Professor Tim Hughes, an Adelaide-based haematologist, has spent over two decades transforming the outlook for patients with chronic myeloid leukaemia (CML), a blood cancer that was once almost always fatal within three to four years. His pioneering work has led to breakthroughs that now allow some patients to achieve treatment-free remission.
From fatal to manageable
When Professor Hughes began his career, CML was a death sentence. Most patients survived just three to four years. However, around 2000, the emergence of targeted therapies known as tyrosine kinase inhibitors revolutionised treatment. These drugs target the exact genetic fault that causes CML, switching off the overdrive signal in blood cells. Professor Hughes and his team were among the first to trial these therapies, with dramatic results.
“It was a completely different way of treating cancer – targeting the specific driver of the disease rather than using more general treatments,” he explains.
Three major breakthroughs
Professor Hughes’s work has driven three key advances. The first was the development of a highly sensitive blood test that can detect tiny traces of the disease. This allows doctors to track exactly how well a patient is responding to treatment, providing a clearer picture of the disease status.
The second breakthrough was the discovery that some patients could stop treatment altogether. Previously, patients were told they would need to take medication for life. However, some patients achieved such low levels of leukaemia that it became undetectable. These patients could cease therapy and remain free of detectable leukaemia. Currently, about one-third of patients achieve this treatment-free remission, but Professor Hughes notes there is still a long way to go.
The third breakthrough was the development and early use of a highly specific next-generation drug that works differently from earlier treatments. This new targeted therapy is much more precise, blocking the cancer-driving protein without affecting normal enzymes. “It is so specific in its ability to inhibit the leukaemia kinase that it doesn’t affect other normal kinases at all,” says Professor Hughes. “It’s the first time that’s ever been achieved. We’ve now treated over 200 patients in Australia with this drug as first-line therapy, and the results have been extremely good.”
Future directions
Despite these advances, five to ten per cent of patients still develop aggressive disease. Professor Hughes believes the next step lies in combining better drugs with new approaches, particularly harnessing the immune system. He and his colleagues are exploring how immune responses might help maintain deep remissions and reduce the likelihood of relapse over time.
This work is carried out by a large multidisciplinary team at the South Australian Health and Medical Research Institute (SAHMRI), alongside collaborators at the Royal Adelaide Hospital and Adelaide University, with strong integration between laboratory research and clinical care.
Patients drive progress
Professor Hughes credits his patients for their willingness to participate in clinical trials. “Almost everyone is willing to take part in clinical trials,” he says. “Even if it won’t help them directly, they want to help the next person. That’s pretty remarkable.”
For Professor Hughes and his team, the sense of purpose is what continues to drive the work forward. “You can see the difference this work makes,” he says. “That’s what matters – to have patients who generally don’t even know they are being treated is a huge step forward.”
