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Related Concept Videos

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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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Enzyme-linked receptors are proteins that act as both receptor and enzyme, activating multiple intracellular signals. This is a large group of receptors that include the receptor tyrosine kinase (RTK) family. Many growth factors and hormones bind to and activate the RTKs.
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Related Experiment Video

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Author Spotlight: Finding New Therapeutic Targets for Malignant Peripheral Nerve Sheath Tumor Through Genome-Scale shRNA Screens
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Therapeutic targets for neuroblastomas.

Garrett M Brodeur1, Radhika Iyer, Jamie L Croucher

  • 1Children's Hospital of Philadelphia, Division of Oncology , CTRB Rm. 3018, 3501 Civic Center Blvd., Philadelphia, PA 19104-4302 , USA +1 215 590 2817 ; +1 215 590 3770 ; Brodeur@email.chop.edu.

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Neuroblastoma (NB) is a deadly childhood cancer with poor outcomes for high-risk cases. Targeted therapies offer promise but require integration with existing treatments to improve efficacy and reduce toxicity.

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Area of Science:

  • Pediatric Oncology
  • Cancer Therapeutics
  • Molecular Oncology

Background:

  • Neuroblastoma (NB) is the most common and lethal pediatric solid tumor.
  • High-risk NB has a dismal long-term survival rate below 50% despite treatment advances.
  • Current therapies for NB are associated with significant short- and long-term toxicities.

Purpose of the Study:

  • To review current and future therapeutic targets in neuroblastoma.
  • To classify potential and actual NB therapeutic targets.
  • To inform the development of more effective and less toxic neuroblastoma treatments.

Main Methods:

  • Literature review of relevant scientific publications.
  • Identification of therapeutic targets critical to NB malignant transformation and progression.
  • Classification of targets into four categories based on genetic alterations and expression patterns.

Main Results:

  • Identified four categories of NB therapeutic targets: amplified/mutated/overexpressed genes, inactivated genes, selectively expressed membrane genes, and common oncogenes.
  • Some targets have associated therapeutic approaches, but many remain untargeted.
  • Single targeted agents are unlikely to achieve long-term cure for high-risk NB.

Conclusions:

  • Further research is needed to develop targeted therapies for neuroblastoma.
  • Integrating targeted agents with each other and conventional therapies is crucial.
  • The goal is to enhance efficacy while minimizing systemic toxicity in neuroblastoma treatment.