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.
Role of oxidative stress in lung disease in patients with A-T
Correction of the defective gene in A-T using olfactory stem cells
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.
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.
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:
Foundation ‘Razem Zdazymy’ (Poland)
Ataxia Telangiectasia Hispana (Spain)