Grandfather shares hope for rare brain condition
A retired builder says he hopes taking part in a clinical trial at University Hospital Southampton could help find a treatment for others living with his rare neurological condition.
David Booth was diagnosed with Progressive Supranuclear Palsy (PSP) three years ago. It is a rare and incurable condition that affects around 3,000 people in the UK.
He was the first person in the UK to join an international clinical trial. The treatment being tested could potentially slow PSP’s progression.
On this year’s Clinical Trials Day, David shares his experience of taking part in a study at the NIHR Southampton Clinical Research Facility.
David, a retired builder, started to experience falls in late 2022. His family put this down to a recent hip operation.
However, David’s wife, Sarah, noticed that he was also struggling to find the right words to express himself. She felt worried that he may have dementia due to a family history of the disease, but a memory test did not reveal any problems.
David’s family became increasingly concerned as his loss of balance continued. They went to see a neurologist who assessed David’s symptoms and ordered an MRI scan.
At this time, he was also struggling with eye movements – a unique feature of PSP that helps differentiate it from Parkinson’s disease.
David and his family felt shocked when he was diagnosed with PSP in May 2023.
“None of our friends and family had ever heard of it,” he said.
PSP is an atypical parkinsonian disorder. These share many of the same symptoms as Parkinson’s disease.
People with PSP experience problems with balance, movement, vision, speech and swallowing that get worse over time. The disease differs to Parkinson’s in that it is not usually associated with tremor.
Like many other neurological conditions, PSP is caused by changes to a protein called tau that is found in the brain. It builds up, causing damage to the brain’s nerve cells.
The international trial is testing a drug designed to stop tau from harmfully clumping together. It may help reduce or slow disease progression.
Over 200 people with PSP, all aged between 50 and 80, will join the trial. There are sites in the USA, UK and throughout Europe.
People taking part in the trial will take a tablet three times a day for a year. They will be randomly split into two groups, with half receiving the treatment and the other half receiving a placebo.
Dr Boyd Ghosh, a Consultant Neurologist at UHS, is leading Southampton’s involvement in the trial.
He runs one of the largest clinics in the country for people with atypical parkinsonian disorders.
Dr Ghosh said: “For conditions like PSP, where there are currently no treatments to slow progression, clinical research is essential. Patients who choose to take part are helping to create hope for the future and move us closer to finding effective therapies.”
The average life expectancy of people diagnosed with PSP is just seven years.
Treatment focuses on managing symptoms. For example, people who are experiencing problems with balance may be offered physiotherapy. Others may have speech and language therapy to help them with speech or swallowing problems.
After David received his diagnosis, he began researching clinical trials related to PSP.
“He met the diagnosis with so much determination and positivity,” Sarah explained. “It is true to David’s style to not give up after hearing ‘there is nothing we can do’.”
David reached out to Dr Ghosh after reading about the trial online. Dr Ghosh was impressed by David’s determined outlook, and agreed that he could take part in the trial at UHS if he passed the screening checks.
David passed the checks, becoming the first person in the UK to be accepted onto the trial.
“It was a very proud moment for us as a family,” Sarah shared.
Neither David, nor the research team, know if he is receiving the trial drug or a placebo. Despite this, he says he is grateful to be part of research that could provide a much-needed treatment option for people with PSP in the future.
“I see it as nothing ventured, nothing gained,” he said. “There’s no cure for this disease, but to be part of finding something that could slow its progression – even if not for me – would be fantastic.”