A multimillion-pound study lead by the University of Bristol has shown that a novel experimental drug could offer hope for restoring damaged brain cells in Parkinson’s patients.
The treatment, MedGenesis Therapeutix' Glial Cell Line Derived Neurotrophic Factor (GDNF), involves robot-assisted surgery to have tubes placed into the brain to deliver GDNF, a naturally-occurring protein, leading to some “extremely promising” effects on damaged neurons of those who received the treatment.
The clinical trial was not able to prove that the treatment slowed or halted the neurodegenerative disease, however the researchers said the therapy warranted further investigation even though it failed to demonstrate improvement of symptoms in patients who received it when compared to others given a placebo.
Some results, however, showed that damaged cells throughout the putamen were regenerated, reversing the brain damage by roughly six years.
Parkinson’s is a neurodegenerative disease that affects around 1-2% of people over the age of 65. It causes tremors, muscle stiffness and movement and balance problems. Although some medicines can improve symptoms, there is no current cure or treatment that can slow progression of the disease.
Pfizer had previously agreed to fund the drugs’ future development, in return for a worldwide licence to distribution.
Professor Steven Gill, the designer of the device and a neuroscientist at Southmead Hospital in Bristol, told the media: “Even at a low dose we have seen evidence of patient improvement, which is incredibly encouraging. Now we need to move towards a definitive clinical trial using higher doses and this work urgently needs funding. I believe that this approach could be the first neuro-restorative treatment for people living with Parkinson’s which is, of course, an extremely exciting prospect.”
"While the results are not clear-cut, the study has still been a resounding success," added Dr Arthur Roach, director of research at Parkinson's UK. "It has advanced our understanding of the potential effects of GDNF on damaged brain cells, shown that delivering a therapy in this way is feasible and that it is possible to deliver drugs with precision to the brain."
The study was funded by Parkinson's UK with support from The Cure Parkinson's Trust and in partnership with the North Bristol NHS Trust.