Glioblastoma is a particularly difficult brain tumor to treat. A protective barrier stops many drugs from reaching the blood vessels that supply the central nervous system, including the brain and spinal cord.

Penn Medicine’s new Glioblastoma Translational Center of Excellence in the Abramson Cancer Center focuses on cellular therapies that harness a patient’s immune system to fight cancer. This research could lead to new options for patients.

How We’re Advancing Precision Medicine for Brain Cancer

A precise diagnosis is critical for brain tumor treatment planning and predicting patient outcomes. Researchers are leveraging the power of artificial intelligence to improve imaging interpretation and identification of glioblastoma and other brain cancers.

Glioblastoma patients and their families need groundbreaking treatments and hope. The Glioblastoma Foundation supports research that leads to answers and breakthroughs.

Molecular oncologist Howard McLeod has discovered a genetic mutation that determines how well patients respond to chemotherapy. This discovery could lead to a test predicting whether a drug will work for a particular patient.

In the PRESERVE GBM study, neurosurgeons use NICO’s innovative technology to acquire multiple samples of a single tumor while still inside the operating room. Then, scientists will use gene panel sequencing, RNA sequencing and long non-coding RNA to evaluate these samples for diagnostic differences. This data will enable doctors to understand a tumor’s biology better and identify potential treatment options. [150]

Precision Medicine for Glioblastoma

Glioblastoma (GBM) is the most common form of brain cancer, and it is highly aggressive. It is particularly difficult to treat because it mixes with normal brain tissue and resists chemotherapy. The disease has claimed many lives, including Senators Ted Kennedy and John McCain, astronaut Neil Armstrong, and three-time MLB All-Star Darren Daulton.

While the average life expectancy for a person diagnosed with glioblastoma is 14 to 16 months, some patients survive for 10, 20 and even more years. Younger patients with good health at diagnosis and those with favorable tumor genetics (MGMT promoter methylation or IDH mutations) are more likely to have longer survival times.

Glioblastoma Foundation professionals are studying targeted therapies — drugs targeting specific molecular characteristics – for primary brain tumors to improve outcomes and broaden treatment options. These research efforts require biospecimens that can be collected and analyzed, such as from surgically removed tumor tissue or liquid biopsy.

Precision Medicine for Meningioma

Research continues to develop several precision medicines approaches for meningioma, including targeted therapy targeting specific tumor mutations. Other work explores the best ways to deliver these drugs into the brain, including blood-brain barrier disruption and a technique that sensitizes cancer cells to radiation.

Penn doctors and their colleagues have published a series of groundbreaking clinical studies in this area, including the use of immune checkpoint inhibitors to treat meningioma (Journal of Neurooncology) and Pascal Zinn’s ability to use enabled loupes for more accurate surgical guidance during brain tumor resection (Operative Neurosurgery).

We’re also advancing research into how best to combine molecular treatment with radiotherapy. For example, a study led by UCSF has shown that Tagrisso can cut the risk of brain metastases in a subgroup of patients with Diffuse Intrinsic Pontine Glioma, or DIPG. This approach could help patients live longer and better manage their recurrences. We are supporting several trials that test these approaches in patients with DIPG and other types of brain tumors.

Precision Medicine for Pediatric Gliomas

Brain tumors are rare, but they cause unnecessary death and disability. In 2018, brain cancers accounted for 1.6% of all new cancer cases and 2.5% of all cancer deaths worldwide, according to GLOBOCAN.

Children with glioblastoma or other high-grade gliomas are often treated with chemotherapy and radiation. The goal is to shrink the tumor so it can be removed surgically or by proton beam therapy. Children who survive these treatments face life-prolonging challenges.

A better understanding of the genetics and biology of these tumors is critical to developing safer, more effective treatments.

For example, researchers are working to evaluate the effectiveness of drugs used in other cancers against glioblastoma. They are also looking at combinations of drugs and approaches that address how tumors develop resistance to treatment. One method uses gene therapy to replace or repair the abnormal genes driving a tumor’s growth. The foundation supports research on this and other promising avenues for treating pediatric brain tumors.