BRAIN CANCER

Compared with historic controls, the use of adjuvant VAL-083 after chemoradiotherapy and temozolomide may improve outcomes for select patients with glioblastoma multiforme.

During the past 4 decades, glioblastoma has remained a cancer with one of the worst prognoses and is among the most debilitating.

LP-184 is moving forward in the course of development and was granted an orphan drug designation by the FDA.

For patients with neuroendocrine tumors, the chances of dying of cancer were higher than dying of other causes, but mortality largely varies by primary tumor site.

While brain tumor rates are declining, 5-year survival remains low at 36%. Incidence rates of adult brain tumors are decreasing; however, 5-year survival rates remain low.

The FDA has granted fast track designation to the highly selective inhibitor of casein kinase 2 inhibitor, silmitasertib for the treatment of patients with recurrent sonic hedgehog-driven medulloblastoma.

The immune biology of the neuroblastoma microenvironment is an emerging field.

The benefit of using a drug off-label is that it may provide the best available intervention for a patient with no other option.

Rising interest in SSTR2-targeted radiopharmaceuticals has led to the evaluation of numerous ways to optimize peptide receptor radionuclide therapy in patients with neuroendocrine tumors.

DAY101, an investigational, oral, brain-penetrant, and highly selective type II pan-RAF kinase inhibitor for the treatment of rare, pediatric low-grade glioma, was granted a rare pediatric disease designation by the FDA.

Safety and efficacy data for natural killer T (NKT) cells bolster the use of this cellular therapy in patients with stage IV relapsed/refractory neuroblastoma, according to early findings.

Final overall survival and long-term safety results of the phase 3 NETTER-1 trial were presented during the World Congress on Gastrointestinal Cancer 2021.

The FDA has accepted the filing of a new drug application for surufatinib to be indicated as treatment of patients with advanced neuroendocrine tumors. The FDA set a Prescription Drug User Fee Act target action date of April 30, 2022.

The first patient has been dosed in a study designed to evaluate the safety and feasibility of IL13Rα2 chimeric antigen receptor T cell therapy for the treatment of leptomeningeal brain tumors, such as glioblastoma, ependymoma, and medulloblastoma.

A rolling submission of a new drug application to the FDA has been initiated for surufatinib as a potential treatment option for patients with pancreatic and extra-pancreatic neuroendocrine tumors.

The FDA has granted a rare pediatric disease designation to the novel p-STAT3 inhibitor WP1066 for the treatment of patients with the rare brain and spinal cord malignancy, ependymoma.

The FDA has granted orphan drug designation to CYNK-001, a non-genetically modified cryopreserved human placental hematopoietic stem cell-derived natural killer cell therapy, for the treatment of patients with malignant gliomas.

The FDA has issued a letter to the developer of the immunotherapy vaccine, ERC1671, recommending that the phase 2 clinical trial of ERC1671 in combination with granulocyte-macrophage colony-stimulating factor, and cyclophosphamide for the treatment of glioblastoma be terminated.

The FDA has granted CYNK-001, a natural killer cell therapy, with a fast track designation for the treatment of adult patients with recurrent glioblastoma multiforme, according to a press release from developer Celularity.

The FDA granted orphan drug designation to the CDK2/4/6 inhibitor, NUV-422, for the treatment of patients with malignant gliomas.

Treatment with lanreotide autogel in patients with pancreatic neuroendocrine tumors, and midgut neuroendocrine tumors did not cause deterioration in quality-of-life, according to new data from the Phase II CLARINET FORTE study.

The final patient with glioblastoma multiforme has been enrolled in the ongoing phase 2 clinical trial of VAL-083, which is evaluating the efficacy, safety, and pharmacokinetics of the agent in patients who have been pre-treated with temozolomide prior to disease recurrence.

The investigational, potent, oral CDK9 inhibitor, zotiraciclib in combination with temozolomide, demonstrated early improvement in progression-free survival as treatment of patients with recurrent high-grade gliomas, meeting the primary end point of the phase 1B 17-C-0009 trial clinical trial.

The first patient with newly diagnosed glioblastoma multiforme has been dosed with the first-in-class small molecule enzastaurin in combination with temozolomide and radiotherapy in the phase 3 ENGAGE clinical trial with an aim of determining the overall survival outcome of the drug in this patient population.

The FDA has granted approval to the naxitamab as treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease on prior treatment.

The investigational drug Rhenium NanoLiposome demonstrated efficacy in an interim analysis presented at the 2020 Society for Neuro-Oncology Annual Meeting for patients with recurrent glioblastoma.

Patients with newly-diagnosed, recurrent, and first-line MGMT-unmethylated glioblastoma multiforme exhibited a promising median progression-free survival and median overall survival in two phase 2 trials of VAL-083 compared with historical data.

Positive preliminary data from the phase 1 DUET-1 study examining XmAb18087 in patients with neuroendocrine tumors demonstrated promising safety and efficacy findings.

Results of the CLARINET FORTE study indicate that a dosing strategy of lanreotide given every 14 days may be a viable and safe option when the standard administration of every 28 days is not acceptable.

The FDA has accepted an investigational New Drug application for the investigational CDK2/4/6 inhibitor NUV-422 for the treatment of patients with high-grade gliomas.