Drug to reduce brain bleeding, identified stroke risk

Scientists have identified a drug, already approved for the treatment of urea-related disorders, that could reduce brain bleeding and the risk of stroke.

Brain bleeding, caused by a defect in a gene called collagen IV, can cause strokes. Mutations in the IV collagen genes cause a genetic form of ocular, renal and vascular disease that affects the blood vessels of the brain and can cause brain bleeding even during childhood.

Researchers at the University of Manchester and the University of Edinburgh in the United Kingdom studied mice with a similar defect in the collagen IV gene and developing the same disease as the patients.

Using phenylbutyric acid sodium, they were able to reduce brain bleeding, according to the study published in the journal Human Molecular Genetics. .

However, treatment did not treat ocular or renal disease badociated with these genetic abnormalities.

The researchers believe that, for future treatments, patients with these mutations should be divided into groups according to their symptoms and medical characteristics. history and the mechanism that causes the disease.

This precision medicine approach will enable more effective and targeted treatment of patients, they said.

Because 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 who could benefit from this work.

Additional research is now needed. understand how these mutations cause disease in different tissues to develop strategies to treat all clinical symptoms, including ocular and renal diseases,

Specific treatments that target bleeding from the brain, including common diseases such as haemorrhagic stroke

Researchers now hope that this new knowledge will provide new treatment opportunities for patients with these conditions.

"There is no cure for disease caused by collagen IV mutations and cerebral haemorrhage This work has identified a potential treatment strategy in mice and is the first step to translating it into patients", said Tom Van Agtmael, of the University of Glasgow.

"It will also help identify patients for whom this strategy can be used effectively and for which patients is not recommended or may be contra-indicative.This personalized approach to medicine will be important for developing treatments. future most effective ones, "said Agtmael.