Brain Cancer

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.

Adilia Hormigo, MD, PhD, discusses the current treatment landscape of glioblastoma.

The investigational immunotherapeutic ERC1671 in combination with granulocyte-macrophage colony-stimulating factor, cyclophosphamide, and bevacizumab showed promising activity in patients with recurrent glioblastoma.

David A. Reardon, MD, discusses the design of a phase 1/2 trial with INO-5401 and INO-9012 in glioblastoma.

The FDA has granted a fast track designation to paxalisib for the treatment of patients with glioblastoma.

David A. Reardon, MD, discusses the role of INO-5401 and INO-9012 in glioblastoma.

The biologics license application for the investigational B7-H3–targeting monoclonal antibody omburtamab for use in pediatric patients with central nervous system/leptomeningeal metastases from neuroblastoma has been submitted to the FDA under the agency’s Rolling Review process.

The FDA has granted a fast track designation for the PKCβ inhibitor enzastaurin for the treatment of patients with newly diagnosed glioblastoma.

Efforts are underway to expedite the identification of therapies that offer promise for patients with glioblastoma

A biologics license application has been submitted to the FDA for omburtamab, previously referred to as burtomab, for the treatment of pediatric patients with central nervous system/leptomeningeal metastases from neuroblastoma.

The FDA has granted an orphan drug designation to berubicin for the treatment of patients with malignant gliomas.