Targeted therapy is a type of cancer treatment that uses drugs or other substances to precisely identify and attack cancer cells, usually by focusing on specific molecular targets associated with cancer. This approach aims to minimize damage to normal cells, thereby reducing side effects compared to traditional treatments like chemotherapy and radiation.

Mechanisms of Targeted Therapy

1. Targeting Specific Molecules
   • Molecular Targets: Targeted therapies work by interfering with specific molecules involved in tumor growth and progression. These molecules can be proteins, genes, or the environment of cancer cells.
   • Mechanism: By focusing on these targets, targeted therapies can block the growth and spread of cancer cells more effectively.
   • Sources: National Cancer Institute, American Cancer Society
2. Types of Targeted Therapies
   • Monoclonal Antibodies: These lab-created molecules can attach to specific targets on cancer cells, flagging them for destruction by the immune system or directly blocking their growth.
   • Small Molecule Inhibitors: These drugs can enter cells and interfere with specific molecules involved in cancer cell growth.
   • Sources: Mayo Clinic, National Cancer Institute

Use in Pediatric Cancer Treatment

1. Common Pediatric Cancers Treated with Targeted Therapy
   • Leukemias: Certain types of leukemias in children, such as Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL), can be treated with targeted therapies like tyrosine kinase inhibitors.
   • Neuroblastoma: Targeted therapies such as anti-GD2 monoclonal antibodies are used to treat high-risk neuroblastoma.
   • Brain Tumors: Targeted therapies can be used for specific genetic mutations found in pediatric brain tumors, such as medulloblastomas and gliomas.
   • Sources: Children's Oncology Group, American Cancer Society
2. Examples of Targeted Therapies
   • Imatinib (Gleevec): Used to treat chronic myelogenous leukemia (CML) and Ph+ ALL in children by inhibiting the BCR-ABL tyrosine kinase.
   • Dinutuximab (Unituxin): A monoclonal antibody used to treat high-risk neuroblastoma by targeting the GD2 antigen on cancer cells.
   • Vemurafenib (Zelboraf): Used for treating pediatric patients with melanoma harboring the BRAF V600E mutation.
   • Sources: National Cancer Institute, American Cancer Society

Benefits of Targeted Therapy

1. Precision: By focusing on specific molecular targets, targeted therapies can more precisely attack cancer cells while sparing healthy cells, leading to fewer side effects.
2. Effectiveness: Targeted therapies can be very effective against cancers that have specific genetic mutations or abnormal proteins.
3. Combination with Other Treatments: Targeted therapies can be used alone or in combination with other treatments such as chemotherapy, radiation, or immunotherapy to enhance overall effectiveness.
   • Sources: Mayo Clinic, National Cancer Institute

Side Effects and Management

1. Common Side Effects
   • Side effects vary depending on the specific drug but may include diarrhea, liver problems, skin problems, high blood pressure, and issues with blood clotting and wound healing.
   • Sources: American Cancer Society, National Cancer Institute
2. Managing Side Effects
   • Close monitoring by healthcare providers and supportive care measures can help manage and mitigate side effects.
   • Sources: Mayo Clinic, American Cancer Society

Conclusion

Targeted therapy offers a promising and precise approach to treating pediatric cancers by focusing on specific molecular targets involved in cancer progression. This treatment can be highly effective, especially when tailored to the unique genetic makeup of the child's cancer, and often results in fewer side effects compared to traditional therapies.

Further Reading

• National Cancer Institute: Targeted Cancer Therapies
• American Cancer Society: Targeted Therapy
• Mayo Clinic: Targeted Therapy
• Children's Oncology Group: Targeted Therapies

These resources provide comprehensive information on targeted therapy and its application in pediatric cancer treatment.