Brain cancer treatment is entering an era of innovation with several promising clinical trials and novel therapeutic approaches that could reshape the treatment landscape. Leading experts from USC Norris Comprehensive Cancer Center are highlighting significant developments in targeted therapies, immunotherapy combinations, and unique drug delivery methods.
Advances in Brain Metastases Treatment
The management of brain metastases has evolved significantly with the emergence of targeted systemic therapies. Dr. Elizabeth Ren Zhang-Velten, a radiation oncologist at Keck Medicine of USC, points to improved outcomes with drugs like osimertinib, which effectively penetrates the blood-brain barrier. The ongoing NRG-NB012 trial is investigating the optimal timing of stereotactic radiotherapy in relation to surgery for patients with active brain metastases.
"We're exploring whether preoperative stereotactic radiation for brain metastases might lower the risk of leptomeningeal disease dissemination during surgery," explains Dr. Zhang-Velten. "The concept is to potentially sterilize these diseases rather than using postoperative radiation as a cleanup measure."
Innovative Drug Delivery Systems
A groundbreaking approach to brain tumor treatment involves the use of aerosolized medicine. NEO100, a perillyl alcohol formulation, is being investigated in two significant clinical trials. This nasally-administered treatment has shown promising results in early studies, particularly in reaching tumors through the blood-brain barrier.
Dr. Frances Chow, a neuro-oncologist at USC, notes that previous phase 1 studies in glioblastoma patients demonstrated the treatment's ability to cross the blood-brain barrier and affect tumor cells. Current trials are expanding to include both grade III or IV IDH-mutant glioma and meningioma patients, addressing a critical need for effective treatments in these areas.
Personalized Immunotherapy Approaches
The field is also witnessing advances in personalized treatment strategies. A notable phase 2b trial is evaluating an individualized vaccine treatment that utilizes patients' tumor tissue to create targeted immunotherapy. This innovative approach involves a two-step process: creating a vaccine from the patient's tumor tissue and then enhancing the immune response through activated immune cell reinfusion.
"It's a very multidisciplinary and collaborative process," Dr. Chow emphasizes. "We're working with neurosurgeons, pathologists, and radiation oncologists to coordinate treatment and offer the best care possible to patients."
Combining Treatment Modalities
Researchers are increasingly focusing on combination approaches, particularly in immunotherapy. While single-agent checkpoint inhibitors have shown limited success in brain tumors, combinations with radiation or surgery are being explored to enhance therapeutic efficacy.
The field recently celebrated the FDA approval of vorasidenib for low-grade IDH-mutant gliomas in August 2024, marking the first new therapy approval for gliomas in nearly 15 years. This development has reinvigorated hope for more targeted treatments that can effectively penetrate the blood-brain barrier while minimizing toxicity to healthy tissue.
