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FDA Awards Orphan Drug Designation to Gallium Maltolate for Pediatric Glioblastoma Multiforme
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In this last episode of OncChats: Examining LIFU–Aided Liquid Biopsy in Glioblastoma, Manmeet Singh Ahluwalia, MD, MBA, FASCO, and Michael W. McDermott, MD, shed light on the excitement surrounding the use of low-intensity focused ultrasound in cancer and other conditions.

The European Commission has granted an orphan drug designation to Temferon for the treatment of patients with glioma, joining glioblastoma multiforme in its designation.

The addition of the CXCL12 inhibitor NOX-A12 to standard frontline therapy with radiation and bevacizumab led to an overall survival rate of 83% at a median follow-up of 15 months in patients with glioblastoma.

In this fourth episode of OncChats: Examining LIFU–Aided Liquid Biopsy in Glioblastoma, Manmeet Singh Ahluwalia, MD, MBA, FASCO, and Michael W. McDermott, MD, discuss the key objectives of the phase 3 LIMITLESS study (NCT05317858) examining low-intensity focused ultrasound with immunotherapy and chemotherapy in patients with lung cancer and brain metastases.

Varun Monga, MD, discusses the investigation of the protein arginine methyltransferase 5 brain-penetrant inhibitor PRT811 in patients with recurrent high-grade glioma or uveal melanoma.

The FDA has granted an orphan drug designation to the orally bioavailable, reversible small molecule EGFR inhibitor ERAS-801 for the treatment of patients with malignant glioma, including glioblastoma.

In this third episode of OncChats: Examining LIFU–Aided Liquid Biopsy in Glioblastoma, Manmeet Singh Ahluwalia, MD, MBA, FASCO, and Michael W. McDermott, MD, discuss the LIBERATE study (NCT05383872) examining low-intensity focused ultrasound in patients with glioblastoma.

In this second episode of OncChats: Examining LIFU–Aided Liquid Biopsy in Glioblastoma, Manmeet Singh Ahluwalia, MD, MBA, FASCO, and Michael W. McDermott, MD, discuss the success observed with low-intensity focused ultrasound in essential tremors and the hope for this approach in brain cancer.

Timothy I Shaw, PhD, discusses the genetic heterogeneity between paired primary and metastatic solid tumors, as well as the implications for neoantigen-based personalized vaccines in cancer care.

Katherine B. Peters, MD, PhD, discusses findings from the phase 3 INDIGO trial of vorasidenib in patients with recurrent or residual grade 2 glioma harboring an IDH1 or IDH2 mutation and how these results support the addition of vorasidenib to the treatment armamentarium.

In this first episode of OncChats: Examining LIFU–Aided Liquid Biopsy in Glioblastoma, Manmeet Singh Ahluwalia, MD, MBA, FASCO, and Michael W. McDermott, MD, explain how low-intensity focused ultrasound works and the rationale for examining its use in cancer and other conditions.

The protein arginine methyltransferase 5 brain-penetrant inhibitor PRT811 demonstrated preliminary antitumor activity and acceptable safety for patients with recurrent high-grade glioma and advanced or metastatic uveal melanoma, according to data from the dose-expansion portion of a phase 1 trial presented at the 2023 ASCO Annual Meeting.

Treatment with tovorafenib elicited encouraging and fast onset responses in heavily pretreated pediatric patients with low-grade glioma regardless of response assessment criteria, according to data from the phase 2 FIREFLY-1 trial that were presented at the 2023 ASCO Annual Meeting.

Treatment with the IDH1/2 inhibitor vorasidenib reduced the risk of progression or death by 61% compared with placebo for patients with grade 2 IDH-mutant glioma, according to findings from the phase 3, double-blind INDIGO trial.

Experts in the field share the abstracts they found to be the most practice changing at this year’s ASCO Annual meeting.

The gamma-delta T-cell therapies INB-400 and INB-410 have received orphan drug designations from the FDA for use as potential therapeutic options in patients with newly diagnosed glioblastoma.

Bently P. Doonan, MD, discusses the evaluation of targeted inhibition of IRAK-4 with emavusertib in the treatment of metastatic brain melanoma.

The FDA has granted a fast track designation to ERAS-801 for the treatment of adult patients with glioblastoma harboring EGFR gene alterations.

Jezabel Rodriguez-Blanco, PhD, discusses unmet needs for pediatric patients with relapsed brain tumors, including the need for additional treatment approaches for these patients.

The combination of PARP and ATR inhibition with olaparib and ceralasertib was tolerable but showed limited efficacy in pediatric patients with advanced malignancies harboring DNA replication stress and DNA repair deficiencies, according to findings from arm N of the phase 1/2 AcSé-ESMART trial.
Priscilla K. Brastianos, MD, explains the differences between low-grade meningiomas and aggressive high-grade tumors.
Nader Sanai, MD, discusses symptoms associated with high-grade meningiomas, a rare brain tumor.
Priscilla K. Brastianos, MD, reviews recent findings in high-grade meningiomas, including data generated in her laboratory.

At present, there are no FDA-approved systemic therapies for patients with high-grade meningiomas. However, there has been an increase in research surrounding the disease over the past decade, which is improving the prospect a targeted agent may become for this population.

The FDA has granted an orphan drug designation to FORE8394 for the treatment of primary brain and central nervous system malignancies.














































































