New Potential Therapeutic Target for Treatment

A newly published study funded in part by DSRTF shows that an FDA-approved asthma drug already in use improves cognitive function in a mouse model of Down syndrome, providing evidence in support of a new therapeutic target, the beta-2 andregenic receptors in the hippocampus.

Stanford's Dr. Ahmad Salehi

Stanford’s Dr. Ahmad Salehi

The drug, a bronchodilator called formoterol, was shown in the study to strengthen nerve connections in the hippocampus, a brain center used for spatial navigation, paying attention and forming new memories, the study said. It also improved contextual learning, in which the brain integrates spatial and sensory information.  A high dosage of the drug was administered over a period no longer than two weeks, resulting in notable improvements in the mice’s neuronal structures. “The fact that such a short period of giving medication can make these neurons much more complex is very interesting,” said Stanford University’s Ahmad Salehi, MD, Ph.D., the study’s lead author and a DSRTF grant recipient.

Formoterol

Formoterol
Image via Wikimedia Commons under a GNU Free Documentation License.

Salehi called the study, published July 2 in Biological Psychiatry, an “initial proof-of-concept,” cautioning that the dose he and his colleagues used was higher than that used in asthma treatment and cannot be safely adminstered to human patients without the risk of significant side effects.  Nonetheless, these results hold great promise for new classes of drugs to target this receptor, with the goal of improving cognition in people with Down syndrome.

DSRTF is continuing support for this exciting research, and will provide updates on its progress.