BBC - The Parkinson’s Drug Trial: A Miracle Cure? (2019)

Filmed over six years, 42 people with Parkinson’s take part in a groundbreaking medical trial. Can the results give hope to 10 million Parkinson’s sufferers worldwide?
Episode 1
Filmed over six years, this series follows a group of volunteers with Parkinson’s as they take part in a groundbreaking medical trial testing a drug called GDNF. Forty-two patients undergo complex brain surgery and months of infusions via a port embedded into the side of their skull. Can this clinical trial give hope to the estimated 10 million affected by Parkinson’s globally?

In 2012, doctors from Frenchay and Southmead Hospitals in Bristol recruit volunteers to participate in a £3m trial, ending in 2017, to tackle the world’s second most common neurodegenerative condition. 42 volunteers living with Parkinson’s put their trust in Professor Steven Gill and the trial’s principal investigator, neurologist Dr Alan Whone, along with their team of medical experts.

'I’m giving you my body and I want you to find a cure', says Kay Cotton, 57, from Cornwall, who has had Parkinson’s for ten years. Other volunteers living with Parkinson’s include Bryn Williams, 42, from Glasgow, who says it is the chance to be 'part of a trial that could truly shift medicine', whilst Ron Johnson, 72, from Oxfordshire, simply wants to 'be able to play football with my grandchildren'.

The drug being tested is GDNF – glial-cell derived neurotrophic factor. It is a naturally occurring protein found in our brains, first linked to treating Parkinson’s in 1993. It was discovered in the lab that, at a high dosage, GDNF had a restorative effect on dopamine neurons, the cells affected in Parkinson’s leading to slowness, rigidity and tremors, among other symptoms.

In 2001, Bristol neurosurgeon Professor Steven Gill became the first to successfully deliver a therapeutic dose of the drug in five people with Parkinson’s but, controversially, the drug failed in a subsequent Phase 2 trial in the USA in 2004. This new trial sees a further 42 volunteers undergo extensive brain surgery and then be randomised to receive either GDNF or a placebo.

In this moving first episode, cameras follow Professor Gill performing surgery on all the volunteers to implant the device he has designed that enables delivery of GDNF to a deep part of the brain. The device comprises four catheters placed in the putamen (a part of the brain key in Parkinson’s) that link to a port embedded in the side of the skull through which GDNF can be infused monthly.

The surgery and infusions that follow are medical firsts, offering a whole new way of delivering drugs to the brain. It leads to Pfizer, the world’s biggest pharmaceutical company, investing in GDNF and the Bristol delivery system part-way through the trial.

Tom Isaacs, 44, from Hertfordshire, is one of the trial’s most severely-affected volunteers, diagnosed with Parkinson’s aged 27. Tom says, 'It is with you every single second of every single day'.

After five years of one of the world’s most ambitious medical trials, we find out whether the drug is the breakthrough the world has been waiting for.

Episode 2
In the second episode, cameras continue to follow the lives of the Parkinson’s sufferers who agreed to take part in an invasive clinical trial - testing a drug that could be the first to halt progression, or even reverse, their condition.

The surgery and infusions are medical firsts, offering a whole new way of delivering drugs to the brain. It leads to Pfizer, the world’s biggest pharmaceutical company, investing in GDNF and the Bristol delivery system part-way through the trial.

So far, the volunteers have been split into two groups, with half receiving GDNF (glial-cell derived neurotrophic factor – a naturally occurring protein found in our brains) and half just a placebo. Delivered through a revolutionary port in the side of their heads, no one knows who is receiving the active drug in their monthly infusions.

Assessments show significant improvement in some. But Tom Isaacs, 44, from Hertfordshire, who felt better at the end of episode one, now thinks he is 30% worse than he was before the trial. He hopes, along with others who feel they haven’t improved, that he’s been on the placebo.

There is renewed optimism at the half-way point of the trial as all the volunteers start receiving GDNF. 'It wasn’t considered ethical to put people through brain surgery without ever giving them a chance to have the drug', explains trial leader, Dr Alan Whone.

We see some remarkable improvements. Darren Calder, 51, from Wiltshire, can put on his socks himself in the morning, for the first time in years. In Glasgow, 44-year old Bryn Williams’ tremor completely disappears. In Cornwall, Kay Cotton, 58, feels like she’s been cured. But for Tom Isaacs and his wife Lyndsey, the trial proves a physical and emotional rollercoaster. Tom suffers ongoing complications following further brain surgery to replace his device (including a new titanium port on the other side of his head) but his indomitable spirit continues to inspire
the medical team.

In the summer of 2016, it’s time for the trial’s principal investigator neurologist Dr Alan Whone to reveal the much-anticipated results. Will the trial volunteers’ experiences of the drug as potentially life-changing be borne out by the scientific results? And could this ambitious medical trial finally offer the hope of a cure for Parkinson’s?