Pioneering study starts to treat patients with incurable neurological disease

Maggie Walsh_PSP

Southampton researchers have given the first UK patient a new treatment for Progressive Supranuclear Palsy. This international study aims to see if this incurable condition can be halted in its tracks.

Progressive Supranuclear Palsy (PSP) is a rare, fatal and incurable condition. It causes damage to the brain stem, the part of the brain controlling balance, movement, vision, speech and swallowing.

Usually diagnosed around 60 years of age, life expectancy is six years from that point on average.   Often initially leading to falls and loss of mobility, it develops to restrict eye movement, distort speech and make swallowing food safely impossible.

Giving new hope

Now a British person with PSP has been given a new treatment that aims to disrupt the changes that cause PSP. They’re part of a study led in the UK by Dr Boyd Ghosh, a consultant neurologist at University Hospital Southampton and honorary clinical teacher at the University of Southampton.

If successful, it will provide the first treatment targeting the root cause of the condition.

Blocking tangles

PSP, like several other brain conditions is driven by changes to the tau protein. Tau is essential to the structure and working of our nerve cells. But in PSP this altered tau forms tangles and clumps that disrupt and damage those nerve cells.

Proteins like tau are produced by our cells from our DNA via a molecule called RNA. The new treatment being trialled by Dr Ghosh uses small pieces of RNA to block this. These match sections of the tau RNA, sticking to them and preventing translation of the RNA into protein.

Dr Ghosh and team hope that injecting these ‘antisense oligonucleotides’ into the spinal fluid will slow PSP’s effects.

Treating the first patient

The first patient - Maggie Walsh, 65, from Southampton - was treated at University Hospital Southampton on Tuesday 3 August. She was the first UK patient to receive the treatment which is hoped could prove a ‘gamechanger’ in targeting the root cause of the condition.

Maggie’s husband Bob, 67, told how his wife was diagnosed despite there currently being no diagnostic test. The symptoms can be similar to other neurological diseases like Parkinson’s, but Maggie was struggling with eye movements which helped doctors define she had PSP.

Bob said: “It’s a very debilitating condition and I have watched Maggie deteriorate over time. Often the symptoms are not visible from Maggie’s point of view, so I have to ask her about things as I notice them. She regularly suffers falls, can speak very quietly and now struggles to look down. We rely on a wheelchair sometimes, but I make sure Maggie gets her half an hour of exercise every day when I take her for a walk.

“It’s very difficult to see this happen. That’s why this research is so important to us – it may not be in time to help Maggie but if it can help others in the future then that’s what is important to us.”

Despite huge staffing pressures, the team pulled together to make that happen. Dr Ghosh and the ward Matron both worked back-to-back night and day shifts to ensure continuous monitoring for the patient.

The team is looking to treat another three patients here in Southampton, who will be followed up over the course of a year. It is the first centre to open in the UK, with two more planned.

Since build-up of tau is a feature of many neurological conditions including Alzheimer's disease, the treatment may also be trialled in the future to see if it might help patients affected by other conditions.

“This treatment could be life changing,” said Dr Ghosh. “As of now there’s no cure. These people are facing relentless progression of their disease. This has the potential to give them back more of their life, and more time with their families and friends.”

Posted on Wednesday 22 September 2021