Drug to reduce brain bleeding identified

Scientists have identified a drug, already approved for use in patients to treat urea-related disorders, that could reduce brain bleeding and risk of strokes. Brain bleeding, caused by a defect in a gene called collagen IV, can cause strokes. Mutations in collagen IV genes cause a genetic form of eye, kidney and vascular disease which affects the blood vessels in the brain and can cause brain bleeding, even in childhood.

The researchers, from the University of Manchester and University of Edinburgh in the UK, studied mice that have a similar defect in the collagen IV gene and develop the same disease as patients.

Using sodium phenyl butyric acid they were able to reduce brain bleeding, according to the study published in the journal Human Molecular Genetics. While these genetic forms of the disease are rare, the same gene has also been implicated in common forms of brain bleeding in the general population, potentially increasing the number of people that may ultimately benefit from this work.

“There are no treatments for diseases due to collagen IV mutations and brain bleeding. This work has identified a potential treatment strategy in mice and is the first step in translating this to patients,” said Tom Van Agtmael, from the University of Glasgow. “It will also help to identify for which patients this strategy may be effective and for which patients it is not recommended or may be counter-indicative.