Targeted Therapy


An agent targeted at the underlying molecular defect(s) of the cancer is used in treatment. This has the potential to be effective and less toxic.

There are 3 classes of targeted therapy for childhood cancer:

  • Monoclonal antibodies
  • Small molecule agents
  • Nucleic acid based agents

Earliest targeted therapy

  • Antibodies directed against cell surface markers of differentiation 20 (CD20), CD33, and CD52.
  • present on lymphoma and leukemia cells
  • CD20 is also present on normal lymphoid cells, targeting of this molecule affects overall immune function.

Rituximab (Rituxan)

  • Monoclonal antibody directed against the B-cell surface antigen CD20
  • Treats autoimmune diseases (rheumatoid arthritis) and NHL (non-Hodgkin's lymphoma)


Epidermal growth factor receptor= EGFR (also called HER1).

  • A tyrosine kinase EGFR
  • present in multiple tumor types and contributes to cancer cell
    • proliferation
    • invasion
    • migration
  • activated by binding of specific ligands and then triggers intracellular signaling which leads to cancer cell division and spread.
  • This is the treatment target for
    • monoclonal antibodies like cetuximab (Erbitux)
    • small molecule inhibitors such as erlotinib (Tarceva), gefitinib (Iressa) in multiple tumor types.


Vascular endothelial growth factor = VEGF. A

  • Signaling protein involved in angiogenesis
  • Binds to tyrosine kinases (VEGF receptors) to initiate and promote angiogenesis.
  • It is a target of treatment for the monoclonal antibody bevacizumab (Avastin)





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