Brain Cancer

The Japan Ministry of Health, Labour, and Welfare has granted conditional and time-limited approval to teserpaturev for the treatment of patients with malignant glioma; this is the first oncolytic virus to receive approval for use in this indication or any primary brain cancer.

The personalized cancer vaccine AV-GBM-1, developed by AIVITA Biomedical Inc., significantly improved progression-free survival over standard of care in patients with newly diagnosed glioblastoma.

Innovative strategies are vital for patients diagnosed with rare cancers, who are frequently at a disadvantage compared with those who have more common malignancies, experts say.

Sara M. Federico, MD, discusses risk categorization in neuroblastoma.

Standard response assessment in neuro-oncology- and immunotherapy RANO-defined progression-free did not correlate with overall survival vs modified RANO-defined PFS, which strongly correlated with OS in patients with recurrent glioblastoma treated with MDNA55 in a phase 2 trial, suggesting that mRANO may be the optimal means of therapeutic response assessment in recurrent glioblastoma.

Stephanie E. Weiss, MD, FASTRO and Eric M. Horwitz, MD, FABS, FASTRO, discuss ways to leverage radiation in the treatment of central nervous system metastases, areas of active investigation, and different available strategies that are improving outcomes for patients with cancer.

Cell-free methylated DNA immunoprecipitation-sequencing uses plasma cell-free methylomes to detect and classify several types of cancer early on, and provides the opportunity to monitor tumors for response to treatment in noninvasive way.

Natural killer T cells that co-express GD2-CAR and interleukin-15 were found to be safe and to demonstrate evidence of in vivo expansion and localization to metastatic sites in patients with stage IV relapsed/refractory neuroblastoma.

Sara M. Federico, MD, discusses the diagnosis and presentation of neuroblastoma, the role of multidisciplinary care, and novel therapies that could significantly affect the treatment paradigm for this malignancy.

Thomas Bachelot, MD, discussed how to approach treatment of patients with HER2-positive breast cancer and CNS metastases and the impact of tucatinib in this population.

The FDA has granted a rare pediatric disease designation to the p-STAT3 inhibitor WP1066 for the treatment of patients with ependymoma, a rare type of tumor that can develop in the brain or the spinal cord.

Dual inhibition of the MAPK pathway using the BRAF and MEK inhibitors dabrafenib and trametinib, respectively, resulted in durable clinical benefit for patients with BRAF V600E mutant low- and high-grade glioma.

The FDA has recommended the early termination of a phase 2 trial examining the cell-based immunotherapy ERC1671 in combination with granulocyte-macrophage colony-stimulating factor, cyclophosphamide, and bevacizumab in patients with glioblastoma.

Because the current standard-of-care modalities have significant limitations, there has been a great interest in pursuing immunotherapeutic strategies for glioblastoma.

Thomas E. Merchant, DO, PhD, discusses the importance of continued care in pediatric patients with craniopharyngioma.

February 5, 2021 - The potent CDK9 inhibitor zotiraciclib, when used in combination was temozolomide, was found to induce clinically meaningful efficacy with a tolerable safety profile in patients with recurrent, high-grade gliomas.

January 7, 2020 - The FDA has approved the request submitted by Moleculin Biotech, Inc. for rare disease designations to be granted their drug candidate WP1066, an agent that appears to directly elicit tumor cell death and rouse the immune system to eliminate tumors.

January 5, 2020 - A new drug application has been submitted to Japan’s Ministry of Health, Labour, and Welfare for the oncolytic virus teserpaturev for use in the treatment of patients with malignant glioma.

December 23, 2020 — Nivolumab in combination with temozolomide and radiation treatment failed to result in a statistically significant improvement in overall survival in patients with newly diagnosed glioblastoma multiforme with MGMT promoter methylation following surgical resection of the tumor.

December 22, 2020 — The FDA has approved an investigational new drug application for berubicin for the treatment of patients with glioblastoma multiforme.

November 18, 2020 - The FDA has granted a fast track designation and an orphan drug designation to the orally bioavailable small molecule tubulin-binding agent PTC596 for potential use in patients with leiomyosarcoma; the agent also received a rare pediatric disease designation and an orphan drug designation for potential use in patients with diffuse intrinsic pontine glioma.

November 17, 2020 - AV-GBM-1, a personalized cancer vaccine, demonstrated an improvement in progression-free survival in patients with newly diagnosed glioblastoma.

Zarnie Lwin, MBBS, FRACP, discusses the design of the phase 2 LEAP-005 trial in previously treated patients with advanced solid tumors.

The FDA has granted an orphan drug designation and rare pediatric disease designation to the bispecific antibody nivatrotamab for the treatment of patients with neuroblastoma.

The FDA has issued a Refusal to File letter regarding the biologics license application for the investigational B7-H3–targeting monoclonal antibody omburtamab for the treatment of pediatric patients with central nervous system/leptomeningeal metastasis from neuroblastoma.