Scientists tease brain aneurysm treatment with cancer drug

There may be a way to treat sensitive brain aneurysms, traditionally only addressed through delicate surgery and slim success, with an existing drug. The joint effort by scientists from UK’s University of Sussex and US’s University of Washington School of Medicine involves a receptor tyrosine kinase (TK) inhibitor – a class of drug currently used to treat cancer.

Brain aneurysms are denoted by bulge in a blood vessel caused by a weak vessel wall, which fills up and exerts pressure outwards in the limited head (cranial) space. These cause no symptoms until they rupture, and experts estimate their frequency to be from 1 in 100 to as many as 1 in 20 people.

Advanced DNA sequencing technologies allowed the US team to first identify a new genetic form of brain aneurysm (mutations PDGFRB). Usually found in other human developmental disorders, PDGFRB encodes the growth factors in vascular cells. The UK team later discovered that multiple disease-associated mutations in PDGFRB caused a specific abnormality in its encoded protein. This abnormality causes its activity to remain locked in a hyper-active form, subsequently causing the protein to always be ‘turned-on’.

Dr. Manuel Ferreira, Associated Professor of Neurological Surgery at University of Washington School of Medicine, said the research revealed a safer, more efficient treatment option for brain aneurysm patients using specific RTK inhibitors against PDGFRB variants.

Mark O’Driscoll, Professor of Human Molecular Genetics at the Genome Damage and Stability Centre at the University of Sussex, has remarked how basic lab-derived observations on a genetic level can change disease understanding and its treatments. Accordingly: “Our research focused on understanding the genetic and cellular mechanisms underlying an aneurysm; by finding a new genetic basis in some patients, we were able to demonstrate that a known cancer drug could counter this genetic basis in most instances.”

Pharmaceutical drug repurposing is fairly common – some success stories include the use of thalidomide as a treatment for leprosy and also blood cancer multiple myeloma. However, the scientists are working on testing this drug further before public health releases.

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Category: Features, Pharmaceuticals