In 2007, Alliance for Cancer Gene Therapy (ACGT) awarded a multi-year research grant to E. Antonio Chiocca, MD, PhD (Brigham and Women’s Hospital), to develop an oncolytic virus therapy for the difficult-to-treat brain cancer glioblastoma.
ACGT is awarding a second grant to Dr. Chiocca to help him expand his learnings from his first research grant to develop an additional oncolytic virus to treat this brain tumor.
Why glioblastoma is difficult to treat.
The National Brain Tumor Society estimates that 15,000 people in the U.S. each year are diagnosed with glioblastoma, and the average survival is 8 months. It’s the most common primary brain tumor, has one of the poorest survival rates of any type of cancer, and is one of the toughest to treat.
Glioblastoma is a “whole-brain tumor”, Dr. Chiocca explained in an interview with ACGT, meaning it spreads throughout the brain rather than staying in just one part of the organ, which makes surgery difficult.
Glioblastoma cells also have many different clones and mutations, which limits the effectiveness of immunotherapies that target one or two mutations. Brain tumors are also protected by the blood-brain barrier, which defends the organ from bacteria but also blocks chemotherapy or immunotherapy in the patient’s bloodstream.
“We know a lot about glioblastoma tumors. The more we know, the more we realize how difficult they are to treat.” — ACGT Research Fellow E. Antonio Chiocca, MD, PhD
How oncolytic viruses fight glioblastoma.
Dr. Chiocca’s approach involves injecting an oncolytic virus into the brain to activate the immune system against glioblastoma. Oncolytic viruses are modified versions of viruses engineered to infect cancer cells while ignoring healthy cells.
After infecting cancer cells, the virus causes the cells to break apart and die. This activates the immune system to send T cells to the area.
When the cancer cells break apart, they release hidden cancerous proteins, which the T cells can use to identify and destroy other glioblastoma cells.
“Once you inject the virus, you get an amazing amount of T cells and other immune cells migrating to the tumor – much more than with other immunotherapies,” Dr. Chiocca said. “We think this is a way to stimulate the immune system to begin attacking the tumor.”
How ACGT funding is supporting Dr. Chiocca.
Funding from ACGT will help Dr. Chiocca perform studies on mice to show the new oncolytic virus is safe and can be manufactured for human patients. These studies – which are poorly funded and expensive to run – are crucial to receiving approval from the U.S. Food and Drug Administration to begin a clinical trial to test the new therapy in patients.
“We make findings in our laboratories and try to bring those findings to humans in the form of therapies. The process of getting from the lab to the patient is very complex. It’s process-development, such as showing that the amount of a virus you used in mice can be scaled up and manufactured for humans. It is showing that the virus is safe. There is a gap in funding for this research, and ACGT is filling that gap. Funding from ACGT is so critical and will allow us to bring the therapy to patients more quickly.” — ACGT Research Fellow E. Antonio Chiocca, MD, PhD
ACGT is committed to supporting scientists who are trying to develop cures for brain tumors through the promising-yet-underfunded science of cell and gene therapy. In 2023, the foundation awarded three grants to scientists using cell and gene therapies for brain tumors: Dr. Chiocca; Hideho Okada, MD, PhD (University of California, San Francisco); and Juan Fueyo, MD (University of Texas MD Anderson Cancer Center).
Dr. Chiocca’s prior research, funded by ACGT, led to a phase 1 clinical trial, the results of which were recently published in Nature.
ACGT continues to fund his critical research to advance further oncolytic virus therapies and trials for people with glioblastoma.
“I use the analogy of the beginnings of the U.S. space program,” Dr. Chiocca said. “President John F. Kennedy said in a great speech that we’re going to the moon not because it’s easy but because it’s hard. ACGT is funding research for brain tumors not because it’s easy but because it’s hard.”
ACGT’s mission is to fund innovative scientists and biotechnology companies working to harness the power of cell and gene therapy to transform how cancer is treated and to drive momentum toward a cure. Since 2001, the foundation has invested more than $37 million and awarded grants to 67 research fellows working to uncover cures for cancer.
Page sources
- About Glioblastoma. National Brain Tumor Society. Retrieved from: https://braintumor.org/events/glioblastoma-awareness-day/about-glioblastoma/. Accessed: 01/24/2024.
