Evidence map›Paper›PMID 41038289›Full record

ReviewPharmacology & therapeutics2025

Towards directed therapy for fusion-positive rhabdomyosarcoma.

George M Turco, Sapna Oberoi, Brian Ladle, Raavi, Lars Wagner, Angela N Koehler, Corinne M Linardic

Abstract readReview
In one paragraph

Review in Pharmacology & therapeutics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

4 citing papers in PubMed.

  1. Article
  2. Article
  3. Review
  4. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors.

George M TurcoDepartment of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
Sapna OberoiDepartment of Pediatrics & Child Health, University of Manitoba, Winnipeg, Manitoba, Canada; Department of Pediatric Hematology/Oncology, CancerCare Manitoba, Winnipeg, Canada.
Brian LadleThe Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Division of Pediatric Oncology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
RaaviDepartment of Biological Engineering, Koch Institute for Cancer Research, MIT, Cambridge, MA, USA.
Lars WagnerDepartment of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
Angela N KoehlerDepartment of Biological Engineering, Koch Institute for Cancer Research, MIT, Cambridge, MA, USA.
Corinne M LinardicDepartment of Pediatrics, Duke University School of Medicine, Durham, NC, USA; Department of Pharmacology & Cancer Biology, Duke University School of Medicine, Durham, NC, USA. Electronic address: corinne.linardic@duke.edu.

Funding

Chemical Approaches to Modulate PAX3-FOXO1 in Fusion-Positive Alveolar RhabdomyosarcomaUM1CA294108 · NCI · MASSACHUSETTS INSTITUTE OF TECHNOLOGY · PI ALEX B BURGIN, Alexandra E Gould · 2024 to 2026
$6.9M
Screening for Cys-Reactive Ligands to Target PAX3-FOXO1U54CA231630 · NCI · DUKE UNIVERSITY · PI COUNTER, CHRISTOPHER M, LINARDIC, CORINNE MARY · 2019 to 2023
$6.5M
NCI NIH HHS U54 CA231630NCI NIH HHS UM1 CA294108
6 · The paper itself

Abstract

Rhabdomyosarcoma is the most common soft tissue sarcoma of childhood. The fusion-positive variant of rhabdomyosarcoma has the dubious distinction of being one of the most difficult to cure childhood cancers. Although the gene fusions PAX3::FOXO1 and PAX7::FOXO1 were discovered in the early 1990s, and since that time shown to be the molecular drivers of the disease, the best treatment to date still remains VAC (vincristine, actinomycin D, cyclophosphamide) combination therapy, first instituted as standard of care in the 1970s. Here we review the history, contemporary application, clinical evaluation, and future of fusion positive rhabdomyosarcoma systemic therapy. It is hoped that a better understanding of the underlying biology and the effective leverage of new strategies for targeting RNA, proteins, and the immune system will result in meaningful advances for treating this aggressive childhood cancer.

Indexed as

Antineoplastic Combined Chemotherapy ProtocolsOncogene Proteins, FusionRhabdomyosarcomaAnimalsCyclophosphamideDactinomycinHumansVincristineCyclophosphamideDactinomycinOncogene Proteins, FusionVincristineAlveolarPAX3::FOXO1RhabdomyosarcomaTherapy

Identifiers

PMID41038289
PMCPMC13112368

What OpenQuestion holds

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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.