A ground-breaking discovery by local researchers could transform the treatment of one of the biggest triggers of cardiovascular disease \u2014 the leading cause of death in Australia.<\/p>\n
Associate Professor Juliana Hamzah and Professor Shirley Jansen, from the Harry Perkins Institute of Medical Research, have developed a world-first drug candidate \u2014 ATH01 \u2014 designed to remove cholesterol and other harmful lipids from artery walls, potentially preventing heart attacks, strokes and amputations caused by atherosclerosis.<\/p>\n
After more than a decade of research and extensive pre-clinical testing, ATH01 is heading towards its first in-human clinical trials.<\/p>\n
The pair will speak about the treatment and their research at a panel discussion in Nedlands next month.<\/p>\n
Professor Hamzah invented the drug after discovering a protein that attaches to cells in the arteries that store cholesterol and lipids, helping these cells clear the accumulated lipids.<\/p>\n
She and Professor Jansen \u2014 a fellow researcher at the institute and head of Sir Charles Gairdner Hospital\u2019s vascular and endovascular surgery department \u2014 founded biotechnology company Atherid Therapeutics, which aims to trial ATH01.<\/p>\n
It will first be trialled on patients genetically predisposed to atherosclerotic disease, known as familial hypercholesterolemia (FH), who cannot prevent plaque accumulation and are at an even higher risk of cardiovascular complications.<\/p>\n
\u201cATH01 targets the root cause by breaking down a build-up of cholesterol and other lipids, offering a transformative solution,\u201d Professor Hamzah said.<\/p>\n
\u201cATH01, developed at the Perkins, combines two powerful molecules. One is based on a natural agent already found in the body, and the other is a \u2018homing\u2019 molecule that zeroes in on cholesterol and fats in the artery walls.\u201d<\/p>\n
Professor Jansen helps patients living with peripheral arterial disease \u2014 which blocks blood flow to organs and limbs, leading to heart attacks, strokes and amputations \u2014 who could also be helped by the treatment.<\/p>\n
\u201cThis precise targeting ensures the drug reaches exactly where it is needed, which is what we designed it to do,\u201d Professor Jansen said.<\/p>\n
\u201cIt works consistently throughout the entire arterial system, and we understand how it helps the body\u2019s cells break down the cholesterol and fatty deposits.<\/p>\n
\u201cIf it continues to show success, it could transform the lives of millions.\u201d<\/p>\n
The researchers have spent 11 years working on the treatment, which Professor Hamzah said started as a doubtful idea.<\/p>\n
\u201cOur research started from nothing \u2014 an unknown, high-risk idea with many doubts about whether it was even possible to clear arteries with a medicine,\u201d she said.<\/p>\n
\u201cBut after a decade, we have proven its concept, feasibility, and consistent results. Now, we have reached the point where the drug must be manufactured to the highest standards, suitable for patient use, so we can move forward to clinical trials.<\/p>\n
\u201cThis is an exciting milestone for a new therapy that could dramatically change lives, and it\u2019s time to push ahead.\u201d<\/p>\n
The next stage of production and clinical trials is expected to cost up to $20m.<\/p>\n
Investigative journalist Miriam Borthwick will host the panel discussion on September 29 with the pair, alongside Limbs4Life chief executive Melissa Noonan and Familial Hypercholesterolaemia Australia deputy chair Christine Tawtel.<\/p>\n