STANFORD, Calif. -- A once-a-day, short-term treatment with a drug compound substantially improved learning and memory in mice with Down syndrome symptoms, say researchers at the Stanford University School of Medicine and Lucile Packard Childrens Hospital. Whats more, the gains lasted for months after the treatment was discontinued. The researchers are now considering a clinical trial to test whether the compound has a similar effect in humans with Down syndrome.
"This treatment has remarkable potential," said Craig Garner, PhD, professor of psychiatry and behavioral sciences and co-director of Stanfords Down Syndrome Research Center. "So many other drugs have been tried that had no effect all. Our findings clearly open a new avenue for considering how cognitive dysfunction in individuals with Down syndrome might be treated." The center was created by researchers at Stanford and Packard Childrens in 2003 to rapidly translate research discoveries into useful treatments for people with Down syndrome.
The research, which will be published Feb. 25 in the advance online edition of Nature Neuroscience, was conducted by Fabian Fernandez, a graduate student in Garners laboratory. Fernandez found that affected mice were significantly better able to identify novel objects and navigate a maze tasks that simulate difficulties faced by children and adults with Down syndrome after being fed 17 daily doses of milk containing a compound called pentylenetetrazole, or PTZ. Treated mice performed as well as their wild-type counterparts for up to two months after drug treatment was discontinued.
"Somehow the drug treatment creates a new capacity for learning," said Garner, who cautions that this new ability may decay over longer periods of time as older, drug-experienced neurons are replaced by younger cells.
The researchers believe that the key to the improvement lies in the fact that PTZ blocks the action of an inhibitory neurotransmitter
Contact: Louis Bergeron
Stanford University Medical Center