New Center Accelerates Research on Neurodegenerative Diseases
Though scientists have known for decades that ultrasound waves can be used for medical purposes, recent technological advances have brought ultrasound to the forefront of medical innovation. Clinicians, engineers and researchers are now using MRI to guide focused ultrasound waves in the treatment of various brain disorders — including Alzheimer's and Parkinson's disease, essential tremor, neuropathic pain and even brain cancer.
Florida Atlantic is part of this medical breakthrough, entering a new phase of its long-standing collaboration with Insightec, a pioneer in focused ultrasound. The two institutions have signed a Memorandum of Understanding to advance academic research in the area. Insightec will provide the ultrasound technology; Florida Atlantic will provide the MRI and clinical research.
"Ultrasound has the potential to be a game changer. We're incredibly excited to be partnering with Insightec," said Gregg Fields, Ph.D., vice president for research, who will oversee clinical research for the Florida Atlantic/Insightec collaboration. Fields is the principal investigator on a State of Florida grant that applies magnetic resonance-guided ultrasound to treat Alzheimer's disease patients and develops approaches to monitor the effectiveness of ultrasound treatments using blood drawn from patients.
A burgeoning scientific understanding of the changes in the brain that drive neurodegenerative disorders — Alzheimer's, Parkinson's, and others like them — suggests that the field is entering a new era, one that could bring new ways of interfering with their progression and one day eliminating them at their origins.
The University's new David and Lynn Nicholson Center for Neurodegenerative Disease Research at Florida Atlantic's Stiles-Nicholson Brain Institute is poised to help usher in these advancements. Established with a $2 million gift from philanthropist and wealth manager David J. S. Nicholson, the center brings together researchers and resources from across the University in a coordinated attempt to promote innovative research and education.
"We need new methods to diagnose patients earlier in the course of disease and new therapeutics to interfere with the mechanisms that drive it," says Corinne Lasmézas, DVM, Ph.D., the center's inaugural director. "I believe we — the field and FAU — are ready to develop them."
Much of this work is already well underway, according to Randy D. Blakely, Ph.D., executive director of the Brain Institute. He notes that faculty members across the University's campuses, colleges and departments are already working on these conditions and related topics. Moreover, a significant number of doctoral trainees in the recently initiated Neuroscience Graduate Program are planning to pursue careers in neurodegenerative disease research, a welcome sign to the center's newly recruited scientists.
"We have built up gained considerable momentum in this area in just a few years," Blakely said. "This is why we need a center: To bring it all together and make the whole more than the sum of its parts."
In Alzheimer's, Parkinson's, Huntington's, amyotrophic lateral sclerosis (ALS) and scores of other neurodegenerative diseases, neurons – the message-sending cells in the nervous system – become damaged, stop functioning or die. Their loss leads to deterioration in the faculties we otherwise take for granted, such as moving smoothly or maintaining a posture or a train of thought. Over time, these changes grow more debilitating, eventually becoming fatal.
"Fifteen years ago, many researchers would have told you, ‘we have no idea what causes these diseases,'" said Lasmézas, who is also a professor in the Department of Clinical Neurosciences in the Charles E. Schmidt College of Medicine and has joint appointments with the Brain Institute and the Institute for Human Health and Disease Intervention. "We're now at a point where the scientific community has a lead on the mechanisms that lead brain cells to die."
One significant advancement came when researchers realized that neurodegenerative disorders share a common, underlying biology: They are driven by malformed proteins that form clumps. These clumps, or aggregates, damage neurons and propagate from cell to cell.
Earlier in her career, Lasmézas' research focused on a rare variety of rapidly fatal, degenerative, neurological diseases: those caused by infectious proteins known as prions. In these conditions, which include Creutzfeldt-Jakob disease and mad cow disease, prions form these toxic aggregates. The scope of her research gradually
expanded to the more common variety of neurodegenerative diseases as studies showed the prion-like behavior in the proteins responsible for them, such as alpha-synuclein in Parkinson's, SOD1 or TDP-43 in ALS, and amyloid-beta and tau in Alzheimer's.
Since then, research in her lab, which she moved to Florida Atlantic from the Herbert Wertheim UF Scripps Institute for Biomedical Innovation and Technology, uncovered new types of toxic protein clumps and found that these impair the brain's ability to produce energy, contributing to neuron's death in a handful of these conditions. The team also identified compounds that could interfere in this process. Lasmézas has founded a biotech company, Vova Ida Therapeutics, to develop therapies based on this research.
"The Center for Neurodegenerative Research will help us and our colleagues to bridge the gap to the clinic," Lasmézas said.
The center, which will officially celebrate its inauguration in March 2025, was funded as part of $10 million Nicholson gave to the University to fund neuroscience research and education initiatives. This gift also supported construction of the 60,000-square-foot Stiles-Nicholson Brain Institute research facility on the John D. MacArthur Campus in Jupiter, where the center's offices are now located.
Although headquartered here, the center will have connections across the university and its disciplines. Some of its faculty members, including Lasmézas and Blakely, explore fundamental biological questions. Others have complementary expertise such as clinical research, pharmacology, advanced imaging, chemistry, engineering, and biostatistics. The center's membership will include faculty from the Charles E. College of Science, the Charles E. College of Engineering and Computer Science, the College of Medicine and the Christine E. Lynn College of Nursing, which cares for patients with Alzheimer's and other memory disorders, through the memory clinic and the Louis and Anne Green Memory and Wellness Center. Lasmézas also anticipates involving the College of Social Work and Criminal Justice.
The center's creation capitalizes on work already underway, according to Blakely. Biochemistry and chemistry faculty have been conducting numerous studies related to neurodegenerative disorders, for example. Likewise, in the College of Medicine, philanthropic gifts from Ann and John Wood and the Carl Angus DeSantis Foundation are supporting research and improvements to care for patients.
Through Nicholson's gift, Florida Atlantic has recruited two additional faculty: Srinivasa (Srini) Subramaniam, Ph.D., who studies Huntington's disease, and Alzheimer's disease researcher Qi Zhang, Ph.D. In addition, the gift has provided pilot grants to faculty interested in pursuing related research and the purchase of new equipment, which will allow researchers to analyze individual cells' gene expression from within whole tissue.
"The Center for Neurodegenerative Disease Research will wave the flag broadly across our campuses for this kind of research and draw investments that stimulate further research and education in this area," Blakely said.
As the center gets off the ground, Lasmézas has received funding for an initiative building off her lab's work on how clumps of abnormal protein impair brain cells' ability to produce energy. So far, these studies have been conducted in cells and animal models. Now, with help from Florida Atlantic colleagues, she plans to look for similar deficits in the brains of patients with Alzheimer's, with the ultimate goal of aiding drug development.
The researchers plan to recruit patients through the Memory and Wellness Center and examine them with magnetic resonance imaging at Florida Atlantic's Clinical Research Unit. Other faculty in the College of Medicine and College of Nursing are also part of the project.
Because this disruption to energy production appears common in neurodegeneration, the teams' findings could be applicable not just to Alzheimer's but to other conditions as well. The same is likely true for other avenues of research related to protein misfolding or neuro-inflammation, also common features of these diseases, according to Lasmézas.
"My personal vision is that, by better understanding these diseases and the mechanisms they share, we can tackle several of them at the same time," she said.