First drug to help Down syndrome people now being tested

Michael Cook
20 Apr 2013
Reproduced with Permission

The first drug to help people with Down syndrome overcome cognitive deficits is being tested on humans, the Swiss pharmaceutical giant Roche has announced.

Roche has set up a clinical trial with 33 adults to see whether a drug called RG1662 can reverse the effects of a neurotransmitter called GABA which inhibits brain activity. This seems to be the mechanism which is responsible for the memory and learning problems in people with Down's.

This small trial is testing the safety of the drug, but researchers are also examining how it affects the patients' motor skills, reaction time and memory. The drug has already had good results in mice, according to a paper in the Journal of Neuroscience.

"Our drug research in Down syndrome may offer a novel therapeutic avenue to treat the cognitive deficits in people with Down syndrome, enhance their communications skills and ultimately help them have greater independence in their daily lives," said Luca Santarelli, of Roche.

Down syndrome affects around one in 650 to 1,000 live births. Worldwide some 30,000 babies are born with Down syndrome each year. People with Down syndrome have a wide range of abilities but the majority have cognitive deficits which can lead to challenges with independence in their daily activities, education and employment. At present, there are no treatment options that address the cognitive impairments associated with Down syndrome.