Roche Starts Early Stage Clinical Trial In Down Syndrome
First potential therapy designed to improve cognition and adaptive behavior in individuals with Down syndrome
Basel, Switzerland and Nutley, NJ /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.
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.