Novel Treatment Gains Accelerated Approval for Relapsed/Refractory Neuroblastoma
Approval was based on results from two phase 2 single-arm, open-label trials that demonstrated overall response rate and duration of response.
CNS Cancers
FDA Grants Orphan Drug Status to Berubicin for Malignant Gliomas
he designation is supported by data from a phase 1 trial conducted by Reata Pharmaceuticals in 2016. Findings from the study showed 44% of patients treated with berubicin (n=11 out of 25) experienced a statistically significant improvement in progression free survival.
Pedmark Receives Priority Review for Prevention of Cisplatin-Induced Ototoxicity
The application is supported by data from 2 pivotal open-label, randomized phase 3 trials (SIOPEL 6 and ACCL0431), which compared the efficacy of sodium thiosulfate plus cisplatin to cisplatin alone for the prevention of cisplatin-induced hearing loss.
Phase 3 Study of Investigative Glioblastoma Tx Halted
AbbVie has halted enrollment in all ongoing studies of depatuxizumab mafodotin, after an Independent Data Monitoring Committee (IDMC) recommended a phase 3 study of the treatment be stopped due to a lack of survival benefit.
Neoadjuvant PD-1 Blockade Seems Effective in Glioblastoma
Neoadjuvant administration of programmed cell death protein 1 (PD-1) blockade seems to enhance local and systemic antitumor immune response in glioblastoma, according to a study published online in Nature Medicine.
Novel Glioblastoma Treatment Granted Orphan Drug Designation
Moleculin Biotech announced that the Food and Drug Administration (FDA) has granted Orphan Drug Designation to WP1066 for the treatment of glioblastoma. WP1066 is an immuno-stimulating STAT3 (signal transducers and activators of transcription) inhibitor being investigated as a potential treatment for brain tumors, pancreatic cancer, and acute myeloid leukemia. In preclinical models, the inhibition of…
Sex Differences ID’d in Response to Glioblastoma Treatment
HealthDay News — Sex differences have been identified in response to therapy among glioblastoma (GBM) patients, according to a study published in the January 2 issue of Science Translational Medicine. Wei Yang, PhD, from the Washington University School of Medicine in St. Louis, and colleagues used a quantitative image-based measure of response to examine the…
NIH Releases Large-Scale Dataset of CT Images
Unlike most lesion medical image datasets currently available, which can detect only 1 type of lesion, DeepLesion has much diversity and contains critical radiology findings from across the body, including lung nodules, liver tumors, and enlarged lymph nodes.
Modified Polio Vaccine Looks Promising in Early Glioblastoma Trial
PVSRIPO is a prototypic oncolytic recombinant polio virus vaccine that recognizes the poliovirus receptor CD155, which is widely expressed in neoplastic cells of solid tumors.
Afinitor Disperz Approved to Treat TSC-Associated Partial-Onset Seizures
The approval was based on the Phase 3, randomized, double-blind, placebo-controlled EXIST-3 (EXamining everolimus In a Study of TSC) study which evaluated adjunctive Afinitor Disperz (low exposure: 3-7ng/mL; high exposure: 9-15ng/mL) in patients with treatment-resistant TSC-associated partial-onset seizures, defined as inadequate control of partial-onset seizures despite use of ≥2 sequential regimens of single or combined anti-epileptic drugs.
