Grant to bolster research into treatment for liver and lung disease

Associate Professor Ashley Buckle
Associate Professor Ashley Buckle, at the Monash Biomedicine Discovery Institute (BDI) has been awarded a $US200,000 grant by the Alpha-1 Foundation to further his research into a treatment for Alpha-1 Antitrypsin Deficiency (AATD).
Alpha-1 is an inherited condition in which a malfunctioning gene causes a severe lack of the protein alpha-1 antitrypsin (AAT), which is produced by the liver. It can result in liver damage and lead to liver cancer and/or lung disease.
Associate Professor Buckle, Head of the Protein Engineering and Design Laboratory at the Monash BDI, was awarded the difficult-to-obtain Alpha-1 Foundation Grant this month after a lengthy review process.
The grant, over two years, will help further the laboratory’s work into producing a synthetic molecule that would improve on the treatment available for people with AATD.
Currently those with the condition need weekly injections of the protein, purified from batches of donated plasma.
“The problem is that the protein becomes inactive after a week and also that its purity and precise make-up varies between batches,” Associate Professor Buckle said.
“We’ve made a synthetic version in the lab, which doesn’t go ‘off’ and which we can make in large amounts and very cheaply,” he said.
“The Alpha-1 Foundation grant will enable us to take this to the next round of protein engineering in which we’ll try to understand the properties of this synthetic molecule to find how to best produce a candidate for a treatment that can be used less frequently,” he said.
The funds will be used to finance a PhD student’s visit to US labs later this year to undertake collaborative work on the protein.
Associate Professor Buckle said he was grateful to Professor Roger Daly, Head of the Cancer Program at the Monash BDI and Head of the Department of Biochemistry and Molecular Biology, for the financial support to upgrade the biophysics instrumentation that will be instrumental in generating the data for the work proposed in the grant application.
The US-based not-for-profit Alpha-1 Foundation is devoted to finding a cure for AATD and to improving the lives of people affected by it globally.