Is growth factor protein the future of Parkinson’s treatment?

A major, multi-year trial of the GDNF protein as a treatment for Parkinson’s disease published its results in February. While the trial did not meet its primary endpoint, other findings were very encouraging. Is there still hope that GDNF could form the basis for an effective Parkinson’s therapy? Abi Millar finds out.

In February 2019, the pioneering GDNF clinical trials programme finally published its full results. A major, multimillion-pound project, which ran between 2012 and 2017, the study investigated whether the GDNF protein might be suitable as a treatment for Parkinson’s disease. While far from clear-cut, the results offer hope to the estimated ten million people worldwide who live with Parkinson’s.

“My outcome was as positive as I could have wished for,” said trial participant Tom Phipps. “I feel the trial bought me some time and has delayed the progress of my condition.”

Although the study was by some measures disappointing – for one thing, it failed to reach its primary endpoint – the findings are extremely nuanced and do offer grounds for positivity. Parkinson’s UK, which co-funded the study, has described it as a ‘resounding success’.

“Even though the treatment didn’t meet the primary endpoint after nine months, there are lots of other aspects to the study that suggest GDNF was doing quite a lot of good work in the brain,” says Professor David Dexter, deputy director of research at Parkinson’s UK.

Read the rest of this article in the June 2019 edition of Pharma Technology Focus