By Darci Slaten –
Alzheimer’s Research Pioneer Awarded $37.5 Million Grant
The moment you meet neuroscientist Roberta Diaz Brinton, you are amazed by her profound sense of purpose, her brilliant mind, her ability to clearly articulate complex ideas and her welcoming demeanor.
Then, as you listen to Brinton describe her groundbreaking Alzheimer’s research, you realize she is a modern-day pioneer – boldly trailblazing a new frontier to find the cure for this debilitating disease.
In 2016, Brinton was appointed the inaugural director of the Center for Innovation in Brain Science (CIBS) at the University of Arizona Health Sciences.
In August 2019, the National Institute on Aging awarded Brinton and her team at CIBS a $37.5 million clinical trial grant to research a potential regenerative therapy for Alzheimer’s.
The award supports the goals of the National Alzheimer’s Project Act, a U.S. law that calls for a stepped-up national effort on research, care and services for Alzheimer’s and related dementias.
The grant is one of the largest that the UArizona Health Sciences has ever received.
“If successful, this study could be the next step in developing the first regenerative therapy to prevent – and potentially cure – Alzheimer’s,” Brinton said.
According to the Alzheimer’s Association, more than 50 million people worldwide, and 5 million Americans, are living with the degenerative brain disease. By 2050, that number is expected to increase to 14 million Americans. In Arizona, more than 200,000 people age 65 and older will be living with the disease by 2025.
Research, Innovation and Impact
Brinton’s study, “Allopregnanolone as Regenerative Therapeutic for Alzheimer’s: Phase 2 Clinical Trial” is funded by the largest grant she has received during her stellar career. And it is indeed stellar: Her groundbreaking Alzheimer’s research has been continuously funded by the National Institutes of Health for more than 20 years.
Brinton is well-equipped to lead this pioneering research project. It is a five-year, national multisite Phase 2 clinical trial. The primary aim is to determine how effective the neuro-steroid allopregnanolone, or allo, is as a treatment.
Although the specific cause of Alzheimer’s is still unknown, researchers do know the disease causes brain cells to die and the connection between the cells to decrease.
Research has established that individuals with healthy brains have higher levels of allo in their brains than those with Alzheimer’s.
Before arriving at UArizona, Brinton had a highly successful career at the University of Southern California. At USC, Brinton and her team discovered that allopregnanolone activates neural stem cells in the brain, generating new nerve cells. This was a stunning discovery, and Brinton still has the image showing the first regenerated brain cells.
After this discovery, the team designed and conducted a Phase 1 clinical trial to evaluate the safety of allo in treating mild cognitive impairment and Alzheimer’s disease. Their results confirmed that allo is safe and well tolerated in the target population, and they determined the correct dosage levels to advance to the Phase 2 clinical trial.
These promising results laid the groundwork for the team to design the next stage of the research – the Phase 2 clinical trial.
Brinton said she is optimistic that allo could be the first regenerative therapy for individuals with early-stage Alzheimer’s who have the genetic risk factor for the disease.
“The most prevalent risk factor for Alzheimer’s is the APOE4 gene,” Brinton said. “Previous research established that allo has the most effect on individuals who have this gene, so we will be recruiting individuals with APOE4 for the Phase 2 study.”
Brinton and her team are working with physicians from Banner Alzheimer’s Institute in Phoenix and Tucson. They plan to start the recruitment process this spring.
“I believe wholeheartedly in the collaborative relationship between researchers and physicians,” said Brinton. “It’s essential when conducting translational research.”
“I am impressed by Dr. Brinton’s comprehensive understanding of what it takes to accelerate research and ensure its public impact – in this case for people impacted by Alzheimer’s,” said Elizabeth “Betsy” Cantwell, senior VP for research and innovation at UArizona. “She is charting a course of excellence in innovation at this university that has the potential for statewide and global impact.”
UArizona President Dr. Robert C. Robbins also had praise for the neuroscientist. “Dr. Brinton is spearheading innovative clinical research that is vital to advance our pursuit of cures for neurodegenerative diseases such as Alzheimer’s,” he said.
“The global challenge of extending the cognitive health span to match our life spans is a strategic priority for the university, and we are primed to be a global leader in this important area – thanks to Dr. Brinton and her team of world-class translational Alzheimer’s researchers.”
Awards, Accolades and Accomplishments
Brinton is an internationally acclaimed neuroscientist and STEM educator and has garnered a host of awards and accolades over her career.
Of note, she was the first woman to receive the 2017 Melvin R. Goodes Prize for Excellence in Alzheimer’s Drug Discovery for her substantial contributions toward developing treatments and cures for Alzheimer’s.
In 2016, she was named Science Educator of the Year by the Society for Neuroscience. Two years earlier, she was named Woman of the Year by Los Angeles magazine for her Alzheimer’s disease research.
Brinton was awarded one of the nation’s highest civilian honors – the Presidential Citizens Medal – presented by President Barack Obama in 2010. That was for her work in promoting careers in science, technology, engineering and mathematics (STEM) among minority students. U.S. News & World Report listed her as one of the Ten Best Minds in 2005.
Brinton has more than 200 articles published in scientific journals and has authored more than 30 book chapters and invited reviews. She has delivered more than 250 presentations worldwide. What’s more, she holds numerous patents and founded two startups.
She also is a proud Wildcat. Brinton earned a bachelor’s degree in psychology and biology, a master’s degree in neuropsychology and a doctorate in neuropharmacology and psychobiology from the UArizona.
A native of New Jersey, Brinton moved to Tucson in her early 20s.
Soon after arriving in Tucson, Brinton explained, she was “divinely fortunate” to land a job at the UArizona as a lab technician in the Department of Pediatrics clinic. While there, she taught interns, residents and medical students how to conduct laboratory procedures and how to interpret lab results.
