Gene Therapy Future for Propionic Acidemia

When Charlize underwent her life-saving liver transplant, mum Julie made sure the cells went to the Gene Therapy Unit at Children’s Medical Research Institute (CMRI) in Sydney which is led by Professor Ian Alexander.

As a scientist and a clinician, he wants to get to a point where children don’t need to have a transplant and could instead use gene therapy to replace or correct a faulty gene.

“This technology could translate into saving the lives of infants with life-threatening conditions,’’ Prof Alexander said. “It’s about getting cures into the clinic as soon as we can.

“We’re trying to get to a point where instead of a liver transplant in a very young infant, we can genetically repair the liver without major surgery. It’s a very exciting time to be doing gene therapy.’’

Professor Alexander works with Dr Leszek Lisowski who leads the Translational Vectorology Group at Children’s Medical Research Institute. He produces the vectors to make gene therapy possible. Together they would like to establish a facility in Westmead that would manufacture vectors, so patients weren’t waiting years for them to come from overseas.

“CMRI is globally competitive in gene therapy, with a special interest and a strong track record in liver-targeting therapies,’’ Professor Alexander said. “Currently, our expertise is in gene therapies for the liver, for which we are globally recognised, but we also work on other tissues besides liver. It is not just about the technology, it is about the patients.’’

Together Professor Alexander and Dr Lisowski want to see things work faster in Australia.

“Gene therapy has the potential to cure kids right now,’’ Professor Alexander said. “Gene therapy has the potential to cure kids right now, and we need to be able to conduct trials here in Australia.

“Australia punches well above its weight, given only 3% of global biomedical research happens here. However, most trials are conducted overseas, and our aim is to bring overseas clinical trials to Australia as soon as possible so we don’t have to wait two years for clinical vectors.

We need a facility in this country to be able to compress a 5-7-year journey into a 2-3-year journey for children with devastating diseases.

“Delivering gene therapies from bench to bedside within two years would be extremely exciting.’’

From the Summer 2019 OAA Newsletter