Brain Tumor Research News & Updates

Research Progress


Accelerating glioblastoma therapeutics via venture philanthropy
March 30, 2021
Drug Discovery Today
[free full-text/open-access]

National Brain Tumor Society staff and board members teamed up with internationally-renown financial expert and MIT professor, Dr. Andrew Lo, on a new paper published in Drug Discovery Today that demonstrates the promise of leveraging venture philanthropy (or a “megafund”) to stimulate and fund more drug development for glioblastoma.

Zika Virus helps kill deadly brain cancer in mice
March 29, 2021

The Zika virus can activate immune cells to destroy glioblastoma, an aggressive brain cancer, a new study with mice shows.

Watch: Microrobots fight brain tumors in mice
March 24, 2021

Scientists have created a microrobot that can pass the near-impenetrable blood-brain barrier to treat brain tumors in mice, they report in a new study in Science Robotics.

A vaccine targeting mutant IDH1 in newly diagnosed glioma
March 24, 2021
[free full-text/open-access]

Data from a small phase I clinical trial in Europe were recently published in the journal Nature. Thirty-three patients with newly diagnosed glioma with mutations to their IDH1 gene were treated with a vaccine-based immunotherapy targeting these mutations in a trial called NOA16. NOA16 met its primary endpoints by demonstrating the safety and immunogenicity of the IDH1 vaccine in patients. Vaccine-induced immune responses were observed in 93.3% of patients.

The transcriptional landscape of Shh medulloblastoma
March 19, 2021
Nature Communications [free full-text/open-access]

A large team of researchers from multiple institutions published a study observing differences among molecular subtypes of sonic hedgehog medulloblastoma (Shh-MB) and highlight the key roles of specific pathways in the pathogenesis of Shh-MB and open up opportunities for therapeutic intervention.

Non-IDH1-R132H IDH1/2 mutations are associated with increased DNA methylation and improved survival in astrocytomas, compared to IDH1-R132H mutations
March 19, 2021
Acta Neuropathologica
[free full-text/open-access]

A large team of researchers from multiple institutions published a study observing that non-R132H IDH1/2-mutated astrocytomas have a more favorable prognosis than their IDH1R132H mutated counterpart, indicating that not all IDH-mutations in gliomas are identical. This difference is clinically relevant and should be taken into account for patient prognostication.

N-cadherin upregulation mediates adaptive radioresistance in glioblastoma
March 15, 2021
Journal of Clinical Investigation
[free full-text/open-access]

Researchers at the University of Alabama at Birmingham performed studies in animal models and human and mouse cells, which indicate that an adhesive cell surface protein known as N-cadherin — or N-cad — may be key in overcoming glioblastoma’s resistance to radiation therapy.

Novel gene-silencing RNA drug shows promise in glioblastoma
March 10, 2021
Fierce Biotech

Scientists at Northwestern University have developed a drug —dubbed NU-0129 and consisting of a gold nanoparticle core densely packed with small interfering RNA (siRNA) — that targets the cancer-promoting Bcl2L12 gene in glioblastoma tumors. A small “phase 0” clinical trial, published in Science Translational Medicine, showed it could penetrate the blood-brain barrier, suggesting that the experimental drug, also known as a spherical nucleic acid (SNA) drug, should undergo further testing to verify its safety and therapeutic effect.

Bionaut Labs launches with plans to attack brain tumors with tiny, guided robots
March 3, 2021
Fierce Biotech

A company is developing tiny, remote-controlled devices, built to travel through into the brain and deliver a dose of medicine where it’s needed the most: at the site of the tumor.

Inhibitory CD161 receptor identified in glioma-infiltrating T cells by single-cell analysis
February 15, 2021
[free abstract, subscription required for full article]

The authors used single-cell sequencing techniques to create an atlas of T cells in gliomas and highlighted CD161 and other NK cell receptors as immunotherapy targets.

PRMT5 inhibition disrupts splicing and stemness in glioblastoma
February 12, 2021
Nature Communications
[free full-text/open-access]

The authors report on a new rationale for developing brain-penetrant PRMT5 inhibitors as a treatment for glioblastoma.

