As Director of Research for ISRT, Dr. Bacon examines the role of nonprofits in advancing medical research.
Gene therapy breakthrough
Chondroitinase has great promise for spinal cord repair because of the way it digests scar proteins that prevent damaged spinal cord nerves from re-growing. But Spinal Research knows that certain obstacles need to be overcome before clinical trials can proceed. Elizabeth Bradbury and her team at King’s College have been trying to address a potential problem in administering Chondroitinase to patients in the future. Repeated invasive doses to the spinal cord to aid repair would be needed, undermining its usefulness. It was decided instead to try using gene therapy.
Injecting a gene therapy (the chondroitinase ChABC gene packaged in a viral vector which enables it to enter spinal cord cells) into the spinal cords of spinally injured rats produced ground-breaking results, which were published this Spring in The Journal of Neuroscience. Just one single gene injection led to spinal cord cells producing their own chondroitinase in large quantities spanning the entire injury area. The health of the damaged spinal cord was maintained and led to long term recovery of hind limb function.
“It looks like we have taken a big step forward with these results,” said Dr. Mark Bacon, for Spinal Research. “Of course there needs to be further confirming research done, but it accelerates the development of testable therapies for spinal cord repair.” “This is a very exciting time in spinal cord research,” said Elizabeth Bradbury, whose team works as part of the multi-site Chondroitinase consortium. “These results are very effective – we have never seen anything work as well as this. The next stage is to do some safety and efficacy work that can eventually lead to clinical trials.” This research was supported by the UK Medical Research Council, Spinal Research and The Henry Smith Charity.
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