Florida Atlantic University researchers have secured two key grants to investigate targeting a gene for the first time as a new approach to treat glioblastoma, a very aggressive and fast-growing type of brain cancer.
Malignant gliomas, such as glioblastoma multiforme and astrocytomas, are the most common type of primary brain tumor in the United States. They make up about 78% of all malignant brain tumors. Although these tumors are not very common, especially in the U.S., they cause a high number of cancer deaths because they are so aggressive.
These awards, granted by the Florida Department of Health's Cancer Connect program for $562,000 and the Palm Health Foundation for $50,000, support a distinctive collaboration among FAU researchers that unites complementary expertise in neuroscience and cancer biology. Together, this partnership aims to advance highly innovative projects that have the potential to make a meaningful impact on cancer therapy.
The grants will explore a promising new target – a gene called MBLAC1 – whose properties encouraged a collaboration between two FAU research labs, one focused on molecular neuroscience and the other on cancer mechanisms and treatment.
MBLAC1 plays a key role in regulating copper levels within cells, which affects mitochondrial function and oxidative stress – both critical factors in cancer cell survival and growth. Since glioblastoma cells rely heavily on mitochondrial energy production and protection against oxidative damage, targeting MBLAC1 could disrupt these processes and slow tumor progression.
Investigators of this grant are Randy D. Blakely, Ph.D., executive director of the FAU Stiles-Nicholson Brain Institute, the David J.S. Nicholson Distinguished Professor in Neuroscience and a professor of biomedical science within FAU's Charles E. Schmidt College of Medicine; and Gregg B. Fields, Ph.D., FAU vice president for research and executive director of the FAU Institute for Human Health and Disease Intervention (I-Health).
By combining our expertise in cancer biology and neuroscience, we are approaching glioblastoma with a fresh and powerful perspective. By identifying how MBLAC1 supports tumor growth and testing drugs that block its function, our team hopes to lay the groundwork for developing new, effective therapies that could improve outcomes for patients with glioblastoma and possibly other cancers."
Gregg B. Fields, Ph.D., FAU vice president for research and executive director of the FAU Institute for Human Health and Disease Intervention
The project will investigate how MBLAC1 influences glioblastoma invasion and copper homeostasis using advanced 3D tumor models and genetically engineered mice lacking the gene.
"I'm excited to bring my background in neuroscience to explore new frontiers in cancer research," said Blakely. "Our work centers on how a specific gene regulates copper – a vital micronutrient – in brain cells, which directly influences how these cells generate energy and manage stress. Because cancer cells depend heavily on energy to grow and spread, uncovering and interrupting this process could transform treatment. This collaboration is a perfect example of how combining different scientific perspectives can spark innovation and accelerate progress in the fight against cancer."
The Blakely lab discovered that MBLAC1, a previously unstudied gene, plays a crucial role in brain cell function by regulating copper, which is essential for cell energy production and protection against oxidative stress. Lower levels of MBLAC1 have been linked to better survival in patients, suggesting it could be a key target for new treatments.
Researchers will investigate whether MBLAC1 from support brain cells or from the cancer cells themselves drives tumor invasion. Using 3D tumor models that closely mimic real tumors, they will observe how blocking MBLAC1 or copper affects tumor growth and spread. They are also developing a new test to quickly identify drugs that specifically inhibit MBLAC1 activity.
Blakely is collaborating on this project with cancer biologists Fields and Ania Knapinska, Ph.D., principal investigator and a research professor at FAU I-Health.
"Our project breaks new ground by uncovering a molecular pathway that actively promotes cancer through a gene linked to copper balance and glioblastoma risk," said Knapinska. "We're also pioneering novel genetic models to study glioblastoma in ways never done before. Our discoveries that mutations in this gene reduce mitochondrial function and increase cellular stress have driven us to explore its fundamental role in copper regulation and metabolism in living systems. Because this gene is highly targetable by drugs, our research opens promising new avenues for developing effective glioblastoma treatments."
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