With ‘rapid’ progress of pediatric brain cancer treatment, Day One sees broad excitement in new crossover round
February 10, 2021

This article discusses progress being made by a company developed specifically to make new treatments for pediatric brain cancer.

H3.3-K27M drives neural stem cell-specific gliomagenesis in a human iPSC-derived model
February 4, 2021
Cancer Cell
[free abstract, subscription required for full article]

NBTS-funded researchers studied the effects of H3K27M, a frequent pediatric glioma driver mutation, in different DIPG-relevant cell types and found the mutation upregulated certain developmental genes, producing diverse outcomes in different stem and progenitor cell types.

Small-molecule screen reveals synergy of cell cycle checkpoint kinase inhibitors with DNA-damaging chemotherapies in medulloblastoma
January 20, 2021
Science Translational Medicine
[free abstract, subscription required for full article]

To identify better treatments for high-risk medulloblastoma, the authors screened more than 3000 compounds using six different human cell lines and showed that inhibitors of cell cycle checkpoint kinases (CHK1/2) increased could be used to increase the efficacy of standard chemotherapeutics for treating these tumors.

NBTS Brain Tumors Clinical Trials Report: Q4 2020
January 14, 2021

The latest quarterly report from the National Brain Tumor Society providing a summary of the clinical trials that have recently opened or started enrolling for primary brain tumor patients in the United States.

Very low mutation burden is a feature of inflamed recurrent glioblastomas responsive to cancer immunotherapy
January 13, 2021
Nature Communications
[free full-text/open-access]

This study reports that very low tumor mutation burden is associated with longer survival after recombinant polio virotherapy or after immune checkpoint blockade in recurrent glioblastoma patients.

Pten Deficiency Leads To Proteasome Addiction, A Novel Vulnerability In Glioblastoma
January 11, 2021
[free abstract, subscription required for full article]

This NBTS-funded study found that proteasome inhibition is a potential synthetic lethal therapeutic strategy for GBM with proteasome addiction due to a high protein synthesis rate and autophagy deficiency.

New Treatment Arms Opening for Patients in NBTS-Funded GBM AGILE Clinical Trial
January 7, 2021

The revolutionary GBM AGILE adaptive clinical trial platform is opening two new treatment arms, with one evaluating Kintara Therapeutics’s chemotherapy, VAL-083, and Kazia Therapeutics’s targeted therapy, paxalisib.

Glioblastoma Linked with Brain Healing after Trauma
January 5, 2021
Genetic Engineering and Biotechnology News (GEN)

Studies by researchers in Canada suggest that the healing process that follows a brain injury—such as trauma, infection or stroke—could spur the growth of glioblastoma tumors, when new cells generated to replace those lost to the injury are derailed by mutations.


4 Big Takeaways From the 2020 Annual Meeting of the Society for Neuro-Oncology
December 19, 2020

National Brain Tumor Society staff distill the key takeaways from the 2020 Annual Meeting of the Society for Neuro-Oncology that have the potential to shape the future of the brain tumor research and treatment landscape for years to come.

Inhibition of 2-hydroxyglutarate elicits metabolic reprogramming and mutant IDH1 glioma immunity in mice
December 17, 2021
Journal of Clinical Investigation
[free full-text]

This work focuses on glioma subtypes harboring mIDH1, TP53, and ATRX inactivation. The authors combined D-2HG inhibition/IR/TMZ with anti–PDL1 immune checkpoint, and demonstrated that metabolic reprogramming elicits anti–mIDH1 glioma immunity, leading to increased MS and immunological memory.

Histone H3.3G34-Mutant Interneuron Progenitors Co-opt PDGFRA for Gliomagenesis
December 10, 2020
[free abstract, subscription required for full article]

This study found that an estimated 50% of gliomas with H3.3G34 mutations also carry activating PDGFRA mutations, which may be more potent and targetable drivers of tumor growth than G34R/V.