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Acute Myeloid Leukemia Mutations and Future Mechanistic Target to Overcome Resistance

Rehan Uddin1, Noureldian H E Darwish1,2, Shaker A Mousa3

  • 1The Pharmaceutical Research Institute, Albany College of Pharmacy and Health Sciences, 1 Discovery Drive, Rensselaer, NY, 12144, USA.

Current Treatment Options in Oncology
|July 2, 2021
PubMed
Abstract

Insights

Targeting residual leukemic stem cells (LSCs) in acute myeloid leukemia (AML) is crucial for improving patient survival. Future therapies may involve immunotherapy and nanotechnology to overcome treatment resistance and achieve complete remission.

Area of Science:

  • Hematology
  • Oncology
  • Biomedical Engineering

Background:

  • Cytogenetics and mutation identification have enabled targeted therapies for acute myeloid leukemia (AML).
  • Despite recent FDA approvals like gilteritinib and azacitidine, overall survival for AML remains stagnant at 25%.
  • Residual leukemic stem cells (LSCs) are a key factor limiting complete remission and driving treatment relapse/resistance.

Purpose of the Study:

  • To explore the relationship between the bone marrow niche and LSCs in AML.
  • To identify novel therapeutic strategies for overcoming treatment resistance and improving AML patient outcomes.
  • To investigate the potential of immunotherapy and nanotechnology in achieving complete remission for AML patients.

Main Methods:

  • Review of current literature on AML pathogenesis, treatment resistance, and emerging therapeutic modalities.
  • Analysis of the role of the bone marrow niche in supporting LSCs.
  • Exploration of nanotechnology and immunotherapy approaches, including nano-encapsulation and chimeric antigen receptor T-cell therapy.
  • Investigation of novel molecular targets such as integrin αvβ3 and acid ceramidase.

Main Results:

  • Residual LSCs in the bone marrow niche are implicated in treatment failure and relapse in AML.
  • Nanotechnology offers potential for improved drug bioavailability, resistance evasion, and targeted combination therapy.
  • Immunotherapy, specifically targeting surface antigens like CLL1 and CD123 with CAR T-cells, shows promise for improving survival.
  • Inhibition of integrin αvβ3 and acid ceramidase are identified as potentially efficacious therapeutic strategies.

Conclusions:

  • Understanding the bone marrow niche's interaction with LSCs is critical for developing effective AML therapies.
  • Advanced approaches like immunotherapy and nanotechnology, alongside novel molecular targets, are essential for improving complete remission rates and overall survival in AML.
  • Future research should focus on integrating these strategies to overcome AML treatment resistance and achieve long-term patient benefit.
Keywords:
Acid ceramidaseAcute myeloid leukemia resistanceBCL-2Bone marrow microenvironmentCAR TFLT3HedgehogImmunotherapyNanotechnology

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