Roche initiates its first clinical trial for Down Syndrome

BASEL, Switzerland and NUTLEY, NJ: Roche has announced the commencement of its first Phase 1 clinical trial to evaluate the safety and tolerability of a drug candidate that has been developed to treat cognitive and behavioral discrepancies related to Down syndrome.

Global Head of Roche Neurosciences Disease Translational Area, Luca Santarelli commented, “There is currently a large unmet medical need for the treatment of cognitive impairments in individuals suffering from Down syndrome. 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.”

Individuals with Down syndrome can be helped to lead a more independent life by improving brain functions such as cognition and language. This might be accomplished be enhancing their capability to perform routine practical tasks such as looking for an apartment, sustaining a job, or having a more fulfilling social life. These advances can prove to have a considerable effect on the functioning and quality of life of individuals with Down syndrome, which would further decrease burden for families, caregivers, and society.

Head of Translational Medicine in the Roche Neurosciences Disease Translational Area, Paulo Fontoura said, “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. 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.”
Through animal models, an imbalance between excitatory and inhibitory neurotransmission has been proposed as one of the fundamental causes for brain function alterations in Down’s syndrome individuals. Roche’s experimental drug is designed to target the GABAergic* system and thereby investigate its capacity to tackle this imbalance.