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The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
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Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
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Related Experiment Video

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Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
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Targeted Therapy Development in Acute Myeloid Leukemia.

Tulasigeri M Totiger1, Anirban Ghoshal2, Jenna Zabroski1

  • 1Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami, FL 33136, USA.

Biomedicines
|February 25, 2023
PubMed
Summary

Novel targeted therapies are crucial for curing acute myeloid leukemia (AML), addressing unmet needs beyond current treatments like venetoclax and azacitidine. This review explores innovative approaches for long-term AML remission.

Keywords:
acute myeloid leukemiadrug developmentmyeloid neoplasmnon-selective drugsprecision medicineselective drugssmall molecule inhibitortargeted therapy

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Area of Science:

  • Hematology
  • Oncology
  • Molecular Biology

Background:

  • Acute myeloid leukemia (AML) therapy has seen advancements with targeted drugs, yet a cure remains elusive due to disease heterogeneity and drug resistance.
  • Existing treatments, including venetoclax combined with azacitidine, improve outcomes but are limited by cytotoxicity and lack curative potential.
  • There is a significant need for therapies targeting specific AML abnormalities while sparing healthy cells and eradicating leukemia-initiating cells.

Purpose of the Study:

  • To comprehensively review the development of novel targeted therapies for acute myeloid leukemia (AML).
  • To discuss the translational perspective of these innovative therapies.
  • To cover both selective and non-selective drug development strategies for AML.

Main Methods:

  • Literature review of therapeutic developments in acute myeloid leukemia.
  • Analysis of small molecule drug efficacy and resistance mechanisms.
  • Examination of novel targeted therapies addressing leukemogenesis.

Main Results:

  • Multiple targeted therapies have been approved, but long-term remission and cure for heterogeneous AML remain challenging.
  • Venetoclax in combination with azacitidine shows improved response rates and survival but is not curative and has cytotoxicity.
  • Drug resistance limits the long-term efficacy of monotherapies, highlighting the need for innovative approaches.

Conclusions:

  • Despite progress, a curative treatment for AML is still needed, necessitating innovative therapies targeting specific molecular abnormalities.
  • The complexity of AML heterogeneity drives the development of novel agents targeting diverse leukemogenesis mechanisms.
  • This review provides a comprehensive overview of novel targeted therapies and their translational potential for AML treatment.