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Peer-reviewedCC BY 4.0Cancers

Interferon Therapy in Acute Myeloid Leukemia: Biological Rationale, Clinical Evidence, and Future Directions

  • Jan Philipp Bewersdorf1
  • Hsin-An Hou2
  • Evelyn Yingtzu Yen3
  • Shinya Imada4
  • Shaoyu Chang4
  • Xavier Cheng-Hong Tsai2
  • Albert Qin3
4 affiliations
  1. 1Section of Medical Oncology and Hematology, Department of Internal Medicine, Yale School of Medicine and Yale Comprehensive Cancer Center, New Haven, CT 06510, USA
  2. 2Division of Hematology, Department of Internal Medicine, National Taiwan University Hospital, Taipei 100215, Taiwan
  3. 3PharmaEssentia Corporation, Taipei 115603, Taiwan
  4. 4PharmaEssentia USA, Burlington, MA 01803, USA
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Abstract

Acute myeloid leukemia (AML) is a heterogeneous hematologic malignancy characterized by uncontrolled clonal proliferation of immature myeloid blasts in bone marrow and peripheral blood. The contemporary therapeutic landscape for AML spans a broad spectrum of interventions, relying on cytotoxic chemotherapy, hypomethylating agents, and an expanding array of targeted therapies to accommodate both patients eligible for intensive induction and those requiring lower intensity regimens. Allogeneic hematopoietic stem cell transplantation is performed with curative intent in eligible high-risk patients, and donor lymphocyte infusion is utilized to prevent or treat post-transplant early relapse. Despite ongoing therapeutic advances, the overall prognosis for AML remains unsatisfactory with a five-year relative survival rate of approximately 31.9%. Driven by an evolving understanding of AML biology, particularly the roles of leukemic stem cells and measurable residual disease, interferon (IFN) therapy, specifically type I and type II IFNs, has emerged as a compelling therapeutic candidate, distinguished by its direct anti-leukemic effect and indirect anticancer activities, notably via immunomodulatory effects. This review outlines the biological rationale and clinical evidence for current IFN therapy in AML, aiming to inform future preclinical and clinical investigations.

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Cite

BibTeXRIS
BibTeX
@article{bewersdorf2026interferon,
  title = {{Interferon Therapy in Acute Myeloid Leukemia: Biological Rationale, Clinical Evidence, and Future Directions}},
  author = {Jan Philipp Bewersdorf and Hsin-An Hou and Evelyn Yingtzu Yen and Shinya Imada and Shaoyu Chang and Xavier Cheng-Hong Tsai and Albert Qin},
  journal = {Cancers},
  year = {2026},
  month = oct,
  doi = {10.3390/cancers18203266},
  url = {https://www.mdpi.com/2072-6694/18/20/3266},
  note = {CC BY 4.0},
}
RIS
TY  - JOUR
TI  - Interferon Therapy in Acute Myeloid Leukemia: Biological Rationale, Clinical Evidence, and Future Directions
AU  - Jan Philipp Bewersdorf
AU  - Hsin-An Hou
AU  - Evelyn Yingtzu Yen
AU  - Shinya Imada
AU  - Shaoyu Chang
AU  - Xavier Cheng-Hong Tsai
AU  - Albert Qin
PY  - 2026
DA  - 2026/10/01
JO  - Cancers
DO  - 10.3390/cancers18203266
UR  - https://www.mdpi.com/2072-6694/18/20/3266
AB  - Acute myeloid leukemia (AML) is a heterogeneous hematologic malignancy characterized by uncontrolled clonal proliferation of immature myeloid blasts in bone marrow and peripheral blood. The contemporary therapeutic landscape for AML spans a broad spectrum of interventions, relying on cytotoxic chemotherapy, hypomethylating agents, and an expanding array of targeted therapies to accommodate both patients eligible for intensive induction and those requiring lower intensity regimens. Allogeneic hematopoietic stem cell transplantation is performed with curative intent in eligible high-risk patients, and donor lymphocyte infusion is utilized to prevent or treat post-transplant early relapse. Despite ongoing therapeutic advances, the overall prognosis for AML remains unsatisfactory with a five-year relative survival rate of approximately 31.9%. Driven by an evolving understanding of AML biology, particularly the roles of leukemic stem cells and measurable residual disease, interferon (IFN) therapy, specifically type I and type II IFNs, has emerged as a compelling therapeutic candidate, distinguished by its direct anti-leukemic effect and indirect anticancer activities, notably via immunomodulatory effects. This review outlines the biological rationale and clinical evidence for current IFN therapy in AML, aiming to inform future preclinical and clinical investigations.
SN  - 2072-6694
ER  -