Possible pharmaceutical therapies to improve learning and memory for people with Down syndrome

To read on Down Syndrome Education International:

DSE to participate in Roche Global Down Syndrome Advocacy Group Advisory Board

DSE is to participate in a meeting of Down syndrome organisations from around the world to discuss recent advances in clinical research and clinical trials of possible pharmaceutical therapies to improve learning and memory for people with Down syndrome.

DSE’s CEO, Frank Buckley, and DSE’s Director of Science and Research, Professor Sue Buckley OBE, are to participate in a meeting of international, national and regional Down syndrome organisations being hosted by Roche, a leading healthcare company that has recently initiated a programme investigating possible pharmaceutical therapies to improve learning and memory for people with Down syndrome. Frank Buckley is representing Down Syndrome Education International and Down Syndrome Education USA. Professor Sue Buckley has been invited as a guest speaker to present on past, present and future Down syndrome research.

The meeting, being hosted at Roche’s headquarters in Basel, Switzerland, aims to present information about recent advances in clinical research and to discuss the potential ethical and legal implications of clinical research with Down syndrome organisations from across Europe and the US.

Last year, Roche began studies of a drug (RG1662) that might help improve learning and memory function for people with Down syndrome. A Phase 1 safety and tolerability study of RG1662 involving adults with Down syndrome began at several centres in the US and the UK. Roche also initiated a molecular and functional imaging study of RG1662 with individuals with Down syndrome and healthy controls and a study to evaluate assessments and neurocognitive tests for the measurement of cognitive changes in clinical trials. Roche expects to complete Phase I studies in 2013.

Frank Buckley, DSE’s CEO, commented, « We are pleased that Roche is interested in exploring therapies that may improve the lives of people with Down syndrome – Roche’s engagement and investment in research in this area is to be welcomed. We are also pleased that Roche is working to engage the global Down syndrome community in communicating their research and in discussing the practical and ethical implications of their research studies. »

Roche’s compound targets specific receptors that are involved in passing signals between nerve cells in specific parts of the brain. It is thought that some of the learning difficulties experienced by people with Down syndrome may result from an over-inhibition of signalling between nerve cells. By counteracting the over-inhibition, it is hoped the drug may lead to improvements in memory function and learning. Roche recently published research investigating a similar compound in mice that carry extra copies of many genes similar to those found on human chromosome 21. The study reported improvements in learning and memory when the mice were treated with the drug.

Frank Buckley notes, « Over the past decade, there have been many promising advances in our understanding of the genetic, biochemical and neurological features of Down syndrome. It is hoped these will lead to therapies that might improve life for people living with the condition. Much work remains and it is not clear how successful it will be. Most of the evidence to date relies on mouse models that may or may not accurately reflect neuropsychological function in people with Down syndrome. Many promising treatments in mice fail to work in humans. The limited trials to date of compounds thought likely to aid learning and memory for people with Down syndrome have been generally disappointing. Properly conducted trials are needed to provide clear evidence of the benefits and safety of these compounds for people with Down syndrome, and these will take time. We welcome Roche’s interest and investments in the rigorous studies that are needed to evaluate these therapies properly. »


Professor Sue Buckley has provided and continues to provide consulting services to F. Hoffman-La Roche Ltd., and receives compensation for these services. Down Syndrome Education International has been engaged to provide advisory services to F. Hoffman-La Roche Ltd., and received compensation for out-of-pocket expenses relating to these services. These disclosures were correct at the time of publication.

Prof Sue Buckley Picture University of Portsmouth

Frank Buckley Picture DSE International

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