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Roche Starts Early Stage Clinical Trial in Down Syndrome

* Reuters is not responsible for the content in this press release

 

Fri Sep 9, 2011 9:38am EDT

 

~ First potential therapy designed to improve cognition and adaptive behavior in individuals with Down syndrome ~

PR Newswire

BASEL, Switzerland and NUTLEY, N.J., Sept. 9, 2011

BASEL, Switzerland and NUTLEY, N.J., Sept. 9, 2011 /PRNewswire/ -- Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced the initiation of its first Phase 1 clinical trial to investigate the safety and tolerability of a molecule designed to address the cognitive and behavioral deficits associated with Down syndrome.
"There is currently a large unmet medical need for the treatment of cognitive impairments in individuals suffering from Down syndrome," comments Luca Santarelli, Global Head of Roche Neurosciences Disease Translational Area. "Our strategy at Roche neurosciences is to specifically address these serious conditions that have no approved, effective or safe treatment. This is why we have a strong commitment to neurodevelopmental disorders, including genetic disorders like Down syndrome or Fragile X, as well as autism spectrum disorders."
Enhancing brain functions such as cognition and language in individuals with Down syndrome holds the promise to help these individuals conduct a more independent life. This may result from the improved ability to carry out every day's practical tasks such as finding an apartment, maintaining a job, or having a more fulfilling social life. These improvements can have a significant impact on functioning and quality of life of Down syndrome individuals as well as help reduce the burden for families, caregivers and the society.
"This study will target only adults between 18 and 30 years old, but we believe that an earlier intervention in Down syndrome has the potential for a greater medical impact," says Paulo Fontoura, Head of Translational Medicine in the Roche Neurosciences Disease Translational Area. "While we are still at the early stage, we are confident that our drug's mechanism of action can potentially open the door to further promising investigations in upcoming years."
Based on animal models, an imbalance between excitatory and inhibitory neurotransmission has been proposed among the underlying causes of altered brain function in individuals with Down syndrome. Roche's investigational drug is being assessed for its ability to address this imbalance by targeting the GABAergic* system.
*GABA: gamma-amino butyric acid
About the study
This placebo-controlled study will assess the safety and tolerability of the investigational drug in individuals with Down syndrome. It will recruit up to 33 individuals in one or two countries. For more information on the study, access the Roche Clinical Trials Protocol Registry and Results Database:  http://www.roche-trials.com/
About Down syndrome
Down syndrome is the most common genetic cause of intellectual disability and developmental delay, and affects one in every 700-1000 newborns. Also called Trisomy 21, this condition occurs when an individual has three, rather than two, copies of the 21st chromosome. This additional genetic material causes some impairment of cognitive ability and physical growth, and is often associated with other complications ranging from neurological and cardiac defects to hearing and vision problems. It is now thought that many people with Down syndrome are more likely to develop Alzheimer's disease as they age.
About Roche
Headquartered in Basel, Switzerland, Roche is a leader in research-focused healthcare with combined strengths in pharmaceuticals and diagnostics. Roche is the world's largest biotech company with truly differentiated medicines in oncology, virology, inflammation, metabolism and CNS. Roche is also the world leader in in-vitro diagnostics, tissue-based cancer diagnostics and a pioneer in diabetes management. Roche's personalised healthcare strategy aims at providing medicines and diagnostic tools that enable tangible improvements in the health, quality of life and survival of patients. In 2010, Roche had over 80,000 employees worldwide and invested over 9 billion Swiss francs in R&D. The Group posted sales of 47.5 billion Swiss francs. Genentech, United States, is a wholly owned member of the Roche Group. Roche has a majority stake in Chugai Pharmaceutical, Japan. For more information: www.roche.com.
SOURCE Roche

First animated series with a character with Down Syndrome

  

Target picks up Punky, will give half of royalties to charity
06/09/11
Target Entertainment has picked up rights to Punky, the first animated series with a character with Down Syndrome as the lead character, and will give half of sales royalties to a Down Syndrome charity.

The 20x7mins 2D animated series is produced by Ireland's Monster Animation and written by Andrew Brenner, best known for creating Humf. It follows the everyday adventures of Punky, a six year old who lives with her mum, brother, granny and pet dog.

The show has had strong reviews since debuting on Irish free-to-air broadcaster RTE and Target Entertainment will launch it internationally at MIPCOM Junior. Half of sales royalties will be donated to Down Syndrome Education International, which pioneers inclusion at schools for all children with Down Syndrome.

Target CEO Alison Rayson told TBIvision: "Punky is such an important series for all families with children who have learning difficulties. Punky shows the world that having Down Syndrome is not a negative thing and that children with Down Syndrome can have as fulfilling and regular a life as those without."

Gerard O' Rourke, Punky's says: "Punky is not a Down Syndrome person, but a person with Down Syndrome. If we can pass this message to our next generation through Punky then we have created a very worthwhile TV series".




                 

Punky