Neuroblastoma in the Era of Precision Medicine: A Clinical Review

A Wahba, R Wolters, JH Foster - Cancers, 2023 - mdpi.com
Simple Summary Patients with high-risk neuroblastoma, especially those whose disease
either does not respond or recurs, have a poor chance of survival. Additionally, the majority …

MAPK inhibitor sensitivity scores predict sensitivity driven by the immune infiltration in pediatric low-grade gliomas

R Sigaud, TK Albert, C Hess, T Hielscher… - Nature …, 2023 - nature.com
Pediatric low-grade gliomas (pLGG) show heterogeneous responses to MAPK inhibitors
(MAPKi) in clinical trials. Thus, more complex stratification biomarkers are needed to identify …

[HTML][HTML] Paediatric Strategy Forum for medicinal product development in mitogen-activated protein kinase pathway inhibitors: ACCELERATE in collaboration with the …

ADJ Pearson, C Allen, J Fangusaro, C Hutter… - European Journal of …, 2022 - Elsevier
As the mitogen-activated protein kinase (MAPK) signalling pathway is activated in many
paediatric cancers, it is an important therapeutic target. Currently, a range of targeted MAPK …

Tazemetostat for tumors harboring SMARCB1/SMARCA4 or EZH2 alterations: results from NCI-COG pediatric MATCH APEC1621C

SN Chi, JS Yi, PM Williams… - JNCI: Journal of the …, 2023 - academic.oup.com
Abstract Background National Cancer Institute-Children's Oncology Group Pediatric
Molecular Analysis for Therapy Choice assigns patients aged 1-21 years with refractory …

Successful treatment of non-Langerhans cell histiocytosis with the MEK inhibitor trametinib: a multicenter analysis

A Aaroe, R Kurzrock, G Goyal, AM Goodman… - Blood …, 2023 - ashpublications.org
Erdheim-Chester disease (ECD) and Rosai-Dorfman disease (RDD) are rare non-
Langerhans cell histiocytoses (non-LCHs), for which therapeutic options are limited. MAPK …

Outcome of children and adolescents with relapsed/refractory/progressive Malignancies treated with molecularly informed targeted drugs in the pediatric precision …

AE Heipertz, KW Pajtler, E Pfaff, K Schramm… - JCO precision …, 2023 - ascopubs.org
PURPOSE INFORM is an international pediatric precision oncology registry, prospectively
collecting molecular and clinical data of children with recurrent, progressive, or very high …

Hydroxychloroquine prevents resistance and potentiates the antitumor effect of SHP2 inhibition in NF1-associated malignant peripheral nerve sheath tumors

SF Sait, KH Tang, SP Angus, R Brown, D Sun… - Proceedings of the …, 2025 - pnas.org
Malignant peripheral nerve sheath tumors (MPNSTs) are aggressive sarcomas and the
primary cause of mortality in patients with neurofibromatosis type 1 (NF1). These …

Palbociclib in Solid Tumor Patients With Genomic Alterations in the cyclinD-cdk4/6-INK4a-Rb Pathway: Results From National Cancer Institute-Children's Oncology …

ME Macy, R Mody, JM Reid, J Piao, L Saguilig… - JCO Precision …, 2024 - ascopubs.org
PURPOSE The National Cancer Institute-Children's Oncology Group Pediatric Molecular
Analysis for Therapy Choice trial assigned patients age 1-21 years with relapsed or …

Basket Trials: Past, Present, and Future

YR Murciano-Goroff, M Uppal, M Chen… - Annual Review of …, 2023 - annualreviews.org
Large-scale tumor molecular profiling has revealed that diverse cancer histologies are
driven by common pathways with unifying biomarkers that can be exploited therapeutically …

Unlocking the power of precision medicine for pediatric low-grade gliomas: molecular characterization for targeted therapies with enhanced safety and efficacy

S Cipri, G Del Baldo, F Fabozzi, L Boccuto… - Frontiers in …, 2023 - frontiersin.org
In the past decade significant advancements have been made in the discovery of targetable
lesions in pediatric low-grade gliomas (pLGGs). These tumors account for 30-50% of all …