Clinical trials for Down syndrome drug at Roche Labs

from PR Newswire

Roche 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

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/

This is wonderful news. Once the pharmeceutical community catches on to the fact that there is more money (or at least as much) in improving the lives of those with Down syndrome than there is in ending them through prenatal testing and abortion, then we might have a true chance to give people with Down syndrome the same chance at a normal life that has been given to many others who have medical conditions.

Its about time!

This GABA drug sounds like what Dr William Mobley was describing in his lecture at Cold Spring Harbor Labs in spring of 2009. He estimated it would take ten years to bring the drug to the market, and now clinical trials are beginning after two years. That's eight years to go.

Read the entire article here.