Purdue University researchers are developing and validating a patent-pending treatment for the incurable glioblastoma brain tumor. Glioblastoma is almost always fatal, with a median survival time of 14 months. Traditional methods used to treat other cancers, such as chemotherapy and immunotherapy, are often ineffective against glioblastoma.
A research team led by Sandro Matosevic, associate professor of industrial and molecular pharmacy at Purdue University School of Pharmacy, is developing a novel immunotherapy to combat glioblastoma. Matosevic is a faculty member at the Purdue Cancer Institute and the Purdue Drug Discovery Institute.
The research, led by Matosevic, has been published in a peer-reviewed journal Nature communication.
Glioblastoma Treatment at Purdue University
Traditional cell therapies are almost always autologous, meaning they are taken from the same patient and returned to the same patient, Matosevic said. The patient’s blood cells are modified to better recognize and bind to cancer cell proteins and are then returned to the same patient to bind to and attack the cancer cells. Unfortunately, these therapies have limited or no efficacy against glioblastoma.
“In contrast, we are developing immunotherapies based on novel, genetically engineered, completely off-the-shelf or allogeneic immune cells. Allogeneic cells do not come from the same patient, but from another source,” Matosevic explains. “In our research, we created – or rather engineered – cells from derived pluripotent stem cells. So, we eliminated the need for blood and instead differentiated the stem cells into immune cells or natural killer cells and then genetically modified them.”
Matosevic says the new Purdue immunotherapy can be considered a true off-the-shelf source.
“We can imagine an unlimited supply of these stem cells that could be used for engineering,” Matosevic said, “that would not require a blood source. And because these are human cells, they can be used directly in human patients.”
Validation and next development steps
The team tested its therapeutic effect in animal studies on mice with human brain tumors, which were treated with direct injections of the newly engineered immune cells.
“Our preclinical studies have shown that these immune cells can specifically target and completely eradicate tumor growth,” said Matosevic, “We found that we can develop these cells at appropriate doses for clinical use in humans. This is one of the reasons why it is so important. While major barriers to the translation of cell-based therapies into human clinical settings are poor proliferation and the lack of ability to obtain cells directly from patients, fully synthetic methods break down important barriers to generating these cells.”
The next step in glioblastoma treatment development is to conduct clinical trials to treat patients with brain tumors that cannot be successfully removed with surgery, Matosevic said.
“Our ultimate goal is to bring this therapy to brain tumor patients,” Matosevic said. “These patients urgently need better, more effective treatment options. We believe this therapy has real potential and we have the motivation and ability to advance it into the clinic.”
“We are working with neurosurgery clinicians to not only secure funding but also to initiate clinical protocols,” he added. “We are also open to anyone interested in supporting our goal of transforming this therapy into clinics where it is most needed and are always looking for new collaborations and partnerships.”
Matosevic disclosed this innovative glioblastoma treatment to Purdue University’s Office of Technology Commercialization Innovation, which filed a patent application with the US Patent and Trademark Office to protect intellectual property.
Matosevic and the research team received funding from the National Institutes of Health, the V Foundation for Cancer Research, the Purdue University Cancer Institute, and industry partners.
