CAR T Therapy: Brain Cancer Growth Slowed
- An experimental dual-target CAR T cell therapy is showing promise in slowing tumor growth in glioblastoma (GBM), a particularly aggressive form of brain cancer.
- While long-term survival data is still being gathered, several patients lived for a year or more after receiving the investigational therapy.
- The research, conducted by scientists at the Abramson Cancer center (ACC) of the University of Pennsylvania, builds on earlier findings from a Phase I clinical trial.
early results from an experimental CAR T cell therapy show great promise for those battling aggressive brain cancer. The new dual-target approach led to tumor reduction in nearly two-thirds of patients with recurrent glioblastoma. This innovative treatment, targeting specific proteins, has already helped some patients outlive typical survival rates. Clinical trials are set to launch for newly diagnosed patients soon. news Directory 3 is following this promising development closely.Discover what’s next in the fight against brain cancer.
CAR T-Cell Therapy Slows growth of aggressive Brain Cancer
Updated June 1, 2025
An experimental dual-target CAR T cell therapy is showing promise in slowing tumor growth in glioblastoma (GBM), a particularly aggressive form of brain cancer. The CAR T-cell therapy led to tumor reduction in almost two-thirds of patients, according to findings presented at the 2025 American Society of Clinical Oncology (ASCO) annual meeting and published in Nature Medicine.
While long-term survival data is still being gathered, several patients lived for a year or more after receiving the investigational therapy. This is a notable advancement, considering the typical survival for this patient population is less than 12 months.
The research, conducted by scientists at the Abramson Cancer center (ACC) of the University of Pennsylvania, builds on earlier findings from a Phase I clinical trial. Other researchers across the U.S.have reported similar results.
GBM is the most common and deadly brain cancer in adults. Even with aggressive treatment, the cancer recurs in almost all patients, with a median survival rate of six to 10 months after recurrence.
“Seeing recurrent GBM tumors shrink like this is unusual because the immunotherapy drugs that we’ve tried in the past have been unable to do that,” said Dr. Stephen Bagley, assistant professor of Hematology-Oncology and Neurosurgery, and principal investigator.
“Before the trial,many of these patients had tumors that were growing rapidly,and the treatment changed the trajectory of their disease,which is very meaningful to patients with GBM,” Bagley added.
Dual-Target Approach
CAR T cell therapy is a personalized immunotherapy that uses a patient’s own immune cells to fight cancer.While successful in treating blood cancers, it has struggled to make inroads against solid tumors like brain cancer. the brain cancer treatment developed at penn targets two proteins commonly found in brain tumors: epidermal growth factor receptor (EGFR) and interleukin-13 receptor alpha 2 (IL13Rα2). It is administered via injection into the cerebrospinal fluid.
Dr. Donald M. O’Rourke, director of the Glioblastoma Translational Center of Excellence in the Abramson Cancer Center at Penn Medicine, created the dual-target CAR in his lab and served as a scientific advisor to the trial.
The study involved 18 patients with recurrent GBM who underwent surgery to remove as much of the tumor as possible,followed by infusion of the dual-target CAR T cell therapy into the cerebrospinal fluid.
in 62% of patients with at least 1cm of tumor remaining after surgery, the tumors shrank after CAR T cell therapy. While the tumors regrew after one to three months in most patients, some encouraging results emerged:
- 11% of patients remain alive with stable disease beyond six months.
- 43% of patients with at least 12 months of follow-up were still alive after a year.
- One patient’s cancer has remained stable for more than 16 months, despite advanced disease spread at enrollment.
Researchers also found evidence that the CAR T cell therapy remains in the immune system to prevent tumor growth. In one patient who underwent surgery again due to tumor regrowth, the removed tissue showed T cell infiltration and tumor clearance by immune cells.
Similar signals of immune system stimulation were observed in spinal fluid samples from other patients, including one patient with detectable CAR T cells one year after treatment.
“these results reaffirm that we’re onto somthing with our dual target therapy,and that we have a good template that we can begin refining for even better outcomes,” O’Rourke said.
“Periods of stability, when tumors shrink or don’t grow, vastly improve the quality of a patient’s life. Our goal is to refine the treatment so that more patients experience longer-lasting results.”
The team plans to administer multiple doses of the CAR T cell therapy in the final cohort of the study to see if repeat dosing extends the time before tumors regrow.
What’s next
With safety established, clinical trials for newly diagnosed GBM patients will open soon. Ten of 18 patients experienced manageable grade 3 neurotoxicity, with no unexpected side effects.
“By the time GBM recurs, it has become even more challenging to treat, and the patient has already been through a lot,” Bagley said.
“We’re hopeful that by moving quickly to test this CAR T cell therapy in the newly diagnosed setting, the cancer will be more vulnerable to therapy and more patients will see a benefit.”