Molecularly Targeted Therapy for Pediatric Brain Tumors

Ke, Warren

doi:10.1007/s11060-005-6765-5

Cited by 9 publications

(2 citation statements)

References 69 publications

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“…As noted above, alterations of EGFR expression or of the ErbB signaling pathway are involved in tumor growth, angiogenesis, invasion, metastasis, and apoptosis inhibition. Truncation, overexpression, and amplification of EGFR have been reported as drivers in various types of pediatric brain tumors [ 117 , 118 ].…”

Section: Potential Role Of Aptamers In Pediatric Neuro-oncologymentioning

confidence: 99%

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Aptamers: Novel Therapeutics and Potential Role in Neuro-Oncology

Amero

Khatua

Rodríguez-Aguayo

et al. 2020

Cancers

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A relatively new paradigm in cancer therapeutics is the use of cancer cell–specific aptamers, both as therapeutic agents and for targeted delivery of anticancer drugs. After the first therapeutic aptamer was described nearly 25 years ago, and the subsequent first aptamer drug approved, many efforts have been made to translate preclinical research into clinical oncology settings. Studies of aptamer-based technology have unveiled the vast potential of aptamers in therapeutic and diagnostic applications. Among pediatric solid cancers, brain tumors are the leading cause of death. Although a few aptamer-related translational studies have been performed in adult glioblastoma, the use of aptamers in pediatric neuro-oncology remains unexplored. This review will discuss the biology of aptamers, including mechanisms of targeting cell surface proteins, various modifications of aptamer structure to enhance therapeutic efficacy, the current state and challenges of aptamer use in neuro-oncology, and the potential therapeutic role of aptamers in pediatric brain tumors.

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Section: Potential Role Of Aptamers In Pediatric Neuro-oncologymentioning

confidence: 99%

“…It regulates tumor progression, cell proliferation, invasion, angiogenesis, and neuronal differentiation. Alterations of the receptor, as well as its ligands, have been observed in gliomas, medulloblastoma, and ependymoma [ 117 , 118 ].…”

Section: Potential Role Of Aptamers In Pediatric Neuro-oncologymentioning

confidence: 99%

Aptamers: Novel Therapeutics and Potential Role in Neuro-Oncology

Amero

Khatua

Rodríguez-Aguayo

et al. 2020

Cancers

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Magnetic Resonance Spectroscopy of Pediatric Brain Tumors

Gill

Panigrahy

Arvanitis

et al. 2012

MR Spectroscopy of Pediatric Brain Disorders

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Current therapy for medulloblastoma

Gottardo¹,

Gajjar

2006

Curr Treat Options Neurol

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In the past three decades, the survival for patients with medulloblastoma has improved remarkably. Contemporary "standard" therapy for children with medulloblastoma consists of maximal surgical resection followed by craniospinal irradiation with a boost to the posterior fossa, combined with adjuvant chemotherapy. The use of such multimodal therapeutic approaches results in progression-free survival (PFS) rates of 75% to 80% for patients with average-risk disease and approximately 60% for high-risk patients. However, despite the marked improvements in survival, many therapeutic challenges remain. Children with macroscopic metastatic disease (M2/M3) at presentation continue to fare poorly, with the best reports only attaining PFS rates up to 40%. Furthermore, despite intensive multimodal therapy, some patients have disease progression or recurrence, which for most remains incurable. The early recognition of these patients is imperative in order to institute treatment modifications, such as intensification and/or the use of novel experimental therapies. Additionally, the price for cure is clearly evident in survivors, who suffer from significant, often debilitating long-term neurocognitive and neuroendocrine sequela. Using the current clinical stratification system, a significant number of patients are overtreated and unnecessarily subjected to these long-term toxicities. This group of patients would benefit from reductions in therapy. Refinements in patient stratification and further improvement in outcome are unlikely to be achieved without improved knowledge of tumor biology. Several molecular alterations have already been identified, many of which appear to have prognostic significance. Furthermore, the disruption of molecular alterations in signaling pathways involved in the development and maintenance of medulloblastoma using novel molecularly targeted therapies promises to improve outcomes and reduce toxicity for patients with medulloblastoma. It is envisaged that in the near future children diagnosed with medulloblastoma will be more accurately stratified based on a combination of clinical variables and molecular profiles. Improved risk stratification will permit delivery of individualized therapy using conventional treatment modalities in conjunction with novel targeted therapeutic approaches.

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Molecularly Targeted Therapy for Pediatric Brain Tumors

Cited by 9 publications

References 69 publications

Aptamers: Novel Therapeutics and Potential Role in Neuro-Oncology

Aptamers: Novel Therapeutics and Potential Role in Neuro-Oncology

Magnetic Resonance Spectroscopy of Pediatric Brain Tumors

Current therapy for medulloblastoma

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