Ultrasound-mediated drug delivery using micro/nanobubble technology holds great potential for revolutionizing the treatment of neurological diseases. Low-frequency ultrasound enables precise and safe delivery of therapeutic agents across the BBB, marking a key advancement in treating neurological disorders previously difficult to manage with conventional approaches. While essential clinical trials are underway, they represent just the initial phase of this groundbreaking technology. Advancing ultrasound-mediated drug delivery will require cross-disciplinary collaboration, optimized bubble technology, and strict adherence to FDA guidelines.

Our study exhibits an extensive investigation of the prognostic factors influencing survival outcomes in a cohort of patients diagnosed with IDH-wildtype glioma submitted to biopsy. This study highlights the importance of KPS both before and after surgery and any form of adjuvant treatment in determining patient outcomes. A decline in KPS post-operatively was indicative of poorer prognosis. We also clarified the adverse impact of cognitive impairment on PFS and OS for patients with mild cognitive deficits, and even higher for those severely impaired. Contrary to traditional fears regarding aggressive treatments in older patients, our data did not show a significant age-related impact on the three-month survival rate after adjuvant therapy in patients able to comply with treatment. While the volumetry, location, and hemisphere dominance of gliomas did not appear as significant predictors, lack of contrast uptake in imaging studies did, and it was associated with a notably poorer PFS and OS, as an independent variable. Finally, a critical observation from our data is the clear survival benefit associated with adjuvant therapies post-biopsy, irrespective of their pre-operative clinical status.

Scientists from the University of Geneva have developed CAR-T cells targeting the PTPRZ1 marker on glioblastoma cells, a promising step toward more effective treatments. These engineered immune cells showed the ability to attack both marked and unmarked tumor cells without harming healthy cells, successfully extending the lives of mice in preclinical trials.

Researchers have developed a promising CAR-T cell therapy targeting glioblastoma, the most aggressive brain tumor. The therapy uses genetically modified T cells to target a specific marker on tumor cells, potentially overcoming the limitations of traditional treatments. Notably, these CAR-T cells also show an ability to fight tumor cells lacking the marker, suggesting a bystander effect. Early trials in mice demonstrate that this approach effectively controls tumor growth with minimal toxicity, setting the stage for human clinical trials.

Researchers have discovered that the antidepressant vortioxetine effectively combats glioblastoma, a deadly brain tumor, in lab tests and in mice. This drug, already FDA-approved, crosses the blood-brain barrier and could potentially improve treatment for glioblastoma patients when combined with standard therapy.

In a first-in-human clinical trial, an mRNA-based cancer vaccine for glioblastoma showed promising results by inducing T-cell responses in over three-quarters of patients with an acceptable safety profile. The vaccine was able to generate new immune responses and break through immune tolerance to the tumor.

An international team of investigators, including Providence’s Santosh Kesari, MD, PhD, has achieved a significant breakthrough in glioblastoma treatment. Glioblastoma is one of the deadliest types of brain cancer, with a median overall survival for GBM patients is around 15 months. The researchers found that a personalized peptide vaccine showed improved survival and strong immune response in glioblastoma patients.

Pour cela, ce petit génie en herbe a élaboré une formule chimique composée d’acide salicylique, d’acide glycolique et de trétinoïne, qui favorise l’activation de cellules indispensables au bon fonctionnement du système immunitaire et à la détection d’agents pathogènes potentiels. Il a ensuite ajouté ce mélange à une nanoparticule à base de lipides afin qu’elle ne se dissolve pas avec la mousse du savon et puisse pénétrer la peau.

Investigators from St. Jude Children’s Research Hospital found that proton therapy had efficacy similar to conventional photon therapy with fewer negative neurocognitive outcomes.

Recent research has demonstrated that survivors of childhood cancer are at risk for a myriad of late effects that affect physical and mental quality of life. We discuss the patterns and prevalence of neurocognitive problems commonly experienced by survivors of CNS tumors and acute lymphoblastic leukemia, the two most commonly researched cancer diagnoses.

Despite its higher up-front costs, proton therapy has been shown to be more cost effective than X-ray therapy owing to the dramatic reduction in the excess costs of managing long-term toxicities

Pediatric craniopharyngioma is a rare, benign brain tumor that arises near the pituitary gland and hypothalamus, crucial areas for hormonal regulation and neurological function. Despite being non-cancerous, these tumors can cause significant health issues due to their location, leading to symptoms such as visual disturbances, hormonal imbalances, and cognitive challenges. Typically diagnosed in children aged 5 to 14, craniopharyngiomas require careful management to mitigate their impact on the developing brain and overall health.

This guideline is intended to be a reference document for clinicians presented with the challenge of managing children and young adult patients (CYP) with craniopharyngioma up to the age of 19 years. It addresses the clinical assessment, diagnosis, treatment and follow-up of affected patients, informed by information gathered from peer reviewed scientific reports identified through a robust literature search. The expertise and experience of a range of nationally and internationally respected clinicians and scientists whose opinion has been sought as experts bring a clinically meaningful interpretation to these data, in a clear, pragmatic set of management guidelines.

Child health research is at the core of our commitment to deliver the best clinical care for kids. During the research process, our scientists study the genetic, biologic and cellular mechanisms of disease, injury and repair. The discoveries from these studies are then translated into improved diagnostics and treatments, providing cutting-edge therapies to our patients. Armed with new answers to the problems our patients face, we're constantly adapting our bedside protocols to deliver the best care.

This perspective article aims to summarize recent changes and knowledge gains in the field of pediatric CP, outlining updated treatment recommendations, a concept of integrative interdisciplinary care and the implication of novel potential diagnostic tools. A comprehensive update on the multimodal treatment of pediatric CP is presented, focusing on “function-preserving” therapies and their implications.

We conclude that monitoring of growth and weight development including HC can lead to early CP diagnosis and treatment. This might prevent higher grades of hypothalamic involvement and lead to an improvement of quality of life after CP. Further studies on the specific value of HC as a diagnostic marker are warranted.

A single dose of ROBO1-targeted CAR T cells doubled median survival in cell-line-derived xenograft (CDX) models of recurrent GBM. Moreover, in CDX models of adult lung-to-brain metastases and pediatric relapsed medulloblastoma, ROBO1 CAR T cells eradicated tumors in 50–100% of mice. Our study identifies a promising multi-targetable PTP4A–ROBO1 signaling axis that drives tumorigenicity in recurrent GBM, with potential in other malignant brain tumors.

To discover the pathway cancer cells use to infiltrate the brain, researchers used large-scale gene editing technology to compare gene dependencies in glioblastoma when it was initially diagnosed and after it returned following standard treatments. By doing this, researchers discovered a new pathway used for axonal guidance – a signaling axis that helps establish normal brain architecture – that can become overrun by cancer cells.

To stop the invasion of cancer cells, researchers targeted the hijacked signalling pathway using different strategies including a drug developed in John Lazo's group at the University of Virginia, and also by developing a new therapy with help from Kevin Henry and Martin Rossotti at the National Research Council Canada using CAR T cells to target the pathway in the brain. They honed in on a protein called Roundabout Guidance Receptor 1 (ROBO1) that helps guide certain cells, similar to a GPS.

This study presents the implementation of electrokinetic convection-enhanced delivery (ECED) to infuse macromolecules into the brain from a hydrogel reservoir placed at the surface of the brain. The brain acts as a charged porous scaffold that, in the presence of an electric field, allows for electroosmosis: bulk fluid flow in a porous media. From the hydrogel reservoir, this bulk flow of fluid enables the delivery of therapeutic agents.