Novel Therapeutic Approach for Glioblastoma with Improved BBB Penetration
Key Advantages
- High BBB penetration.
- Efficacy against temozolomide-resistant glioblastoma cells.
- Higher brain exposure than temozolomide.
- No cytotoxicity in mammalian cell lines, zebrafish, and C. elegans.
Goal
Seeking a license before development into regulatory preclinical trials, but other collaborations may be considered.
Intellectual Property
EP filed – Priority: July 23rd, 2026
Reference
UBTT0540
Contact
Marc Salazar Badia
Email: msalazar@fbg.ub.edu
Novel Therapeutic Approach for Glioblastoma with Improved BBB Penetration
Executive summary
Researchers from the University of Barcelona (UB) and the University of Santiago de Compostela (USC) have developed novel patent-protected heterocyclic compounds with potent antiproliferative activity against glioblastoma and proven blood-brain barrier penetration. Lead candidates demonstrate efficacy comparable or superior to temozolomide, including activity in resistant tumor models.
Introduction
Glioblastoma (GBM) is the most aggressive primary brain tumor in adults, with a survival of only 12-15 months despite surgery, radiotherapy, and chemotherapy.
Current pharmacological treatment relies mainly on temozolomide, whose efficacy is limited, showing poor long-term outcomes, resistance, and little control of relapse.
A major challenge in GBM treatment is the blood-brain barrier (BBB). As a result, there is a critical unmet medical need for new BBB-permeable therapies capable of overcoming treatment resistance and improving patient outcomes.
Glioblastoma represents a significant commercial opportunity, with a global treatment market (TAM) estimated at $3 billion. Since 50-60% of patients develop resistance to temozolomide, the addressable second-line treatment (SAM) would be $1.8 billion. Focusing on Europe, the United States, and Japan, the obtainable market (SOM) is estimated at approximately $1.1 billion.
Description
Researchers have developed a novel family of compounds with antiproliferative activity against glioblastoma. More than 90 compounds have been synthesized through efficient one-step multicomponent reactions, generating a broad proprietary chemical platform. Selected compounds demonstrated potent antiproliferative activity in glioma cell lines, including temozolomide-resistant models, while exhibiting excellent BBB permeability. Lead candidates showed superior brain exposure compared with temozolomide in neuropharmacokinetic studies and generated therapeutic benefit in a glioblastoma Drosophila model, supporting their potential as novel treatments for CNS malignancies.
Next steps of development
- Lead optimization and candidate selection (ADME, PK).
- MoA determination.
- In vivo efficacy validation
- Non-regulatory preclinical development
The group has won competitive public calls to fund these activities.