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Molecular targeted therapies for pediatric atypical teratoid/rhabdoid tumors

Chang Zhang1, Hao Li1

  • 1Department of Neurosurgery Children's Hospital of Fudan University Shanghai China.

Pediatric Investigation
|July 1, 2022
PubMed

Insights

Atypical teratoid/rhabdoid tumors (AT/RTs) are aggressive infant brain tumors. This review explores targeted molecular therapies for AT/RTs, focusing on genetic and epigenetic drivers like SMARCB1/SMARCA4 inactivation.

Area of Science:

  • Pediatric oncology
  • Neuro-oncology
  • Molecular biology

Background:

  • Atypical teratoid/rhabdoid tumors (AT/RTs) are highly lethal pediatric central nervous system cancers.
  • Current treatments (surgery, radiotherapy, chemotherapy) offer poor prognoses and severe side effects.
  • Tumorigenesis is driven by inactivation of the SMARCB1 or SMARCA4 genes.

Purpose of the Study:

  • To review the current literature on targeted molecular therapies for pediatric AT/RTs.
  • To highlight the role of genetic and epigenetic alterations in AT/RT pathogenesis.
  • To explore potential therapeutic strategies targeting these molecular derangements.

Main Methods:

  • Comprehensive literature search of studies on AT/RTs and targeted therapies.
  • Analysis of genetic (SMARCB1/SMARCA4 inactivation) and epigenetic alterations.
  • Review of preclinical and clinical data on molecularly targeted agents.

Main Results:

  • SMARCB1/SMARCA4 inactivation is a key event in AT/RT development.
  • Specific epigenetic dysregulations are associated with AT/RT subtypes.
  • Emerging targeted therapies show promise in preclinical models.

Conclusions:

  • Targeted molecular therapies offer a promising avenue to improve AT/RT treatment outcomes.
  • Further research into targeting genetic and epigenetic drivers is crucial.
  • Personalized therapeutic approaches based on molecular profiles are needed.
Keywords:
Atypical teratoid/rhabdoid tumorsSMARCA4SMARCB1SWI/SNF complexTargeted molecular therapy

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