Research - BrAshA-T, Brisbane, Australia

Research

Along with other scientists from around the world, we are very fortunate to have two dedicated and highly talented A-T research teams in our home city of Brisbane, Australia working on a cure for A-T.

Much of this work is based on delivering gene-corrected cells to the brain of a mouse and determine if they integrate and function correctly. Once this
capability becomes clinically viable the hope is to be able to apply this cell-based therapy to children with A-T.

 

  • UQCCR (University Queensland Centre for Clinical Research) led by Professor Martin Lavin
    • Role of oxidative stress in lung disease in patients with A-T

    • Correction of the defective gene in A-T using olfactory stem cells

 

  • AIBN (Australian Institute for Bioengineering and Nanotechnology) led by Professor Ernst Wolvetang
    • Generation of AT-iPSC (A-T induced pluripotent stem cells)

    • Correction of AT-iPSC genes

    • Differentiation of AT-iPSC into cerebellar cells (including generation of reporter lines)

    • Testing integration of cerebellar cells into the hindbrain of mice

Click here for an extract from the AIBN 2015 Annual Report describing the work undertaken by Ernst and his team.

 

Research Papers

ATM Kinase Methods and Protocols (Apr 2017)

Therapeutic targets and investigated treatments for A-T (Nov 2016)

White Matter/Gray Matter Imaging paper (May 2014)

A-T Imaging Review paper (Mar 2014)

AIBN Update (Nov 2013)

UQCCR (formerly QIMR) Update (Nov 2013)

A patient-derived olfactory stem cell disease model for A-T (Jun 2013)

Step closer to understanding childhood degenerative brain disease (Jul 2012)

 

Clinical Trials

We are pleased that as at 30 November 2016, the FDA removed all of its concerns and provided the green light to proceed with the ATTeST trial. We now need approval from the Australian authorities but hopefully that should be a mere formality.

The trial will assess the efficacy and safety of dexamethasone sodium phosphate (the study drug), delivered via the patient’s own red blood cells (RBCs), in treating the neurological symptoms of (A-T).  

Erydex has launched a website providing further information for interested families. The site also has a separate section aimed at children who might participate in the trial, with simple explanations about the trial and the procedures involved. 

Read more about the trial at attest-trial.com.

 

Conference Notes

Third Ataxia-telangiectasia Clinical Research Conference 2016 Warsaw, Poland

 

Interviews

Sam Nayler is one of our devoted A-T researchers who works with Professor Ernst Wolvetang and his team at the AIBN at the University of Queensland.  You can listen to his recent (11 Nov 2016) interview on Stem Cell Research on Radio 4EB here:

 

International Charities

 

 

 

 

 

 

Foundation ‘Razem Zdazymy’ (Poland)

 

The Association for Fighting A-T Disease (Israel)

 

Ataxia Telangiectasia Hispana (Spain)

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