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Related Experiment Video

Updated: Jan 19, 2026

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Hematological malignancies and molecular targeting therapy.

Akira Shimada1

  • 1Department of Pediatric Hematology and Oncology, Okayama University Hospital, Okayama, 700-8558, Japan.

European Journal of Pharmacology
|September 8, 2019
PubMed
Summary

Next-generation sequencing advances understanding of blood cancers. Molecularly targeted drugs, like tyrosine kinase inhibitors, are improving outcomes for leukemia patients, offering new hope beyond traditional treatments.

Keywords:
Acute lymphoblastic leukemiaAcute myeloid leukemiaChronic myeloid leukemiaMolecular targeting therapyMyeloproliferative neoplasmTyrosine kinase inhibitor

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Area of Science:

  • Hematology
  • Oncology
  • Genetics

Background:

  • Genetic analysis, particularly next-generation sequencing (NGS), has significantly advanced the understanding of hematological malignancies.
  • Conventional chemotherapy and hematopoietic stem cell transplantation (HSCT) have reached a plateau in clinical outcomes for blood cancers.

Purpose of the Study:

  • To review the impact of molecularly targeted drugs in treating hematological malignancies.
  • To highlight advancements in therapies for chronic myeloid leukemia (CML), acute lymphoblastic leukemia (ALL), and acute myeloid leukemia (AML).

Main Methods:

  • Review of clinical applications of molecularly targeted drugs.
  • Analysis of treatment outcomes for various leukemia subtypes using tyrosine kinase inhibitors (TKIs) and other targeted agents.

Main Results:

  • Imatinib, a first-generation TKI, revolutionized CML treatment, reducing the need for HSCT.
  • Second-generation TKIs (dasatinib, nilotinib, ponatinib) are available for CML.
  • TKIs combined with chemotherapy and HSCT improved survival in Philadelphia chromosome-positive ALL.
  • Midostaurin (a TKI) improved survival in FLT3-ITD mutated AML.
  • Gemtuzumab ozogamicin is approved for AML treatment.

Conclusions:

  • Molecularly targeted agents are increasingly utilized in managing hematological malignancies.
  • Targeted therapies have significantly improved survival rates and clinical outcomes for various leukemia subtypes.
  • Emerging cell therapies, including CAR-T and bispecific T-cell engagers, show promise for difficult-to-treat leukemias.