Engineered solution delivers key protein to brains of Parkinson’s trial patients

A study into the treatment of Parkinson’s disease has achieved a degree of success thanks to an engineered solution that delivered a therapeutic protein to participants’ brains.

The study, funded by Parkinson's UK with support from The Cure Parkinson's Trust and in association with the North Bristol NHS Trust and Bristol University, wanted to see if increased levels of GDNF (Glial Cell Line Derived Neurotrophic Factor, a naturally-occurring protein) can regenerate dying brain cells in people with Parkinson’s and reverse their condition.

To get GDNF to the brain cells that need it, a specially designed delivery system was developed. A total of 41 participants underwent robot-assisted surgery to have four tubes placed into their brains, which allowed GDNF to be infused directly to the affected brain areas via a port in the side of their head.

Six took part in the initial pilot study to assess the safety of the treatment approach. A further 35 people then participated in the nine-month double blind trial, where half were randomly assigned to receive monthly infusions of GDNF and the other half placebo infusions. After the initial nine months all participants had the opportunity to receive GDNF for a further nine months.

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