PharmaLive reports on a just published article in the Journal of Neurosciencia. Roche scientists, along with researchers from the University of Cantabria and the Cajal Institute, detail studies that used a compound to selectively block specific receptors in the brain of a mouse model with Down syndrome and reverse key neurological defects.
Roche compound(RD1662) is currently being tested in a human clinical trial. This compound “acts on subset of the receptors for the chemical messenger GABA that are present in discrete brain regions associated with cognitive processing where they inhibit communication between nerve cells”, as the article describes. The compound reverses the GABA-A mediated over inhibition that interferes with proper nerve cell signaling.
Research Down Syndrome continues to make grants in this area of research.
Read the full article here.
The article quotes Luca Santarelli, Head of Neuroscience at Roche, “Our drug research in Down syndrome may offer a novel therapeutic avenue to treat the cognitive deficits in people with Down syndrome, enhance their communications skills and ultimately help them have greater independence in their daily lives. The development of this agent is in line with our strategy to discover new medicines that are based on a deep understanding of disease mechanisms and provide options for conditions of high unmet medical need.”
Dr. Santarelli was a recipient of Research Down Syndrome’s “Empower the Extraordinary” award in 2012.