F. Hoffmann-La Roche

Title

MicroRNA biology in neurodevelopmental disorders and pediatric epilepsies

Engagement type

Fully-funded industry project in SFI FutureNeuro Centre, RCSI

Partner

Roche is the world's largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology, and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based.

Challenge

Epilepsy is the most prevalent serious neurological condition in childhood, affecting up to 1% of children. Despite the wide range of treatments available today, 30% of people with epilepsy continue to have seizures. As a result, there is a major need for new therapies that will target seizures in a different way to existing drugs. The opportunities presented by a better understanding of epilepsy at a genetic and molecular level offer great hope for the future, especially for those with rare and severe forms of treatment resistant epilepsy.

RCSI value-added

Prof. David Henshall and his FutureNeuro research group have made pioneering contributions on the role of microRNAs in epilepsy. MicroRNAs offer the unique advantage of gene network-targeting capability to achieve disease modification in epilepsy; targeting multiple pathways as opposed to current epilepsy drugs which act on a single receptor or channel. The partnership builds on recent breakthroughs at RCSI in understanding how gene activity is controlled in the brain. Through this collaboration, the team looked for unusual gene expression patterns in brain tissue and cell models of childhood epilepsies, focusing on the genome’s so-called ‘dark matter’ – stretches of DNA which do not code for proteins but work as molecular switches to activate or inhibit protein production. This large scale, multi-annual collaboration between Roche and RCSI has resulted in new patent filings, commercialisation agreements and facilitated access, for both parties, to resources, technologies and expertise that would otherwise not have been available.

“The research collaboration with Prof. David Henshall at RCSI has identified valuable links between the neurodevelopmental disorders Dravet Syndrome, Angelman Syndrome and Rett syndrome and the regulation of miRNAs. The work has further improved the biological understanding of the role of miRNAs in pediatric epilepsia, and potential future treatments using antagomirs.”

– Dr Jesper Worm, Principle Scientist, Roche Innovation