizer, Jazz, Agios, and Celgene. H.K.—research grants from AbbVie, Agios, Amgen, Ariad, Astex, BMS, Cyclacel, Daiichi-Sankyo, Immunogen, Jazz Pharma, Novartis, Pfizer; honoraria: AbbVie, Actinium (advisory board), Agios, Amgen, Immunogen, Orsinex, Pfizer, Takeda. F.R.—research support and honoraria from BMS. P.S.—stock/ownership for Jounce, Neon, Constellation, Oncolytics, BioAtla, Forty-Seven, Apricity, Polaris, Marker Therapeutics, Codiak; advisory board on Constellation, Jounce, Kite Pharma, Neon, BioAtla, Pieris Pharmaceuticals, Oncolytics Biotech, Merck, BioMx, Forty-Seven, Polaris, Apricity, Marker Therapeutics, Codiak.


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Copyright information

© Springer Nature Switzerland AG 2018

Authors and Affiliations

  • Lucia Masarova
    • 1
  • Hagop Kantarjian
    • 1
  • Farhad Ravandi
    • 1
  • Padmanee Sharma
    • 2
  • Guillermo Garcia-Manero
    • 1
  • Naval Daver
    • 1
    Email author
  1. 1.Department of LeukemiaMD Anderson Cancer CenterHoustonUSA
  2. 2.Department of Immunotherapy PlatformMD Anderson Cancer CenterHoustonUSA

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