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Combination therapies for juvenile Batten disease

The juvenile form of Neuronal Ceroid Lipofuscinoses (juvenile Batten disease) is the most common of a group of fatal inherited disorders that result in childhood dementia.

Approximately 200 – 250 children are affected by Batten disease in the UK with over half of these having the juvenile form and at least 10 new children diagnosed each year.

Children with juvenile Batten disease manifest the condition in early childhood, rapidly become blind, developing seizures that worsen progressively. Many disturbing psychiatric symptoms also occur including anxiety, aggressive and depressive behaviours, sleep disturbances and hallucinations. Over time, the children progressively decline and can remain in a profoundly disabled state for many years, before they typically die in their mid-to-late twenties.

Batten disease is a devastating condition both for the child, their family and the healthcare professionals involved in their care. At present there is no effective cure or treatment available.

Potential impact

Batten disease attacks the brain, but also activates the body’s immune system, causing more harm. Recently research has shown that suppressing the body’s immune response has positive effects on the disease, although the illness remains. Commonly used anti-inflammatory drugs (such as ibuprofen) and drugs which are known to protect brain cells, already in use in children, will be screened alone or together in different combinations with immunosuppression, to determine the best means to treat juvenile Batten disease.

The hope is to fast track treatments through to later phases of clinical trials in children.

Location

King’s College London, University of London

Grant awarded

£148,418 over 24 months