Leukaemia, a challenging form of blood cancer, affects both children and adults and remains one of the most common cancers among young people aged 0–19. In Australia alone, around 20,000 people were diagnosed with a blood cancer in 2023, with over 5,000 deaths recorded annually. Despite the difficulty in treating these cancers, Australian researchers have led remarkable advances in recent decades.
At the Walter and Eliza Hall Institute of Medical Research (WEHI), scientists made a major breakthrough in understanding how leukaemia works at the molecular level. Their work led to the development of a targeted drug called venetoclax. This medication works by blocking a protein called BCL2, which cancer cells use to avoid cell death. What makes venetoclax particularly effective is its precision it targets BCL2 without interfering with other crucial cellular processes, making it both powerful and relatively safe.
Between 2016 and 2019, venetoclax gained approval from health regulators in Australia, Europe, and the US for the treatment of chronic lymphocytic leukaemia (CLL). Soon after, its use was expanded to include acute myeloid leukaemia (AML), especially in patients who are not strong enough to handle aggressive chemotherapy. Clinical results have shown that when combined with other medications, venetoclax significantly improves survival rates and has become a key treatment option, particularly for older and more vulnerable patients.
In more recent developments, researchers at the Children’s Cancer Institute in Sydney have been working on a new drug candidate, ACHM-025, for treating T-cell acute lymphoblastic leukaemia (T-ALL) a highly aggressive cancer that mainly affects children. Treatment options for relapsed T-ALL are extremely limited, and survival rates can drop as low as 50%.
In preclinical trials using advanced animal models implanted with cancer cells from real patients, ACHM-025 has shown extraordinary promise. In some cases, just three weekly doses of the drug were enough to eliminate the disease even in models with drug-resistant and aggressive forms of leukaemia. When combined with standard chemotherapy, ACHM-025 performed even better, suggesting that it could potentially reduce reliance on harsh treatments and improve quality of life for young patients.
While the early results are exciting, ACHM-025 still faces a major hurdle: funding. Because T-ALL affects a relatively small number of children, pharmaceutical companies have been hesitant to invest in large-scale clinical trials. Nevertheless, researchers are actively seeking partners and support to bring this promising drug into human testing.
From the success of venetoclax now a global standard in leukaemia treatment to the hopeful early results of ACHM-025, Australia is playing a leading role in transforming how we treat blood cancers. These achievements reflect years of investment in research, collaboration across institutions, and the unwavering support of the broader community.
https://www.nhmrc.gov.au/about-us/resources/impact-case-studies/new-treatments-leukaemia
https://www.ccia.org.au/blog/new-drug-yields-extraordinary-results-in-childhood-leukaemia