Phase I Pilot Study to Evaluate the Anti-glioblastoma Effect of S-Gboxin in Standard Treatment of Glioblastoma/Diffuse Midline Glioma and Response to Treatment (Regardless of Mutation Status)

The objective of this pilot study is to evaluate the efficacy of adding S-Gboxin to standard RT/TMZ treatment protocols in patients with glioblastoma multiforme (GBM) or midline glioma (DMG), regardless of their mutation status

Start Date11 Apr 2025

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100 Clinical Results associated with Gboxin

100 Translational Medicine associated with Gboxin

100 Patents (Medical) associated with Gboxin

Literatures (Medical) associated with Gboxin

01 Dec 2024·Bioorganic Chemistry

Y9, a Gboxin analog, displays anti-tumor effect in non-small cell lung cancer by inducing lysosomal dysfunction and apoptosis

Article

Author: Tong, Qingyi ; Yin, Jie ; Xiao, Yang ; Yao, Si ; Huang, Jianzheng ; Gu, Lianghu ; Wang, Yue ; Rehmutulla, Mewlude ; Zhang, Yonghui ; Ding, Longjie ; Zhang, Biqiong

Non-small cell lung cancer (NSCLC) ranks among the most prevalent malignancies globally. Gboxin, a novel inhibitor of mitochondrial complex V that exerts unique anti-tumor effects via oxidative phosphorylation inhibition, but shows no efficacy against NSCLC in vivo. Through chemical structure optimization, we designed and synthesized Gboxin analog Y9, which demonstrates significantly enhanced potency over its predecessor. Specifically, Y9 inhibited NSCLC significantly more strongly than Gboxin and possessed the ability to inhibit cell cycle progression and induce oxidative stress similar to Gboxin. Further investigation revealed that unlike Gboxin, Y9 selectively acidifies lysosomes and induces lysosomal dysfunction. This leads to hyperactive autophagy with impaired substrate clearance, and ultimately resulting in apoptosis. Animal studies confirmed the efficacy of Y9 in suppressing tumor growth in a xenograft mouse model. Collectively, Y9 is a distinctive Gboxin analog that outperforms its prototype by inducing lysosomal dysfunction and apoptosis, and has the potential to be developed as a novel anti-NSCLC lead compound.

01 Oct 2024·Cell Reports Medicine

Clinical-proteomic classification and precision treatment strategy of chordoma

Article

Author: Meng, Tong ; Lin, Jun ; Yin, Wenxuan ; Gao, Jianxuan ; Jiang, Cong ; Zhou, Wang ; Cai, Zhengdong ; Wang, Ping ; Su, Tong ; Chang, Zhengyan ; Sun, Wei ; Hu, Jinbo ; Song, Dianwen ; Yin, Huabin ; Xu, Xiaowen ; Jin, Jiali

Chordoma is a rare and heterogeneous mesenchymal malignancy, with distinct clinical and biological behaviors. Till now, its comprehensive clinical-molecular characteristics and accurate molecular classification remain obscure. In this research, we enroll 102 patients with chordoma and describe their clinical, imageological, and histopathological features. Through tandem mass tag-based proteomic analysis and nonnegative matrix factorization clustering, we classify chordoma into three molecular subtypes: bone microenvironment-dominant, mesenchymal-derived, and mesenchymal-to-epithelial transition-mediated pattern. The three subtypes exhibit discrete clinical prognosis and distinct biological attributes of osteoclastogenesis and immunogenicity, oxidative phosphorylation, and receptor tyrosine kinase activation, suggesting targeted therapeutic strategies of denosumab, S-Gboxin, and anlotinib, respectively. Notably, these approaches demonstrate positive treatment outcomes for each subtype in vitro and in vivo. Altogether, this work sheds light on the clinical-proteomic characteristics of chordoma and provides a candidate precision treatment strategy for chordoma according to molecular classification, underscoring their potential for clinical application.

28 Jul 2023·Nature communicationsQ1 · CROSS-FIELD

Cancer cell-mitochondria hybrid membrane coated Gboxin loaded nanomedicines for glioblastoma treatment.

Q1 · CROSS-FIELD

ArticleOA

Author: Shi, Bingyang ; Zheng, Meng ; Wang, Yibin ; Wang, Xin ; Sun, Yajing ; Zou, Yan ; Zhang, Dongya ; Yang, Huiqing

Glioblastoma (GBM) remains the most lethal malignant tumours. Gboxin, an oxidative phosphorylation inhibitor, specifically restrains GBM growth by inhibiting the activity of F₀F₁ ATPase complex V. However, its anti-GBM effect is seriously limited by poor blood circulation, the blood brain barrier (BBB) and non-specific GBM tissue/cell uptake, leading to insufficient Gboxin accumulation at GBM sites, which limits its further clinical application. Here we present a biomimetic nanomedicine (HM-NPs@G) by coating cancer cell-mitochondria hybrid membrane (HM) on the surface of Gboxin-loaded nanoparticles. An additional design element uses a reactive oxygen species responsive polymer to facilitate at-site Gboxin release. The HM camouflaging endows HM-NPs@G with unique features including good biocompatibility, improved pharmacokinetic profile, efficient BBB permeability and homotypic dual tumour cell and mitochondria targeting. The results suggest that HM-NPs@G achieve improved blood circulation (4.90 h versus 0.47 h of free Gboxin) and tumour accumulation (7.73% ID/g versus 1.06% ID/g shown by free Gboxin). Effective tumour inhibition in orthotopic U87MG GBM and patient derived X01 GBM stem cell xenografts in female mice with extended survival time and negligible side effects are also noted. We believe that the biomimetic Gboxin nanomedicine represents a promising treatment for brain tumours with clinical potential.

100 Deals associated with Gboxin

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Indication	Highest Phase	Country/Location	Organization	Date
Diffuse midline glioma, point mutation K27M in histone H3	Phase 1	Georgia	-	11 Apr 2025
Diffuse midline glioma, point mutation K27M in histone H3	Phase 1	Kazakhstan	-	11 Apr 2025
Diffuse midline glioma, point mutation K27M in histone H3	Phase 1	Ukraine	-	11 Apr 2025
Glioblastoma Multiforme	Phase 1	Kazakhstan	-	11 Apr 2025
Glioblastoma Multiforme	Phase 1	Ukraine	-	11 Apr 2025
Glioblastoma Multiforme	Phase 1	Georgia	-	11 Apr 2025
Gliosarcoma	Phase 1	Kazakhstan	-	11 Apr 2025
Gliosarcoma	Phase 1	Ukraine	-	11 Apr 2025
Gliosarcoma	Phase 1	Georgia	-	11 Apr 2025
Glioblastoma	Preclinical	United States	Memorial Sloan Kettering Cancer Center	03 Jun 2018

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