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University College London
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Batten disease
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£83,510
Batten disease is a devastating condition for affected children and their families. Dr Sara Mole, at University College London, will lead a team in testing new drugs to find effective treatments. The project will involve screening thousands of drugs in the hope of finding compounds that could prevent the onset and slow down the progression of this condition.
Current situation
Batten disease is a group of rare conditions which affects cells in the brain gradually causing a loss of physical and mental abilities. Children can be affected at or soon after birth or right up to their teens. It is an inherited condition, passed on through one of 13 faulty genes. Symptoms include failing sight, epilepsy and seizures, the decline of speech and language and loss of mobility. Children and young adults will eventually become completely dependent on their families and carers. The condition leads to premature death. As many as 150 children in the UK are currently thought to be affected.
At present, there is no cure for Batten disease and a lack of drugs to treat the condition. Researchers want to find new ways to ease symptoms and slow the effects of the condition.
How will this help?
Dr Sara Mole is a leading expert in Batten disease and has researched in this area for over 20 years. Her team is aiming to find drugs that could help to slow down progression of the disease or delay its onset at an early stage. As more research is done into this rare condition, scientists are hoping to unlock their understanding of how and why it occurs and to develop ways to prevent and treat symptoms.
In order to do this, they will model the disease in yeast first to test the effects of thousands of drugs. The most promising ones will then be tested in the zebrafish model. Identifying an effective drug could lead to treatments and therapies for children with this condition being developed in five to ten years. Some of these drugs may even help children with other diseases as well. It is hoped that the first benefits to patients could be seen in as little as four years if the project is successful.
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