Novel agents for the targeting of abnormal blood vessels in the brain to prevent stroke

    Press/Media: Research


    Brain Foundation Research Gift - Brainwaves Newsletter


    Brain Arteriovenous Malformations - developing new treatment approaches

    Period15 Dec 2019

    Media coverage


    Media coverage

    • TitleNovel agents for the targeting of abnormal blood vessels in the brain to prevent stroke
      Degree of recognitionNational
      Media name/outletBrain Founation- Brainwaves Newsletter
      Media typeWeb
      DescriptionOur goal is to develop new ways
      to treat a blood vessel disorder
      that occurs in the brain called
      arteriovenous malformations, or
      AVMs. These tangled collections
      of abnormal blood vessels can
      form in the brain during early
      development. They are highly
      prone to rupturing, leading to
      release of blood into the brain,
      causing a type of stroke. This
      form of stroke occurs primarily in
      children and young adults, rather
      than as a result of aging, so has
      a significant impact on affected
      individuals and their families.
      Despite current approaches, one-
      third of these vulnerable young
      patients lack safe treatment
      options and remain susceptible
      to stroke. To fill this gap, we aim
      to develop a vascular targeting
      approach to AVM treatment. This
      involves delivering a drug through
      the bloodstream to induce
      localised clotting and closure of
      the diseased AVM vessels. Key
      to this approach is 1) identifying
      molecular targets unique to the
      surface of the diseased blood
      vessels but absent from normal,
      healthy blood vessels; and 2)
      developing complementary
      targeting molecules that
      recognise and bind these targets
      to deliver vessel-occluding drugs
      specifically to the AVMs. The recent
      discovery that more than half of all
      AVMs are caused by mutations in
      a family of genes called RAS, for
      the first time provides a defined
      molecular cell type for AVMs that
      can be easily modelled for study
      in the laboratory. Our study aims
      to develop an AVM cell culture
      model in the laboratory that
      expresses the RAS mutation
      and then use it to generate novel
      targeting molecules (called DNA
      aptamers) that can specifically
      recognise and bind RAS-induced
      molecular targets on the surface
      of these mutant cells. These novel
      targeting molecules could then be
      used to deliver vessel-blocking
      drugs with high specificity to
      AVM vessels providing a safe and
      effective new treatment approach
      for AVM patients.
      PersonsLucinda McRobb, Marcus Stoodley