dna iconRoche Research Project

Roche Advisory Board
March 2013:   Down syndrome groups from around the world attended a meeting at the Roche research facilities in Basel, Switzerland.  To discuss recent advances in research and the clinical trials of new Roche pharmaceutical therapies that may improve learning and memory for people with Down syndrome.  The Down Syndrome Research Foundation Ltd is a member of this advisory board and is  represented by Peter Elliott, our Research Director.  This meeting was to explore the views of advocacy and support organisations.  Roche, is a leading healthcare company with research underway into pharmaceutical therapies to improve learning and memory for people with Down syndrome.  In 2012  Roche began studies of a drug (RG1662) that may help improve learning and memory.

A Phase 1 safety and tolerability study of RG1662 involving adults with Down syndrome began at several centers in the USAand the UK.  Roche also initiated a molecular and functional imaging study of RG1662 with individuals with Down syndrome and healthy (None Down syndrome ) controls and a study to evaluate assessments and neurocognitive tests for the measurement of cognitive changes in clinical trials. Roche expects to complete Phase 1 studies later in 2013.  The Roche compound targets receptors that are involved in passing signals between nerve cells in the brain. Some of the learning difficulties experienced by people with Down syndrome is believed to be the result of inhibition of signalling within the brain and this results in impaired memory.  This therapy has been shown to improve memory and learning in mice that have been created to have these very similar short term memory problems seen in Down syndrome.  What works for a mouse may not work for humans so complex and very expensive trials are needed.

Safety and tolerability is the first stage in the testing of a new medicine the next stage will be a Phase 2 trial to test the effectiveness of the therapy.  This is not a cure for Down syndrome but it is potential medicine to improve brain function and memory.   We already know this population is very high functioning and only slightly impaired when compared to normal population.   Any improvement in memory would make it possible to learn more easily and then  remember what has been learned. Its not a cure it is a life long therapy.  In animal trials they regressed when the therapy was stopped for about 2 weeks.  

The research discussed on our website is vitally important and you can watch video presentations discussing  these opportunities and the challenges.  Our research is targeting more bnoral health and development based on a better undestanding of trisomy 21 which is the cause of all the problems that result in Down syndorome.   

We are in contact with Roche. You can send us questions to ask their research team.

Roche study information

·         BP25543 - A Study of RG1662 in Individuals With Down Syndrome

·         BP25611 - A Molecular and Functional Brain Imaging Study in Individuals With Down Syndrome and Healthy Controls Following Single Dose RG1662

·         BP25612 - A Non-Drug Study of The Suitability of Neurocognitive Tests And Functioning Scales For The Measurement of Cognitive And Functioning Changes in Individuals With Down Syndrome

·         US NIH Clinical Trials Information

·         UK NHS Clinical Trials information