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PI3King the lock: targeting the PI3K/Akt/mTOR pathway as a novel therapeutic strategy in neuroblastoma

David King1, Daniel Yeomanson, Helen E Bryant

  • 1*Academic Unit of Child Health †Department of Pediatric Oncology, Sheffield Children's Hospital ‡Academic Unit of Molecular Oncology, Department of Oncology, The Medical School, Sheffield, UK.

Journal of Pediatric Hematology/Oncology
|March 27, 2015
PubMed

Insights

High-risk neuroblastoma, a common childhood cancer, has a poor prognosis. Targeting the PI3K/Akt/mTOR pathway offers a promising therapeutic strategy for this aggressive disease.

Area of Science:

  • Oncology
  • Molecular Biology
  • Pediatric Cancer Research

Background:

  • Neuroblastoma is the most frequent extracranial childhood cancer.
  • High-risk neuroblastoma presents a significant therapeutic challenge with poor patient outcomes.
  • Targeting key molecular pathways is crucial for developing effective, biologically based treatments.

Purpose of the Study:

  • To review the evidence for aberrant PI3K/Akt/mTOR pathway activation in neuroblastoma.
  • To explore the mechanisms driving this pathway's dysregulation.
  • To discuss the therapeutic potential and clinical challenges of targeting this pathway in neuroblastoma.

Main Methods:

  • Literature review of preclinical and clinical studies.
  • Analysis of molecular mechanisms underlying PI3K/Akt/mTOR pathway activation.
  • Evaluation of targeted therapies and their associated challenges.

Main Results:

  • The PI3K/Akt/mTOR pathway is frequently activated in neuroblastoma.
  • This aberrant activation contributes to malignant transformation and progression.
  • Several therapeutic agents targeting this pathway are under investigation.

Conclusions:

  • The PI3K/Akt/mTOR pathway is a critical target for neuroblastoma therapy.
  • Overcoming challenges in drug development and clinical implementation is essential.
  • Targeted therapies hold promise for improving outcomes in high-risk neuroblastoma.

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