ArticleJCI insight2026
First-in-child phase I trial of p-STAT3 inhibitor WP1066 in pediatric brain tumor patients.
Article in JCI insight, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04334863 (A Phase I Study of WP1066 in Children With Refractory and Progressive or Recurrent Malignant Brain Tumors), which is not on this map. Cited by 1 paper.
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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.
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.
A Phase I Study of WP1066 in Children With Refractory and Progressive or Recurrent Malignant Brain Tumors (AflacST1901)
Who cites it
1 citing paper in PubMed.
- STAT3 signaling inhibitors for cancer treatment.Trends in pharmacological sciences · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
14 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
BACKGROUNDWP1066 is an orally bioavailable, small-molecule inhibitor of activated phosphorylated STAT3 (p-STAT3) that has demonstrated preclinical efficacy in pediatric brain tumor models.METHODSIn a first-in-child, single-center, single-arm 3+3 design phase I clinical trial, 10 patients were treated with WP1066 twice daily, Monday-Wednesday-Friday, for 14 days of each 28-day cycle to determine the maximum tolerated dose/maximum feasible dose of WP1066. Compassionate-use treatment with WP1066 in 3 pediatric patients with H3.3G34R/V-mutant high-grade glioma (HGG) is also described.RESULTSThere was no significant toxicity, and the maximum feasible dose (MFD) was determined to be 8 mg/kg. Treatment-related adverse events were grade 1-2 (diarrhea and nausea most common); there were no dose-limiting toxicities. Median progression-free and overall survival was 1.8 months and 4.9 months, respectively. One partial response was observed in a patient with pontine glioma. Among the H3.3G34R/V-mutant HGG patients not on study, WP1066 was administered after upfront radiation to one patient for 17 months. At all dose levels tested, WP1066 suppressed p-STAT3 expression by peripheral blood mononuclear cells (PBMCs). Single-cell RNA sequencing analysis of PBMCs demonstrated increased CD4+ and CD8+ T cells, proinflammatory TNFA signaling, differentiation activity in myeloid cells, and downregulation of Tregs after WP1066 treatment, consistent with systemically inhibited STAT3 activity.CONCLUSIONWP1066 is safe, has minimal toxicity, and induces antitumor immune responses in pediatric brain tumor patients. Phase II investigation of WP1066 at the MFD in this patient population is warranted.TRIAL REGISTRATIONClinicalTrials.gov NCT04334863.FUNDINGCURE Childhood Cancer and Peach Bowl Inc.
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