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Much like a scorpion uses toxin components to target and kill its prey, the research team at City of Hope are using it to direct T cells to target the cancerous cells.

Each year as the ground begins to thaw, people across America celebrate National Brain Cancer Awareness Month. While all types of brain cancers are in the spotlight during the month of May, glioblastoma multiforme (referred to simply as GBM) is often at the forefront of everyone’s mind. With only a 5% five-year survival rate, GBM is the deadliest type of cancer. It is a fast-acting disease that warrants little time for trials and research to be conducted. As a result, GBM survival rates have not improved in any meaningful way over the last 30 years. In a recent study, however, researchers have identified the first chimeric antigen receptor (CAR) T cell therapy that can target brain tumor cells. The newly identified piece to the puzzle? Scorpion venom.

Much like a scorpion uses toxin components to target and kill its prey, the research team at City of Hope are using it to direct T cells to target the cancerous cells. Using a component of scorpion venom called chlorotoxin (CLTX), these researchers were able to target cancerous cells without affecting nontumor cells in the brain or in other organs. The team successfully demonstrated that CLTX-directed CAR T cells were highly effective at selectively killing human GBM cells in cell-based assays and in animal models without off-tumor targeting and toxicity. After great success in the laboratory, the City of Hope research team is excited to proceed with the first-in-human clinical trial using the CLTX-CAR T cells.

While the CLTX-CAR T cells are a major step in GBM treatment, there are also many other promising trials underway. The National Foundation for Cancer Research is a founding member of the world’s first global adaptive clinical trial platform for GBM. This revolutionary global collaboration is called GBM Adaptive Global Innovative Learning Environment, or GBM AGILE. 

GBM AGILE changes the model of traditional clinical trials by evaluating multiple therapies simultaneously, ultimately creating a flexible and adaptable trial approach. This new approach allows researchers to identify drugs that are showing promising results and seamlessly transition to a confirmatory stage designed to support drug approval. Similarly, researchers are able to pinpoint and cull the use of underperforming drugs with minimal time and resources being wasted. Simply put, GBM AGILE is patient-centric and provides a streamlined method for researchers to utilize data connectivity within the trial to answer many questions concurrently.

National Foundation for Cancer Research fellow Dr. Rakesh K Jain is also paving the way in which GBM is treated. Dr. Jain is a leader in the field of tumor biology, specifically in anti-angiogenic therapy, which looks at thwarting certain types of blood vessel formation. His seminal research demonstrated that anti-angiogenic therapy works by normalizing the abnormal, leaky blood vessels that usually surround and penetrate tumors. This therapy improves the delivery of chemotherapy drugs, increases the oxygen content of cancer cells and makes radiation treatments more effective. Dr. Jain has specifically been studying the role angiogenesis plays in GBM. His findings help doctors tailor the use of anti-angiogenic therapies by identifying the characteristics that cause resistance to anti-angiogenic therapy for GBM patients. Dr. Jain and his team have identified molecular resistance pathways that may direct the development of new drugs that target these pathways and could extend the benefits of anti-angiogenic therapies for patients.

GBM has been a fierce enemy of the medical field for decades. The National Foundation for Cancer Research along with many organizations across the world have tirelessly searched for answers as to how best to improve treatment for this dreadful disease. After years of research and dedication, new findings and exciting initiatives are emerging to effectively combat GBM. To help researchers combat GBM, please visit the NFCR website.