Crossing barriers

Developing medicines to treat diseases of the brain is notoriously difficult. One reason, aside from the brain’s complexity, is that it is hard to get medicines into the brain, across the blood-brain barrier.

Roche RNA Hub scientists recognise that it is time for a change. “Some drug development projects in the brain have in the past been ended unsuccessfully at very late stages,” says drug metabolism and pharmacokinetics project leader Erich Koller. “Instead, we want better models to be able to find out early on if something isn’t going to work.”

Roche has been tackling this challenge for many years. Now, with the RNA Hub as a source of unique oligonucleotide cargos for the Brainshuttle™ technology and combined with the expertise from Roche’s deep experience in large molecule protein research, the team is making exciting progress. They are using the technology to deliver new medicines where they are needed to be effective, for example for the delivery of oligonucleotide-based drugs to the brain. 

The Brainshuttle project has been successful so far in part due to the right experts coming in at the right time, explains project team member Felix Schumacher.

Pairing the Brainshuttle™ technology with nucleic acid therapeutics could be an exciting approach to make things easier for patients. For example, instead of needing to have medicine injected into the spinal canal, patients could ideally have a less frequent injection in their arm.

“This is an exciting time for Roche, bringing together two platforms - the Brainshuttle™ technology and nucleic acid therapeutics,” Erich adds.

“Even though moving into a new modality is challenging because there are many moving parts, the possibilities are huge,” Felix says. “We are really excited about our approach. At the Roche RNA Hub we have a unique setup, with all the expertise at our fingertips.”