Glioblastoma is the most common and most aggressive primary malignant brain tumor in adults, with rapid progression and poor overall prognosis. Clinical manifestations vary by tumor location and include headache, seizures, neurologic deficits, and cognitive changes. Standard treatment generally combines surgery, radiotherapy, and temozolomide-based chemotherapy.
UVA Health found gliomas may be more receptive to focused ultrasound drug delivery. Related research covers miRNA therapy, low-intensity focused ultrasound, and 3D organoid drug testing.
Lantern Pharma's LP-284 received FDA Orphan Drug Designation for soft tissue sarcomas, while the USPTO allowed a patent for biomarker-guided use of LP-184 in multiple cancers. LP-284 showed a complete metabolic response in a Phase 1 DLBCL patient, and LP-184 demonstrated a 45% disease control rate in advanced solid tumors.
Researchers at MD Anderson developed an extracellular vesicle-based platform that delivers full-length DMD mRNA, restoring dystrophin production and improving muscle function in preclinical Duchenne muscular dystrophy models. The study, published in Nature Biomedical Engineering, showed reduced side effects compared with viral gene therapies.
Recent studies show progress in cancer treatment: HDAC inhibitors enhance pancreatic cancer therapy, AI-guided radiation targets glioblastoma, and reprogrammed tumor cells trigger antitumor immunity. Nanoparticle drug delivery for brain cancer is also being explored.
The global oncology drugs market is projected to grow at an 8-10% CAGR through 2035 as targeted therapies and immunotherapies gain share over chemotherapy. The radiopharmaceuticals market is expected to reach $21.8 billion by 2033, fueled by rising cancer incidence and expanding diagnostic and therapeutic applications. Innovations in drug delivery, including intranasal platforms for glioblastoma, are advancing neuro-oncology treatment options.
A world-first clinical trial testing immunotherapy for glioblastoma has opened in the US. The GIANT trial will evaluate Nivolumab and Relatlimab before surgery, with results expected in about two years. Three patients have been enrolled so far.
A new "expand and pull" treatment strategy combines long-acting interleukin-7 and oncolytic virus therapy to boost T cell response in glioblastoma. In mouse models, the combination improved cytotoxic T cell function and led to long-term tumor-free survival.
BioNTech presented phase 2 data for HER2-directed ADC Trastuzumab Pamirtecan showing 47.9% response rate in endometrial cancer, with FDA submission planned for 2026. BioLineRx dosed first patient in phase 1/2a GLIX1 trial for glioblastoma and reported new preclinical data. Both companies presented at ASCO 2026.
Researchers report advances in RNA-based gene therapy: an FDA-approved small molecule modulates RNA splicing, protein-coated nanoparticles enable safer delivery, and a microRNA platform targets glioblastoma.
A phase I trial and a preclinical study found viral immunotherapy triggered T cell responses in glioblastoma. The clinical study enrolled 41 patients; the mouse study showed prolonged survival and immunologic memory.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07775534 |
Tear Fluid for the Detection of Tumor-associated Extracellular Vesicles in Glioblastoma Patients |
NOT_YET_RECRUITING | NA |
| NCT07758062 |
Levetiracetam Extended-Release Plus Stupp Protocol for Hypoxic IDH-Wildtype Glioblastoma |
NOT_YET_RECRUITING | PHASE2 |
| NCT07748299 |
NuvOx Expanded Access |
AVAILABLE | |
| NCT07735819 |
Positron Emission Tomography/Computed Tomography (PET/CT) Imaging in Patients Diagnosed With a Glioma |
NOT_YET_RECRUITING | PHASE2 |
| NCT07731555 |
Characterization of the Blood-Brain Barrier in High-Grade Glioma Through Immunohistochemical, Transcriptional, Fluorescein and Radiological Analyses |
RECRUITING | NA |
| NCT07715097 |
Survivin-Targeted Dendritic Cell (DC) Cell Injection for Newly Diagnosed Glioblastoma |
NOT_YET_RECRUITING | PHASE2 |
| NCT07689721 |
ctDNA-Guided Intraventricular Therapy for CNS Malignancies |
NOT_YET_RECRUITING | NA |
| NCT07678138 |
Exploratory Clinical Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of Tumor Neoantigen-pulsed Autologous Dendritic Cell Injection (YS247, Beijing YSCell Biotech Co., Ltd. [Abbreviated as YS])in Patients With Recurrent or Progressive Glioblastoma |
NOT_YET_RECRUITING | NA |
| NCT07674095 |
Assessment of a Large US EHR Database as Meeting FDA Fit-for-Purpose Requirements to Provide External Controls for Study BPM31510 in Participants With Glioblastoma |
ACTIVE_NOT_RECRUITING | |
| NCT07670026 |
Intensified Adjuvant Therapy for High-Risk Newly Diagnosed Glioblastoma With Subtotal Resection or Short-Term Progression |
RECRUITING | PHASE2 |
