15 May 2018

For patients with Down syndrome

Cognitive impairment in Down syndrome can be reduced with medication

Anna Kerman, XX2 century, based on New Scientist: Memory drug trialled in people with Down's syndrome

RG1662, the first drug developed specifically to combat cognitive impairment caused by Down syndrome, is being tested in humans. This was announced to the delegates of the Festival of Neuroscience in London by David Nutt, a former pharmacology adviser to the British government, now cooperating with the pharmaceutical company Roche, the creator of the drug.

RG1662 inhibits the effect of gamma-aminobutyric acid (GABA), a neurotransmitter that suppresses the activity of brain cells. The drug affects a certain group of receptors found mainly in the hippocampus. It is believed that the problems with memorization and learning characteristic of people with Down syndrome are directly related to the work of this group of receptors.

The new work involves 33 adults suffering from Down syndrome. Although the aim of the study is the safety and tolerability of the new drug, scientists also evaluate changes in motor skills, reaction time and memory work. Those receiving the drug in the study are compared with patients who are given a placebo.

The current study is essentially preparatory. The authors of the work plan to select the optimal doses of the drug and this year launch the next work – already more detailed.

In the company's statement Roche claims that RG1662 is able to help people with Down syndrome, because it has a "unique pharmacology that allows you to target GABA hyperactivity mainly in those areas of the brain that are important for thinking, learning and memory."

Luca Santarelli, head of neuroscience at Roche, hopes that the new drug will improve communication abilities in people with Down syndrome and "eventually help them achieve greater independence."

Recently, another group of scientists was studying another drug to combat cognitive impairment in Down syndrome. The study was conducted on model mice. As it turned out, the drug really helped to normalize the work of memory, while causing kidney problems. Due to the latter circumstance, the development of the tool was discontinued. RG1662 has no such side effect.

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