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

Press/Media: Research


Brain Foundation Research Gift - Awards night


Development of novel treatment approaches for brain AVMs

Period21 Oct 2019

Media coverage


Media coverage

  • TitleBrain Foundation Research Gift
    Degree of recognitionNational
    Media name/outletBrain Foundation of Australia
    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