One of Spinal Research’s key objectives is to identify the quickest and most efficient route to clinical trials. A crucial step in this process has been the approval of a very exciting programme of work to develop the enzyme chondroitinase through to ‘first in man’ studies.
Chondroitinase is a remarkable bacterial enzyme that has the ability to remove inhibitory chemicals which block nerve regrowth. It also promotes recovery by encouraging nerve cell survival and new growth sprouting which enables intact nerve endings to take on the role of the damaged nerve cells. Results from a project that we fund at the University of Cambridge, among other sites, have been so strong that our Trustees have committed a further £150,000 to continue to develop the enzyme through to clinical trials.
We plan two approaches to development: one by injecting the enzyme directly to the injury site, and the second by using gene therapy for delivery. Gene therapy has demonstrated the most effective results thus far, but it carries a greater number of uncertainties which we need to address in advance of approval by regulatory authorities. We believe this parallel approach is therefore prudent and provides the greatest opportunity for success.
1) Direct delivery – we are now ready to develop a pharmaceutical method that will allow us to produce chondroitinase to clinic standards and begin formal preclinical safety and toxicology studies. This is crucial if we are going to start testing chondroitinase with patients as safety is of paramount importance.
2) Gene therapy strategy
– this approach, delivering chondroitinase through a gene delivery viral vector (virus), will allow cells in the central nervous system to stably secrete chondroitinase at the injury site for a significant period of time, with a single injection.
Additional Information can be found HERE: Spinal Research Connections Autumn 2013