Evidence map›Paper›PMID 42447867›Full record

ArticleCell reports. Medicine2026

Targeted antibody-drug conjugates for rhabdomyosarcoma and other FGFR4-expressing cancers.

Meijie Tian, Katrina Jia, Jerry T Wu, Adam T Cheuk, Siteng Fang, Victor T Ojo, Eleanor G Pope, David Milewski, Yong Yean Kim, Hsien-Chao Chou and 15 more

Registry-linked trialAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Cell reports. Medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06865664 (Phase I Dose Escalation Study of FGFR4 Chimeric Antigen Receptor), which is not on this map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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.

NCT06865664 phase1recruitingnot on this map

Phase I Dose Escalation Study of FGFR4 Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory Rhabdomyosarcoma

TypeinterventionalSponsorNational Cancer Institute (NCI)Ran2025 to 2029Enrolled50ConditionsRhabdomyosarcomaArmsfludarabine, cyclophosphamide, cetuximab, FGFR4-CAR T Cells
3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

25 authors.

Meijie TianOncogenomics Section, Genetics Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Katrina JiaOncogenomics Section, Genetics Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Jerry T WuOncogenomics Section, Genetics Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Adam T CheukOncogenomics Section, Genetics Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Siteng FangSchool of Pharmacy and Pharmaceutical Sciences, Binghamton University, Binghamton, NY, USA.
Victor T OjoSchool of Pharmacy and Pharmaceutical Sciences, Binghamton University, Binghamton, NY, USA.
Eleanor G PopeOncogenomics Section, Genetics Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
David MilewskiOncogenomics Section, Genetics Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Yong Yean KimOncogenomics Section, Genetics Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Hsien-Chao ChouOncogenomics Section, Genetics Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Avani KelekarOncogenomics Section, Genetics Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Soumili DeyOncogenomics Section, Genetics Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Yunkai YuOncogenomics Section, Genetics Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Mohammad Asikur RahmanSchool of Pharmacy and Pharmaceutical Sciences, Binghamton University, Binghamton, NY, USA.
R M Naseer KhanClinical Pharmacology Lab, Clinical Center, National Institutes of Health Clinical Center, Bethesda, MD, USA.
Oluwatobi ArisaClinical Pharmacology Lab, Clinical Center, National Institutes of Health Clinical Center, Bethesda, MD, USA.
William D FiggClinical Pharmacology Lab, Clinical Center, National Institutes of Health Clinical Center, Bethesda, MD, USA; Clinical Pharmacology Program, Center for Cancer Research, National Institutes of Health Clinical Center, Bethesda, MD, USA.
Shyam K SharanMouse Cancer Genetics Program, Center for Cancer Research, National Cancer Institute, NIH, Frederick, MD 21702, USA; Center for Advanced Preclinical Research, Frederick National Laboratory for Cancer Research, Frederick, MD 21702, USA.
Zoe Weaver OhlerCenter for Advanced Preclinical Research, Frederick National Laboratory for Cancer Research, Frederick, MD 21702, USA.
Ludmila SzabovaCenter for Advanced Preclinical Research, Frederick National Laboratory for Cancer Research, Frederick, MD 21702, USA.
Simone DifilippantonioLaboratory Animal Sciences Program, Frederick National Laboratory for Cancer Research, Frederick, MD 21702, USA.
Borys ShorManhattan BioSolutions, Inc., New York, NY, USA.
L Nathan TumeySchool of Pharmacy and Pharmaceutical Sciences, Binghamton University, Binghamton, NY, USA.
Jun S WeiOncogenomics Section, Genetics Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA. Electronic address: weij@mail.nih.gov.
Javed KhanOncogenomics Section, Genetics Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA. Electronic address: khanjav@mail.nih.gov.

Funding

Targeting High-risk Neuroblastoma using high through put siRNA screeningZIABC010806 · NCI · DIVISION OF BASIC SCIENCES - NCI · PI KHAN, JAVED · 2009 to 2025
$7.8M
Legumain to the rescue: A new ADC linker strategy to address the limitations of cathepsin cleavageR01GM144450 · NIGMS · STATE UNIVERSITY OF NY,BINGHAMTON · PI TUMEY, LAWRENCE · 2022 to 2025
$1.2M
Intramural NIH HHS ZIA BC010806NCI NIH HHS HHSN261201500003CNCI NIH HHS HHSN261201500003INIGMS NIH HHS R01 GM144450
6 · The paper itself

Abstract

Compared with chimeric antigen receptor (CAR) T cell therapy, antibody-drug conjugates (ADCs) offer distinct advantages. Here, we report on two FGFR4-targeted ADCs, using the high-affinity monoclonal antibody 3A11, the same binder used in a CAR T cell format that is being evaluated at the NCI (NCT06865664) for patients with relapsed/refractory rhabdomyosarcoma (RMS). These ADCs, conjugated to monomethyl auristatin E (MMAE) or an exatecan derivative, are rapidly internalized, causing potent fibroblast growth factor receptor 4 (FGFR4)-dependent cytotoxicity in vitro. In subcutaneous RMS xenograft models, both ADCs demonstrated robust anti-tumor activities, significantly prolonging survival. Notably, exatecan-ADC showed better efficacy, with durable tumor control, in aggressive fusion-negative (FN) RMS559 and fusion-positive (FP) RH4 RMS cell-line-derived xenografts (CDXs) and a patient-derived RMS xenograft (PDX). Furthermore, exatecan-ADC effectively controls tumors in an FGFR4-expressing MDA-MB-453 breast cancer mouse model, eradicating relapsed tumors with retreatment. These findings highlight FGFR4-targeted ADCs as potent therapeutic agents against aggressive FGFR4-expressing malignancies, supporting their further clinical development.

Indexed as

ImmunoconjugatesReceptor, Fibroblast Growth Factor, Type 4RhabdomyosarcomaAnimalsAntibodies, MonoclonalCell Line, TumorFemaleHumansMiceOligopeptidesXenograft Model Antitumor AssaysAntibodies, MonoclonalFGFR4 protein, humanImmunoconjugatesmonomethyl auristatin EOligopeptidesReceptor, Fibroblast Growth Factor, Type 4antibody-drug conjugatebreast cancerexatecanFGFR4-expressing malignanciesFGFR4-targeted ADClegumain-cleavable linkerMMAEpediatric cancerrhabdomyosarcoma

Identifiers

PMID42447867
PMCPMC13400184

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Registered trials

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.