High-risk and relapsed neuroblastoma: toward more cures and better outcomes

SG DuBois, ME Macy, TO Henderson - American Society of Clinical …, 2022 - ascopubs.org
Approximately half of the patients diagnosed with neuroblastoma are classified as having
high-risk disease. This group continues to have inadequate cure rates despite multiagent …

[HTML][HTML] Lorlatinib with or without chemotherapy in ALK-driven refractory/relapsed neuroblastoma: Phase 1 trial results

…, R Mody, DA Morgenstern, S Shusterman, ME Macy… - Nature medicine, 2023 - nature.com
Neuroblastomas harbor ALK aberrations clinically resistant to crizotinib yet sensitive pre-clinically
to the third-generation ALK inhibitor lorlatinib. We conducted a first-in-child study …

Opportunities and challenges in drug development for pediatric cancers

TW Laetsch, SG DuBois, JG Bender, ME Macy… - Cancer discovery, 2021 - AACR
ME Macy reports other from Johnson & Johnson (stockholder) outside the submitted work.
L. Moreno reports other from Novartis [Consultancy role and Member of DMC (fees to …

Pembrolizumab in paediatric patients with advanced melanoma or a PD-L1-positive, advanced, relapsed, or refractory solid tumour or lymphoma (KEYNOTE-051) …

…, W Nicholls, E Fox, SG DuBois, ME Macy… - The Lancet …, 2020 - thelancet.com
Background Pembrolizumab is approved for the treatment of advanced cancer in adults;
however, no information is available on safety and efficacy in paediatric patients. We aimed to …

Entrectinib and other ALK/TRK inhibitors for the treatment of neuroblastoma

HL Pacenta, ME Macy - Drug design, development and therapy, 2018 - Taylor & Francis
RTK plays important roles in many cellular signaling processes involved in cancer growth
and development. ALK, TRKA, TRKB, TRKC, and ROS1 are RTKs involved in several …

Entrectinib in children and young adults with solid or primary CNS tumors harboring NTRK, ROS1, or ALK aberrations (STARTRK-NG)

…, GW Robinson, K Gauvain, EM Basu, ME Macy… - Neuro …, 2022 - academic.oup.com
… Margaret E Macy , Margaret E Macy … Ami V Desai, Giles W Robinson, Karen Gauvain, Ellen
M Basu, Margaret E Macy, Luke Maese, Nicholas S Whipple, Amit J Sabnis, Jennifer H Foster…

Molecular profiling identifies targeted therapy opportunities in pediatric solid cancer

…, L Maese, TW Laetsch, AR Kim, SI Colace, ME Macy… - Nature medicine, 2022 - nature.com
To evaluate the clinical impact of molecular tumor profiling (MTP) with targeted sequencing
panel tests, pediatric patients with extracranial solid tumors were enrolled in a prospective …

Phase 1/1B trial to assess the activity of entrectinib in children and adolescents with recurrent or refractory solid tumors including central nervous system (CNS) tumors …

…, AJ Gajjar, KM Gauvain, EM Basu, ME Macy… - 2019 - ascopubs.org
10009 Background: Entrectinib is a CNS-penetrant oral inhibitor of TrkA/B/C, ROS1 and
ALK tyrosine kinases. We report the efficacy of entrectinib in children with recurrent/refractory …

Phase II trial of alisertib in combination with irinotecan and temozolomide for patients with relapsed or refractory neuroblastoma

…, MM Granger, B Weiss, JR Park, ME Macy… - Clinical Cancer …, 2018 - AACR
Purpose: In phase I testing, alisertib tablets with irinotecan and temozolomide showed significant
antitumor activity in patients with neuroblastoma. This study sought to confirm activity of …

[HTML][HTML] CFEOM3: a new extraocular congenital fibrosis syndrome that maps to 16q24. 2-q24. 3

EJ Doherty, ME Macy, SM Wang… - … & visual science, 1999 - arvojournals.org
purpose. To define the clinical characteristics and determine the gene localization for a
previously undescribed form of congenital fibrosis of the extraocular muscles (CFEOM), referred …