Evidence map›Paper›PMID 41307127›Full record

ArticleHaematologica2026

A small molecule inhibitor of RNA-binding protein IGF2BP3 shows anti-leukemic activity.

Amit K Jaiswal, Georgia M Scherer, Michelle L Thaxton, Jacob P Sorrentino, Constance Yuen, Milauni M Mehta, Gunjan Sharma, Tasha L Lin, Tiffany M Tran, Amanda Cohen and 6 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Haematologica, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing 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

6 citing papers in PubMed.

  1. Review
  2. Article
  3. Article
  4. Article
  5. Review
  6. 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

16 authors.

Amit K JaiswalDepartment of Pathology and Laboratory Medicine, University of California, Los Angeles, CA.
Georgia M SchererDepartment of Chemistry and Biochemistry, University of California, Los Angeles, CA.
Michelle L ThaxtonDepartment of Pathology and Laboratory Medicine, University of California, Los Angeles, CA.
Jacob P SorrentinoDepartment of Chemistry and Biochemistry, University of California, Los Angeles, CA.
Constance YuenCalifornia Nanosystems Institute, University of California, Los Angeles, CA.
Milauni M MehtaDepartment of Chemistry and Biochemistry, University of California, Los Angeles, CA.
Gunjan SharmaDepartment of Pathology and Laboratory Medicine, University of California, Los Angeles, CA.
Tasha L LinDepartment of Pathology and Laboratory Medicine, University of California, Los Angeles, CA.
Tiffany M TranDepartment of Pathology and Laboratory Medicine, University of California, Los Angeles, CA.
Amanda CohenDepartment of Pathology and Laboratory Medicine, University of California, Los Angeles, CA.
Alexander J RitterDepartment of Molecular, Cell and Developmental Biology and Center for Molecular Biology of RNA, University of California, Santa Cruz, CA.
Rishi S KotechaDepartment of Clinical Haematology, Oncology, Blood and Marrow Transplantation, Perth Childrens Hospital, Perth, Australia; Leukemia Translational Research Laboratory, WA Kids Cancer Centre, University of Western Australia, Perth Australia; Curtin Medical School, Curtin University, Perth, Australia.
Jeremy R SanfordDepartment of Molecular, Cell and Developmental Biology and Center for Molecular Biology of RNA, University of California, Santa Cruz, CA, USA; Center for Biomolecular Science and Engineering, University of California, Santa Cruz, CA.
Robert D DamoiseauxCalifornia Nanosystems Institute, University of California, Los Angeles, CA, USA; Department of Molecular and Medical Pharmacology, University of California, Los Angeles, CA, USA; Department of Bioengineering, Samueli School of Engineering, University of California, Los Angeles, CA, USA; Jonsson Comprehensive Cancer Center, University of California Los Angeles, CA.
Neil K GargDepartment of Chemistry and Biochemistry, University of California, Los Angeles, CA.
Dinesh S RaoDepartment of Pathology and Laboratory Medicine, University of California, Los Angeles, CA, USA; Jonsson Comprehensive Cancer Center, University of California Los Angeles, CA, USA; Broad Stem Cell Research Center, University of California Los Angeles, CA. drao@mednet.ucla.edu.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The RNA-binding protein IGF2BP3 is an oncofetal protein over-expressed in B-cell acute lymphoblastic leukemia and is critical for leukemogenesis in experimental models. With cancer-specific expression, functional dispensability for normal development, and an unleveraged pro-oncogenic function in mRNA homeostasis, IGF2BP3 represents an excellent target. With no small molecule inhibitors of IGF2BP3 in clinical use, we aimed to identify new IGF2BP3 inhibitors using biochemical methods. A biochemical screen, followed by a cell-based counter screen led to the identification of compounds with protein-RNA interaction inhibition and leukemic cell growth-inhibitory activity. One of these compounds, designated I3IN-002, shows consistent cell growth-inhibitory activity, altered cell cycle, and increased apoptosis in multiple leukemia cell lines, and is the most potent inhibitor of IGF2BP3 reported to date. I3IN-002 was tolerated in mice when administered intraperitoneally and showed potent anti-leukemic activity in a syngeneic transplantation model of MLL-Af4 leukemia. I3IN-002 inhibits the function of IGF2BP3, disrupting in situ binding of IGF2BP3 to target mRNA, and altering IGF2BP3-dependent gene expression regulation. Furthermore, cell-free and cellular thermal shift assays, as well as drug affinity responsive target stability assays support on-target activity of I3IN-002 for IGF2BP3. Thus, the identification of I3IN-002 paves the way for the discovery of potent and selective small molecule inhibitors of IGF2BP3.

Indexed as

Antineoplastic AgentsLeukemiaRNA-Binding ProteinsSmall Molecule LibrariesAnimalsApoptosisCell CycleCell Line, TumorCell ProliferationHumansMiceAntineoplastic AgentsIGF2BP3 protein, humanRNA-Binding ProteinsSmall Molecule Libraries

Identifiers

PMID41307127
PMCPMC13231121

What Socratic holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

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 Socratic graph.