An American Brain Foundation grant funds research into a specific enzyme in the brain that could unlock more effective multiple sclerosis treatment
Multiple sclerosis is a disease of the central nervous system that affects nearly 1 million people in the United States. People with multiple sclerosis experience inflammation in the brain and spinal cord. Such inflammation can strip nerves of their protective coating (called myelin). Doctors call these stripped areas lesions.
When the nerve is missing its protective coating, it disrupts communication between the brain and other parts of the body. This disrupted communication is what causes the symptoms of multiple sclerosis, such as blurred vision or limb weakness.
Studies have shown that targeting a specific enzyme called Bruton’s tyrosine kinase (BTK) can reduce these lesions in the brain. However, exactly how the enzyme does that is not well understood.
Farinaz Safavi, MD, PhD, a Neuroimmunology Fellow at the National Institutes of Health, is looking to change that. Her research aims to understand how BTK induces inflammation to better target treatments for multiple sclerosis and other similar diseases. Dr. Safavi received a 2020 Next Generation Research Grant for her work, which is funded by the American Brain Foundation and the National Multiple Sclerosis Society.
What is the Research About?
Dr. Safavi’s work uses cutting-edge technology to isolate and study specific brain cells to determine how the enzyme causes inflammation. “Studying specific human cells, especially brain cells is not easy, so we use different techniques to differentiate those cells in the lab and then deeply study them,” she says.
In order to pinpoint the exact process, Dr. Safavi is studying what happens before, during and after inflammation. “In order to better understand and treat different diseases, we need to know more detail about the process and about the mechanism underlying every step,” she says.
The overall goals of the research are to identify and define how BTK causes inflammation. From there, Dr. Safavi wants to know how that can be used to create better treatments. A multiple sclerosis treatment that inhibits BTK is already showing a promising effect in early phase clinical trials.
How Does this Research Impact Other Brain Diseases?
Dr. Safavi’s research has impacts beyond treatment for multiple sclerosis. “When researchers investigate the root causes of neuroinflammatory disease or any kind of disease, we basically develop and produce new scientific facts,” she says. “And specifically when we work on a specific gene or protein or molecule that expresses in different cell types, we are producing a new set of scientific information that can be helpful for any other diseases related to those cell types.”
Over time, patients with diseases like multiple sclerosis can start developing neurodegeneration, which is also present in diseases like Alzheimer’s disease and Parkinson’s disease. By better understanding the process of inflammation, researchers can define different targets for preventing irreversible damage to the brain. For example, Dr. Safavi says, we know that inhibiting BTK can reduce inflammation. But we don’t know much about how it reacts with other molecules. “So in that situation, when we know more, we may be able to target those specific proteins or genes to better treat the disease or better control the inflammation,” she says.
BTK expresses itself in specific kinds of cells found in the central nervous system. There’s a growing body of evidence that shows those cells also contribute to neurodegeneration in Alzheimer’s disease.
A multiple sclerosis treatment targeting BTK would focus on reducing inflammation. But the fact that BTK can cause inflammation could be helpful for other conditions. For example, in a patient with a brain tumor, a treatment could activate the same cells to attack the tumor cells.
Why Is Brain Research Important?
For Dr. Safavi, studying the brain is essential because the cells that make up the brain are very sophisticated and differentiated. This makes them hard to repair and heal.
She says she’s thankful to have received a grant from the American Brain Foundation because it allows her to use cutting-edge technology and learn skills that will be helpful in her future career. “At the same time, it can help me to develop a better translational scientific research project, which is helpful to treat the diseases, and it builds a huge scientific foundation for me,” Dr. Safavi says.
The American Brain Foundation’s Next Generation Research Grant program recruits the best and brightest researchers, like Dr. Safavi, to work on early diagnoses, treatments and cures for brain disease and further advance the field. This program is possible thanks to our unique partnership with the American Academy of Neurology, which allows us to tap into the largest brain trust of neurologists and brain disease researchers in the world to support research projects.
The American Brain Foundation was founded to bring researchers and donors together in the fight against brain disease. Next Generation Research Grants fund the innovative research of early-career investigators, encouraging passion for research and laying the groundwork for future success. Learn more about brain disease or make a gift to support groundbreaking brain disease research.