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Targeted Therapies in Neuroblastoma: A History and a View to the Future
Nilay Shah1,2
1Division of Hematology/Oncology/BMT, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Abstract:
Neuroblastomas remain a disproportionately morbid and mortal pediatric cancer. Representing 8% of all childhood cancer diagnoses, it still is the cause of 14% of cancer-related deaths in children despite the use of aggressive multimodal treatments with significant long-term sequelae. The development of targeted therapies for neuroblastoma has been and will continue to be the optimal approach to improve durable long-term survival in these patients while reducing the consequences of treatment. Here, I review the history of the development of targeted therapies for the treatment of neuroblastoma. I discuss the history of therapies that have been integrated into current treatment regimens, including isotretinoin, anti-GD2 antibodies and eflornithine. I review the history of candidate biologic targets in this malignancy as well as ongoing and future efforts to improve outcomes.
Insights
Neuroblastomas are aggressive pediatric cancers causing significant deaths despite treatment. Targeted therapies, including isotretinoin and anti-GD2 antibodies, offer improved survival and reduced side effects for neuroblastoma patients.
Area of Science:
- Pediatric Oncology
- Cancer Therapeutics
- Molecular Oncology
Background:
- Neuroblastomas account for 8% of childhood cancer diagnoses and 14% of cancer-related deaths.
- Aggressive multimodal treatments lead to significant long-term sequelae in survivors.
- Targeted therapies are crucial for improving survival and reducing treatment-related harm.
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