“I was incredibly fortunate that Dr. George Comerci was the director of the clinic at the time. He and the entire pediatrics team were fabulous. The physicians, nurses and team members were incredibly welcoming,” Brinton recalled. I’m very grateful and conscious to this day how wonderful these people were.”
Through Brinton’s work at the pediatrics clinic, coupled with the encouragement she received from her colleagues, she gained confidence that she had the intellect, fortitude and determination to accomplish anything she set her mind on.
While working full time as the lab technician, Brinton took night classes at the university. Eventually, she was able to use retirement funds she had accrued to attend classes full time.
“I still had to take out loans, but I realized I could do it, even as a slightly older student in my mid-20s,” she said.
Brinton excelled at the university. She completed her bachelor’s degree program in two years, graduating summa cum laude and as a member of the Phi Beta Kappa honors society.
It was as an undergraduate student that Brinton came to realize she was captivated by the brain. “This was at a time when neuroscience was just emerging,” Brinton said.
As her fascination grew, Brinton advanced in her studies. Her master’s studies and research focused on learning and memory function in people with brain damage. The outcomes surprised her.
“While people with brain damage often recovered sensory and motor function, they rarely recovered cognitive learning and memory function,” she said. “I was so intrigued that I changed my course of doctoral research to investigate the molecular mechanisms that allowed the brain to encode new information – that is, learn, remember and retrieve information.”
Brinton worked in UArizona professor Hank Yamamura’s lab, where she successfully mapped a receptor system involved in learning and memory.
“This was an extraordinary experience, and Hank was a wonderful mentor,” she said.
Her time in Yamamura’s lab was foundational for Brinton and impacted her future research in the area of neurodegenerative diseases.
After completing her doctorate, Brinton was a post-doctoral fellow at Rockefeller University’s Laboratory of Neuroendocrinology in New York. She then was an invited scientist at the Weizmann Institute of Science (Israel), the Rudolf Magnus Institute of Neuroscience (the Netherlands), and the Netherlands Institute for Brain Research.
In 1988, she joined the USC Department of Molecular Pharmacology and Toxicology.
When asked about her leadership style, Brinton is clear.
“My style is very mission- and purpose-focused,” she said. “At CIBS, we are here for a purpose that is greater than ourselves. We bring our extraordinary talents, our passion, our commitment to this challenge of curing neurodegenerative diseases. We’re grateful to be the people who can bring these cures to life.”
CIBS reflects that leadership style.
“Every expertise brought by each person is valued and valuable,” she said. “We work very effectively as a team. We are committed to deliver on the covenant we have with the University of Arizona – to deliver on the promise to discover a cure to those who need it today. The patients may not see us, but we come to work every day with them in mind. And that’s what inspires me as a leader.”
“Dr. Brinton is a visionary mentor and leader,” said May Khanna, a CIBS colleague and assistant professor of neuroscience and pharmacology. “She has opened my eyes to new opportunities and helped me recognize my own potential, both as a researcher and a mentor, in pursuing cures for complex neurodegenerative diseases.”
The Center for Innovation in Brain Science
CIBS was launched in 2016 with Brinton returning to Tucson to become its inaugural director.
At the time, Brinton had a distinguished career at USC, and she and her husband, Theodore, were happy living in Southern California. She seriously contemplated the opportunity to return to her alma mater.
“I asked myself the question, ‘What would it take to pull up stakes and move my research enterprise back to the UA?’ ” Brinton said. “After considerable deliberation, the answer to my question was that in the 21st century there is not a single cure for a single neurodegenerative disease.
“Based on this challenge, I created a vision, mission and plan for the Center of Innovation in Brain Science to achieve what has, thus far, alluded big pharma, university-based research and biotech.
“After submitting my plan, I was doubtful that such an ambitious and audacious idea would be considered. However, it’s the University of Arizona – pioneering, bold and committed to tackling grand challenges that have global and personal impact. They accepted my plan and CIBS was created.”
CIBS brings together UArizona researchers and clinicians across the spectrum of neurodegenerative diseases and pairs them with faculty in several specialties: computational systems biology, biomarker design, synthetic chemistry, translational drug development, clinical trial operations and regulatory affairs. Brinton called it an “all-brains-on-deck research environment.”
“At CIBS, we are focused on four age-associated neurodegenerative diseases – Alzheimer’s, Parkinson’s, multiple sclerosis and amyotrophic lateral sclerosis, or ALS,” Brinton said. “Our mission is to bring innovations in brain science of the future to those who need a cure today.”
Under Brinton’s visionary leadership, CIBS has made remarkable progress that includes an impressive portfolio of therapeutics, research awards, transformational educational programs and contributions to the growth of Arizona’s biotech sector.
In addition to the $37.5 million grant, funded projects at CIBS include a $10.3 million grant to study why women are at a greater risk for developing Alzheimer’s disease than men – which she detailed in a 2017 BizTucson article – and a $5.9 million grant to develop precision medicine interventions to prevent Alzheimer’s.
There is also a $1.8 million grant to develop a cross-disciplinary and translationally oriented workforce to discover new drugs for Alzheimer’s and other neurodegenerative diseases, and a $1.3 million NIH grant to train Navajo students in neuroscience programs.
Brinton said she sees Tucson as an entrepreneurial community that supports UArizona’s efforts to bring research to market. Tucson has the critical mass that allows for effective, innovative collaborations.
“We are at the right place at the right time and we are a ‘can do, will do, must do,’ bold, pioneering environment,” she said. “Here at CIBS, we are creating innovations in brain science of the future for those who need a cure today.”