Glioblastoma, the most aggressive and deadly form of brain cancer, has long posed a significant challenge to medical science. With a near 100% recurrence rate and a median survival time of just 15 months post-diagnosis, the need for innovative treatments is urgent. In a promising development, researchers at the National University of Singapore’s Yong Loo Lin School of Medicine (NUS Medicine) are set to begin clinical trials for a groundbreaking gene therapy aimed at treating glioblastoma by late 2025.
Glioblastoma is notorious for its aggressive nature and poor prognosis. Traditional treatments, including surgery, radiation, and chemotherapy, often extend survival by only a few months. The recurrence of the tumor is almost inevitable, making it a relentless adversary. Dr. Yeo Tseng Tsai, a senior consultant at the National University Hospital (NUH), highlights the dire need for new treatments: "New and more effective therapeutic options are urgently needed to combat this disease."
The novel therapy developed by NUS Medicine researchers involves the use of stem cell-based gene therapy. This innovative approach targets the cancer cells directly and activates the body's immune system to fight the tumor. The therapy builds on a similar drug initially used to treat terminal cancers in pets, which showed promising results. Dr. Sarah Ho, a senior research fellow at NUS Medicine, explains, "Interferon beta activates the body’s immune system, allowing the immune cells to attack the tumor so that you get a long-term suppression of the tumor."
The Science Behind the Therapy
The therapy involves injecting modified stem cells carrying cancer-killing genes into the patient. These stem cells naturally gravitate towards the tumor, where they release a chemotherapy drug called Fluorouracil (5FU). This drug is commonly used in chemotherapy but often causes severe side effects. However, in this targeted approach, the 5FU surrounds only the cancer cells, minimizing side effects.
Dr. Ho elaborates on the process: "The modified stem cells act like suicide bombers, loading the toxic 5FU around the tumor to kill it." This method ensures that the chemotherapy drug is concentrated around the tumor, reducing the risk of damage to healthy cells.
Clinical Trials and Future Prospects
The clinical trial, set to begin in late 2025, will focus on patients with recurrent glioblastoma. These patients will undergo another brain surgery to remove as much of the tumor as possible. Following the surgery, three milliliters of modified stem cells will be injected around the tumor cavity through 20 jabs to maximize exposure to the cancer-killing cells. Patients will also take anti-fungal drugs orally to activate the therapy.
Dr. Yeo emphasizes the significance of this trial: "Since it’s a safety trial, it may not be ethical to offer the new treatment for newly diagnosed patients. But for recurrent glioblastoma, there is no standard of care, and so we can do a research trial like this."
The researchers are optimistic about the potential of this therapy to transform glioblastoma from a terminal illness into a chronic disease, significantly extending patients' lives. Dr. Yeo adds, "I’m not so optimistic that we will cure glioblastoma. But maybe we can convert it into a chronic disease. So instead of patients living 18 to 24 months, like they do now, maybe they can live five or 10 more years. That would already be a great achievement."
Challenges and Future Directions
While the therapy shows great promise, there are challenges ahead. The researchers need to ensure a consistent supply of modified stem cells and secure funding for the trials. Dr. Ho and her team have already set up a biotech start-up, AGeM Bio, to scale up their cancer therapies for both animals and humans.
The success of this clinical trial could pave the way for new treatments for other aggressive cancers. As Dr. Ho notes, "All these point towards: You can’t just kill the cells. You also need to activate the immune system. If not, you will forever be trying to kill every single cell in the tumor environment."
The upcoming clinical trial by NUS Medicine represents a beacon of hope for glioblastoma patients. By combining targeted gene therapy with immune system activation, this innovative approach has the potential to significantly improve survival rates and quality of life for those battling this relentless disease.
WhatsApp
Instagram
Threads
