New partners on fragile X syndrome
A historic gift in the UC Davis MIND Institute’s 20th year aims to help families worldwide living with fragile X and other neurodevelopmental disorders.
By some recent estimates, brain disorders account for more years lost to death and disability than cancer and cardiovascular disease combined. The resulting medical, financial and emotional impacts spur on neuroscientists worldwide as they toil at the coal face of science’s “last frontier” — research on the workings of the brain — in search of new treatments and tools.
Now a historic gift to the UC Davis MIND Institute will help unite researchers from two major nations in their efforts to assist families everywhere who are dealing with a neurodevelopmental disorder that serves as the most common cause of inherited intellectual disability.
The Azrieli Foundation, a foundation in Canada that has helped launch several major neurodevelopmental research initiatives in that country, has announced a $2.3 million donation to the UC Davis MIND Institute. The gift — believed to be the single largest in the MIND Institute’s 20-year history — will help create a large international collaboration designed to leverage expertise and innovation around fragile X-related disorders such as fragile X syndrome. The condition is the most common known single-gene cause of autism.
“Any family that has had to face living with a brain disorder knows what it is like to live like a ‘pioneer’ — stepping into uncharted territory and going off in unknown directions, frequently without the aid of any scientific ‘map’ or treatment options,” said Naomi Azrieli, the foundation’s chair and CEO, and the sibling of a brother with fragile X syndrome. “For these families, the future looks much brighter than it did even just a few years ago, but there is so much more to be done.”
“The UC Davis MIND Institute has a stellar reputation as a collaborative international research center, and we hope to find a treatment together that will improve the quality of life for people with fragile X syndrome.”
The Azrieli Foundation is one of the largest foundations in Canada and Israel and fulfills the philanthropic legacy of David Azrieli, a Holocaust survivor who became a successful architect and real estate developer in both countries before passing away in 2014. The foundation supports initiatives in the fields of education, architecture and design, Holocaust education, developmental disabilities, scientific and medical research, and the arts.
Naomi Azrieli said neuroscience research is an area of extraordinary social impact, noting that about half the Canadian population has had a brain disorder that will impact their family. One in three will also have a disease, disorder or injury of the brain, spinal cord or nervous system at some point, she added.
Besides her brother, some other members of Azrieli’s extended family also have fragile X syndrome. The condition usually comes with a normal life expectancy, but also requires lifelong care and support.
“Through research like that conducted at the MIND Institute, we hope parents and families will have many more tools and treatments to help their children live independent, meaningful and productive lives,” she said.
The new gift will establish a new North American collaborative partnership with research teams from the University of Alberta Women and Children’s Health Research Institute, located in Edmonton, and the University of Montreal CHU Sainte-Justine Research Centre. Both are also receiving support from the foundation.
“Each of our sites has different areas of expertise, and this gift allows us to combine our strengths and experience and create a network that’s more than the sum of its parts,” said Leonard Abbeduto, Ph.D., the MIND Institute’s director and Tsakopoulos-Vismara Endowed Chair of Psychiatry and Behavioral Sciences at UC Davis, who is an investigator in the new collaborative project.
The new network of researchers, along with new infrastructure for collaborative clinical trials, will help the universities jointly conduct the largest controlled trial ever funded from a foundation of a new and potentially promising targeted treatment for fragile X syndrome. Commonly used for diabetes, the drug metformin has also shown proven success rates in fragile X syndrome animal studies and positive effects in human patients during limited observations.
The new study will involve 180 individuals ages six to 25 with fragile X syndrome, and use leading-edge clinical measures developed at the MIND Institute to gauge benefits to learning and functioning.
“If we can demonstrate significant benefit in those with fragile X syndrome, these findings will have an impact worldwide on treatment,” said principal investigator Randi Hagerman, M.D., endowed chair in fragile X research and the MIND Institute’s medical director. Hagerman and her husband, UC Davis physician-scientist Paul Hagerman, M.D., Ph.D., discovered the neurodegenerative fragile X-related disorder FXTAS in 2001.
Foundation support will also help researchers to assess biomarkers related to the findings, in hopes of predicting which patients will respond well to the drug in the future. Research data and samples will be made available to scientists worldwide to inform other genomic studies and assessments of personalized treatment approaches.
“Through the collaborative project, we’ll also be better positioned to quickly test other medications in the future,” Abbeduto said. “In short, the UC Davis MIND Institute will be more nimble when it comes to searching for help for the families who depend on us. We’re extraordinarily grateful for this support.”