On the importance of being translational: Patient-driven therapeutics

17 June 2019
Our second example of the intense exchange of our basic science researchers with the Discovery Sciences team leads us into the field of neurology and peripheral neuropathies. More specifically to Charcot-Marie-Tooth disease (CMT), an incurable progressive weakening and atrophy of distal limb muscles. Patients develop foot deformities and gait difficulties, and also experience sensory abnormalities. CMT has a severe impact on the quality of life of its patients. Ludo Van Den Bosch, who is specialized in CMT, is professor neurobiology at the VIB-KU Leuven Center for Brain & Disease Research. His ultimate goal is to contribute to the development of new therapeutic strategies for neurodegenerative disorders. He partnered with Laurent Galibert of the Discovery Sciences team, who has over 20 years of experience in drug discovery and therapeutic modalities. They are both at the very core of a new ambitious project with the mission to develop innovative therapies for patients suffering from CMT. The project is currently in a proof-of-concept phase and recently external funding was obtained to finance the feasibility study.

What was the basic scientific breakthrough, the spark that set off the collaboration between the VIB-KU Leuven Center for Brain & Disease Research and the VIB Discovery Sciences team?
Ludo: “Our research group focuses on the mechanisms of neuronal degeneration and regeneration. Axonal degeneration of the motor and sensory axons is the main characteristic of CMT. We focus on the pathogenic mechanisms underlying this degeneration, with a particular focus on CMT induced by mutations in HSPB1
and GARS. Our research concentrates on the involvement of cytoskeletal modifications and the​ role of axonal transport defects in the pathogenesis. Histone deacetylase 6 (HDAC6) modifies the components of the tracks needed for axonal transport and selective inhibition of HDAC6 reverses the axonal transport defects, as well as the phenotypes observed in our different disease models.“

“Moreover, our team discovered that HDAC6 inhibition has a broad therapeutic potential both for inherited
peripheral neuropathies, such as CMT, as well as for acquired chemotherapy-induced neuropathies. Thanks
to the Discovery Sciences team, we can further develop this potential.”

Laurent: ”The main question for the VIB Discovery Sciences team is how one can take the scientific breakthroughs of Ludo’s team and translate them into a viable drug discovery project with a competitive edge. We are there to define all parameters for this endeavor, constitute a project team, define the characteristics of the products that will result from this effort and the ways to fund such an endeavor.”

The academia-industry cultural divide is often perceived as being huge. How does the VIB Discovery Sciences team bridge that gap?
Laurent: “Basic science aims at advancing knowledge and is discovery driven. Its final goal is an understanding of the world. The basic rules are those of competition via intellectual insight. The world of the biotech companies on the other hand is application driven and product-centric. In the biotech sector competition is based on innovation. Translational science, the playing field of the Discovery Sciences team, tends to imagine, develop and optimize a product. We have to spot business and valorization potential, take the lead in pre-clinical research, develop drugs and agro-biotech innovations and attract investors. We are walking the bridge both with the academics and the industry to meet in the middle.”

Does a scientist feel more confident when approaching the industry if he or she is assisted in that process?
Ludo: “A researcher is likely to pay more attention to the translation of his fundamental research into products if he is backed by a team offering industrial know-how and business expertise. But the key starting point is always high-quality research and let’s not forget the patients. It is for the benefit of the patients that we want to translate our scientific discoveries into drug candidates. That’s our most powerful motive.”

Laurent Galibert: “We at VIB Discovery Science like to think we are ‘bilingual’, speaking both the academic and industrial language. We have also already walked the path from scientific discovery to industrial innovation several times. This, we hope, provides reassurance to researchers venturing into drug discovery. In addition, we certainly tend to operate in fields with high societal impact that would have little chance of success without our involvement.”

Go back to the overview: 'VIB Discovery Sciences'​​​



Laurent Galibert​

Ludo Van Den Bosch

©VIB-Ine Dehandschutter