Brain cancer, particularly aggressive brain tumors such as glioblastoma, remains one of the most difficult cancers to treat because of the protective blood-brain barrier. 3 New therapies for brain cancer are being explored that use the body’s own immune system to target and destroy tumor cells. These new therapies could replace more traditional treatments, such as surgery, radiation, and chemotherapy.
Key Cancer Patient Takeaways
Discover 3 groundbreaking therapies for brain cancer
Understand the role of clinical trials in advancing treatment options
Learn about the benefits and risks associated with new therapies
Explore how new treatments can impact outcomes for glioblastoma patients
Stay informed about emerging treatments and what they mean for patients and caregivers
New Therapy #1 - Brain Bispecific T-Cell Engagers (BRiTEs)
This is a specialized class of immunotherapies designed to cross the blood-brain barrier and direct a patient’s own T-cells to attack aggressive brain tumors.
How It Works
A BRiTE molecule acts as a bridge. One end attaches to a T-cell (an immune cell), while the other end attaches to a specific protein on the cancer cell (such as EGFRvIII found on glioblastoma cells).
This bridge forces the T-cell closer to the cancer cell. The T-cell then activates and kills the tumor while mostly sparing healthy surrounding tissue.
BRiTE treatments are engineered antibodies and can be mass-produced, stored, and ready for use (unlike CAR-T customized treatments).
Currently In Trial
BRiTE treatments are not available for standard treatment and can only be accessed through a clinical trial.
Researchers are leading a Phase I clinical trial for Grade 4 glioblastoma with EGFRvIII-positive tumors.
See HERE for more information.
Potential Side Effects
Cytokine Release Syndrome (CRS): An overactive immune response causing fever, chills, fatigue, or low blood pressure.
Neurological Symptoms: Temporary confusion, headaches, or dizziness as the immune system reacts near the brain.
Low Blood Counts: May experience a temporary drop in white blood cells or platelets, which can raise infection or bleeding risks.
New Therapy #2 - B7-H3 Targeted Treatment With CAR-T
Researchers are testing engineered T-cells that target specific proteins on brain tumor cells. B7-H3 (also known as CD276) is a protein on the surface of the cell that is highly expressed in many types of brain cancer. It helps cancer cells multiply, spread, and grow.
A recent Yale School of Medicine trial on B7-H3, using CAR-T therapy, showed tumors stopped growing or shrank in over half of the participants. This Phase I clinical trial shows that targeting B7-H3 with CAR-T treatment (which helps pass the blood-brain barrier) is safe and offers promising tumor control for recurrent glioblastoma.
CAR-T treatment changes a patient’s own immune cells (T-cells) in a lab so they can find and kill cancer cells. Doctors inject the modified cells straight into the brain tumor through a small tube (catheter).
New Therapy #3 - Oncolytic Virus Therapy
This is an emerging brain cancer treatment that uses genetically modified viruses to target, infect, and destroy brain tumor cells while leaving the surrounding healthy tissue unharmed. These viruses use a process called oncolysis, which causes direct destruction. Viruses enter cancer cells, make copies of themselves, and cause the cells to burst and die.
As the cancer cells break apart, they release specific antigens that signal the patient’s immune system to recognize and attack any remaining tumor cells. This therapy helps turn an unresponsive tumor into an active tumor that is easier for immune cells to find and infiltrate.
Common Viruses Used
Herpes Simplex Virus (HSV-1): Modified strains are engineered to replicate safely in brain tumors.
Adenoviruses: Altered common cold viruses are designed to target specific tumor receptors.
Poliovirus and Measles Virus: Specially weakened strains of these viruses are adapted to target glioma cells.
Challenges
Delivery: The protective blood-brain barrier makes it difficult for these viruses to reach the tumor in high enough concentrations, so direct injection is often required.
Immune Clearance: The body’s immune system may neutralize and clear the virus before it finishes destroying the cancer.
First approved virus therapy in Japan
G47Δ (Delytact): This is the first approved (in Japan) oncolytic virus therapy for brain tumors. It uses a genetically engineered herpes simplex virus type 1 for patients with malignant gliomas (including recurrent glioblastoma).
In the U.S., oncolytic viruses are still experimental and are widely researched in clinical trials.
DNX-2401 (Adenovirus): Evaluated in multicenter trials and is sometimes combined with programmed death-1 (PD-1) immune checkpoint inhibitors.
G207 (Modified Herpes Virus): Tested in pediatric and adult high-grade gliomas to demonstrate safety and immune activation.
PVSRIPO (Poliovirus): A recombination of polio-rhinovirus chimera that is focused on accessing a strong immune response against glioblastoma.
Cytotoxic T Cells: Recent Phase I Findings in research led by Dana-Farber Cancer Institute physicians continue to report promising survival extensions and the infiltration of immune T-cells from engineered herpes virus injections.
Conclusion
Emerging immunotherapies such as BRiTEs, B7-H3 targeted CAR-T therapy, and oncolytic virus therapy are creating new possibilities for treating aggressive brain tumors such as glioblastoma and recurring glioblastomas. Although each approach works differently, they share a common goal of limiting tumor growth by using and strengthening the body’s immune system to recognize and destroy cancer cells while limiting damage to healthy brain tissue.
New treatments are still being studied, and research will be essential to extending survival time in patients with aggressive brain cancer. Continued clinical research is important to determine how these therapies can be improved and eventually become safe and effective therapies. Hope and options continue to spread with these new therapeutics as they become available to patients with brain cancer.