Randi Hagerman receives International Sisley-Lejeune Foundation Award
Randi J. Hagerman, medical director of the UC Davis MIND Institute, has received the prestigious International Sisley-Lejeune Award 2014 from the Paris-based Jérôme Lejeune Foundation, for her groundbreaking work developing targeted treatments for individuals with fragile X syndrome, a leading cause of intellectual disability and the leading single-gene cause of autism spectrum disorder.
The award recognizes significant accomplishments in therapeutic research for Down syndrome or other genetic intellectual disabilities for researchers who have 'contributed to creating knowledge of these pathologies and the discovery of treatments and cures.'
The Jérôme Lejeune Foundation was founded by Jérôme Lejeune, discoverer of the cause of Down syndrome, which in 1958 he renamed trisomy 21, to accurately describe the genetic abnormality. Hagerman received the award in Strasbourg on March 10, in an event before the European Parliament timed to coincide with World Down Syndrome Awareness Day. The award comes with a €30,000, or $33,855 prize, and a lecture.
"I am very positive about new targeted treatments that have the potential to reverse intellectual disability at a variety of ages, including childhood, adolescence and even in adulthood. My work will continue for this very important goal," said Hagerman, distinguished professor of pediatrics and Endowed Chair in Fragile X Research and Treatment.
Hagerman is one of the world's leading physician-scientists investigating fragile X-related disorders, including fragile X syndrome and fragile X-associated tremor/ataxia syndrome, or FXTAS, a condition affecting primarily older men that she discovered with her husband, Paul Hagerman, in 2001. In 1984 she founded the National Fragile X Foundation in the United States.
Hagerman said during her acceptance speech that she is hopeful that her ongoing trials of investigational drugs, such as ganaxolone, mGluR5 antagonists, minocycline, and sertraline, eventually may improve language, attention, socialization and learning in people with fragile X syndrome and other genetic conditions, such as Angelman syndrome. This work, she said "leads the way for treatments that may reverse neurobiological abnormalities in autism and many other neurodevelopmental disorders." These treatments must be coupled with educational programs to ensure that patients may take advantage of their improved cognitive strengths.
Hagerman is director of the MIND Institute's Fragile X Research and Treatment Center. She is the recipient of numerous honors, including the Jarrett Cole Clinical Award from the National Fragile X Foundation for dedicated service to families in the worldwide fragile X community; the Bonfils-Stanton Foundation Award for her pioneering work in fragile X disorders; and the Distinguished Scholarly Public Service Award from UC Davis. In 2008 she received a Lifetime Achievement Award from the National Fragile X Foundation and, in 2014, she received the C. Anderson Aldrich Award from the American Academy of Pediatrics, for her outstanding contributions to the field of child development.