Find out more about the work that has been made possible.
Seeking new treatments for Alpers’ syndrome
Alpers’ syndrome is a rare inherited disease that usually begins in early childhood and has devastating symptoms – including seizures, dementia, and liver failure. Sadly, there is no cure and children’s lives are cut tragically short.
While liver failure may develop before seizures start in some children, commonly used epilepsy medications may also trigger it. This further limits safe and effective treatment options.
Professor Bobby McFarland and his team at Newcastle University are searching for safer drugs that can effectively control seizures, as well as help slow disease progression in the liver and brain. His laboratory research could ultimately lead to new treatments to help children with Alpers’ syndrome.
Helping more children with epilepsy to live seizure-free
Epilepsy affects around 64,000 babies, children and young people in the UK – and, sadly, medications don’t work for up to one in three. Uncontrolled seizures can be extremely distressing and place children at risk of injury or even death.
Deep brain stimulation (DBS) is a promising new treatment for children with focal epilepsy. It delivers small electrical impulses to a specific area of the brain and can reduce the frequency and severity of seizures. However, DBS currently uses a ‘one-size-fits-all’ approach.
Professor Martin Tisdall, at the UCL Great Ormond Street Institute of Child Health, is investigating whether personalised DBS can achieve better results. This could help to improve seizure control and quality of life for more children with focal epilepsy.
Tackling a rising liver disease threat
Metabolic dysfunction-associated steatotic liver disease, previously known as ‘fatty liver disease’, is the most common liver condition that affects children. Over time, it can cause serious damage – and cases are rising, particularly among children with obesity.
Dr Jake Mann at the University of Birmingham aims to improve understanding of how this condition develops and progresses in children. This could lead to new tests and improved treatments – helping children live longer, healthier lives.
Joining forces with Action for A-T
We’ve also joined forces with the charity Action for A-T to help fund three new studies into Ataxia Telangiectasia (A-T). This life-limiting rare condition causes severe and progressive physical disability, suppressed immune function and a higher incidence of cancer. The projects, focusing on different aspects of the disease, will be taking place in Bristol, Cambridge and Nottingham.
You can read more about other new research funded in 2026 on previous blogs here:
Research to detect and prevent complications during pregnancy