Introduction: Neuroblastoma is a tumor of the developing sympathetic nervous system. Low and intermediate-risk patients usually have good treatment outcomes, whereas high-risk cases have poorer survival and recurrence rates. This review highlights limitations in the treatment procedure and future therapies in development that may be adopted into clinical practice, compiled from a literature search of scientific papers from the last 30 years including ongoing clinical trials.Areas covered: Current treatments for high-risk neuroblastoma have shown efficacy in clinical trials; however, this regimen is not effective in preventing relapse in many cases and has high toxicity for pediatric patients. The two main areas of research for new maintenance therapies focus on immunotherapy and gene targeting with molecular therapy. GD2-CAR-T cells and GD2 vaccines have shown efficacy in pre-clinical trials, and MYCN and ALK inhibition target two of the main driver mutations in neuroblastoma potentially offering a highly specific form of therapy.Expert opinion: Tumor heterogeneity leads to various drivers of neuroblastoma; therefore, combinations of molecular therapies can induce remission, alongside immunotherapy that could lower treatment toxicity. The implementation of ‘liquid biopsies’ could greatly improve genetic characterization of a changing tumor profile (often changing in response to treatment) to inform appropriate therapies.

Abhinav Kumar

John P J Rocke

B Nirmal Kumar

Expert Opinion on Orphan Drugs

EXPERT OPINION ON ORPHAN DRUGS 是一本经过同行评审的国际期刊，涵盖了罕见病和孤儿药研发的各个方面。

Expert Opinion on Orphan Drugs is a peer-reviewed international journal that focuses on the research and development of orphan drugs and rare diseases, covering various aspects of the field.

《Expert Opinion on Orphan Drugs》是一本经过同行评审的国际期刊，专注于罕见病和孤儿药的研发，涵盖了相关领域的各个方面。

Category	Quartile
医学	4
医学, 药学	4

Evolving treatments in high-risk neuroblastoma

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Journal Info

Category	Quartile
PHARMACOLOGY & PHARMACY	4