GSK is licensing an ABL Bio technology that yields bispecific antibodies engineered to leverage a certain transmembrane receptor to cross the blood-brain barrier to treat neurodegeneration. GSK is already partnered with Alector, which has monoclonal antibodies in clinical development for Alzheimer’s disease.

In a Phase I clinical trial reported in Nature Communications, two researchers from UT Southwestern and their colleagues showed this strategy was safe and effective. The team worked with 21 patients at medical centers, including UTSW, who had recurrent glioblastoma. They were divided into six groups, each of which received a different dose of radiation-emitting nanoparticles through CED. Patients who received the highest doses had tolerable side effects and lived an average of 17 more months after treatment, significantly longer than expected for patients with recurrent glioblastoma. The authors suggest this strategy shows promise for improving treatments for these patients.

As recently reported in Neuro-Oncology, a team led by John Liu at the University of California, San Francisco Brain Tumor Center has developed a CRISPR–Cas9-based epigenetic editing approach that sensitizes glioblastoma cells to standard chemotherapy drugs. This technology could improve the treatment of tumours that have developed resistance to these drugs.

Researchers are continuously on the lookout for innovative treatment strategies for glioblastoma, a notoriously aggressive brain cancer. Recently, an exciting preclinical study published in the esteemed journal Oncotarget explored the combination of imipridones—specifically, ONC201 and its analog ONC206—with traditional therapies like radiation (RT) and temozolomide (TMZ). The research team, led by Brown University’s Lanlan Zhou under the guidance of Wafik S. El-Deiry, focuses on a revolutionary treatment regimen termed IRT—imipridones, radiation, and temozolomide. This therapy has demonstrated a potential breakthrough in reducing tumor burden and prolonging survival in an orthotopic IDH-WT glioblastoma mouse model.

A new research paper was published in Oncotarget, Volume 16, on March 27, 2025, titled "Imipridones ONC201/ONC206 + RT/TMZ triple (IRT) therapy reduces intracranial tumor burden, prolongs survival in orthotopic IDH-WT GBM mouse model, and suppresses MGMT."

Using the proliferation–diffusion model to classify tumors, we identified moderately diffuse tumors with methylated MGMT status as a subgroup with significant survival benefits from SUPR. Our findings also emphasize the need for standardized definitions of tumor invasiveness cutoffs and the limitations of current imaging modalities in accurately estimating tumor cell density.

Glioblastoma (GBM) is a difficult disease to treat for different reasons, with the blood–brain barrier (BBB) preventing therapeutic drugs from reaching the tumor being one major hurdle. The median overall survival is only 14.6 months after the standard first line of treatment. At relapse, there is no recognized standard second-line treatment. Our team uses intra-arterial (IA) chemotherapy as a means to bypass the BBB, hence achieving an overall median survival of 25 months.

Researchers from the University of Texas Health Science Center at San Antonio have developed a drug that more than doubled median survival time and progression-free time for patients with glioblastoma during a clinical trial. Patients who received the drug Rhenium Obisbemeda, or 186RNL, also experienced no dose-limiting toxic effects, according to a March 7 news release from UT Health San Antonio.

Recent research has spotlighted ataxin 3 (ATXN3) as a key player in eleviating glioblastoma (GBM) malignancy by promoting epithelial-mesenchymal transition (EMT). The aggressive nature of GBM—the most common and lethal primary brain tumor—poses significant treatment challenges, as reflected by high rates of recurrence and patient mortality. Findings published on March 6, 2025, reveal ATXN3's role as a deubiquitinase for ZEB1, enhancing cell invasion and migration, offering promising insights for therapeutic strategies.

Intraoperative Raman spectroscopy uses near-infrared laser light to gain molecular information without causing damage. It can be used in vivo or ex vivo without exogenous contrast agents. Clinically, the technique was primarily used with machine learning for in situ tumor detection with fiberoptics probes analyzing tissue at sub-millimeter scales one point at the time. Here we report the development of a whole-specimen spectroscopic imaging system designed to detect cancer cells at the margins of surgical specimens.

UCLA scientists have identified a potential new strategy for treating glioblastoma, the deadliest form of brain cancer, by reprogramming aggressive cancer cells into harmless ones.

Craniopharyngioma is an uncommon, slow-growing brain tumor that can occur in children or adults. Though there are two types of craniopharyngioma; children with craniopharyngioma almost always have the adamantinomatous (ACP) subtype. These tumors usually have some areas that are solid and some areas that are cystic (made up of pockets of trapped fluid).

Drug screening based on genetic changes in disease revealed that (S)-amlodipine besylate could shrink tumors in the mouse models and in a patient-derived xenograft mouse model. The therapeutic effects were associated with dampened calcium transients implicated in neuron-tumor cell interactions and with neuroendocrine neuronal activity. Together, the results illuminate tumor biology and suggest new avenues for therapy development.

New research shows that GD2 CAR-T cell therapy offers a potential cure for neuroblastoma, with some patients achieving long-term remission for over a decade—marking a major milestone in solid tumor treatment.

El tratamiento estándar varía en función del paciente, pero en los gliomas de alto grado suele incluir cirugía, radioterapia y quimioterapia. Según el doctor Juan Manuel Sepúlveda, neuroncólogo del Hospital Universitario 12 de Octubre, “en los niños existen más tipos diferentes de gliomas y en adultos, sin embargo, predomina el glioblastoma. En niños es más probable encontrar mutaciones que permiten tratamientos dirigidos, mientras que en adultos el tratamiento, casi siempre, es radioterapia y quimioterapia. Por desgracia, el pronóstico en adultos y en niños es similar, con una mortalidad elevada”.

Our multiparametric MRI-based prediction model has the capability to identify an inherently prognostic tumor characteristic by accurately distinguishing TP from PsP. This distinction significantly impacts patient management and clinical outcomes for months and even years post-diagnosis by enabling early and appropriate therapeutic interventions. However, our findings require further validation in future studies including larger patient populations.

At the forefront of these advancements is the vaccine-induced T cell receptor (TCR) therapy targeting the PTPRZ1 antigen, which is significantly implicated in glioblastoma cell stemness. This promising new treatment stems from the extraordinary adaptability of TCRs, engineered to recognize specific antigens found on the surface of cancer cells. PTPRZ1, or protein tyrosine phosphatase receptor type Z1, has surfaced as a tempting target due to its overexpression within glioblastoma tumors and its association with cancer cell stemness.

Five-year overall survival in patients with glioblastoma, IDH-wildtype is extremely low. Predictors of a longer survival are mostly treatment factors, emphasizing the importance of a complete oncological treatment plan, when achievable. Glioblastoma, IDH-wildtype 5-year survivors could be screened for actionable targets in case of recurrence.

Despite the emergence of new therapies, monotherapy approaches have not shown significant improvements, highlighting the need for innovative therapeutic strategies. Combination therapies appear to be the most promising solution, as they target multiple molecular pathways involved in GBM progression. One area of growing interest is the incorporation of phytotherapy and micotherapy as complementary treatments, which offer potential benefits due to their anti-tumor, anti-inflammatory, and immunomodulatory properties.

"Glioblastoma takes its cues from hormones released by the same central clock in the host that establishes the body's regular daily rhythms," said Erik D. Herzog, PhD, the Viktor Hamburger Distinguished Professor and a professor of biology in Arts & Sciences, senior author of the study.