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