Down Syndrome research

[Guest guest]

FYI

Dear friend,

I am pleased to announce that today marks the achievement of an important

milestone in Down syndrome research. Earlier today, the pharmaceutical

company Roche announced that it is going to enroll participants in clinical

trials to test the safety and tolerability of a drug intended to address the

cognitive and behavioral deficits associated with Down syndrome. The full

text of Roche's press release is below as well as on the DSRTF homepage .

We plan to provide additional information regarding the trial on our web

site as well.

DSRTF is extremely proud to have been a supporter and collaborator in these

efforts. The announcement of clinical trials should be welcome news to the

Down syndrome community, where there had been concern that no major

pharmaceutical company would be interested in developing a new drug with the

primary objective to improve cognition in individuals with Down syndrome.

While this initial phase of trials is limited in number of participants it

will enroll (33 participants total), it lays the groundwork for additional

trials in the future, and should provide ample inspiration to other

researchers working in the field. If you live in one of the trial areas,

please consider enrolling. To find out more about the trials and the trial

sites, visit http://www.rochetrials.com/ .

This achievement belongs to all of us. You can feel confident that your

support of DSRTF has had a direct impact on bringing us to today's

developments. My hope is that this news inspires you with hope for a better

future for your loved ones.

Sincerely, Rose Executive Director

RTF

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Media Release

Basel, 09 September 2011

Roche starts early stage clinical trial in Down syndrome

First potential therapy designed to improve cognition and adaptive behavior

in individuals with Down syndrome

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 o Fontoura, Head of Translational

Medicine in the Roche Neurosciences Disease Translational Area. " While we

are still at the early stage, but 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. The investigational drug has

already been tested in healthy volunteers and demonstrated a good safety and

tolerability profile, without significant adverse events. For more

information on the study, access the Roche ClinicalTrials 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 21 st 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 .

Down Syndrome Research and Treatment Foundation

755 Page Mill Road, Suite A-200, Palo Alto, CA 94304

www.DSRTF.org | Phone: 888.731.5557 | Fax: 650.617.1601 dsrtf@...