Gene therapy offers hope for those with paralysis
pharmafile | June 15, 2018 | News story | Medical Communications, Research and Development | biotech, drugs, pharma, pharmaceutical
Researchers at King’s College London (KCL) have made significant progress to being able to offer a treatment to those with paralysis due to spinal injuries.
The treatment is still in the early stages of testing, after the gene therapy was administered to rats, but offered significant hope as it saw the subjects able to use their front paws again.
The gene therapy works by delivering a gene that promotes the production of an enzyme called chondroitinase directly into the spine.
This particular enzyme is adept at breaking down scar tissue in the body and was chosen because often, when there is an injury to the spine, scar tissue builds up and obstructs connection between nerve fibres responsible for signalling to lower limbs.
Researchers matched the injury most common in humans, at the neck, in the rats and after a period of two months, the rats were found to regain the use of their front paws.
The team had designed the therapy so that it the gene’s signalling could be effectively switched ‘on or off’ through the use of antibiotics. This allowed researchers to switch off the activity of the gene once the desired results had been achieved.
Dr Emily Burnside from the Institute of Psychiatry, Psychology & Neuroscience, at KCL, explains: “Rats and humans use a similar sequence of coordinated movements when reaching and grasping for objects. We found that when the gene therapy was switched on for two months the rats were able to accurately reach and grasp sugar pellets. We also found a dramatic increase in activity in the spinal cord of the rats, suggesting that new connections had been made in the networks of nerve cells.”
The therapy was also found to be fast-acting, after one or two weeks the rats were able to grip the rungs of a ladder.
However, as mentioned by Burnside, to reach and grasp requires far more coordination and complexity, therefore a greater period of time was required to rebuild the connections required to perform the task.
The next stage of research is to test the therapy in rats that have injuries that one month old, with greater amounts of scar tissue.
Ben Hargreaves
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