New targeted treatment offers hope for people with myelofibrosis

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Australian researchers say they have identified a new, highly targeted way to attack myelofibrosis, a rare and serious blood cancer, raising hopes for more effective treatments that go beyond symptom control.

Scientists from the South Australian Health and Medical Research Institute, SA Pathology and the University of Adelaide have developed an immunotherapy-based approach that directly targets the abnormal blood cells driving the disease.

Myelofibrosis disrupts the body’s ability to produce healthy blood cells, leading to fatigue, pain, enlarged spleen and reduced quality of life. While current treatments can help relieve symptoms, there are no available treatments that remove them.

The study was co-led by Professors Daniel Thomas, director of SAHMRI’s Blood Cancer program, and Angel Lopez, Head of Human Immunology at SA Pathology, with significant contribution by Dr Denis Tvorogov from Adelaide University and Cancer Council SA research fellow, Dr Chloe Thompson-Peach.

Professor Thomas says the research represents an important step towards more precise, disease-focused treatments and is a world-first showing Type 1 calreticulin mutations differ from Type 2 mutations in terms of treatment. The team found not just one, but two different targets that optimally remove the culprit cells. A vital and unique resource that led to the discovery were patient cells generously donated for research stored at the South Australian Cancer Research Biobank (SACRB), supported by the Health Services Charitable Gifts Board.

The study highlights the potential of precision immunology, an approach that uses the immune system to recognise and act on disease-causing cells while leaving healthy cells largely unaffected. The findings suggest that different biological forms of the disease may benefit from different targeted strategies.

The study highlights the promise of precision immunology, an approach that harnesses the immune system to recognise and attack disease-causing cells while largely sparing healthy tissue. The findings suggest that different biological forms of myelofibrosis may benefit from different targeted strategies.

Professor Lopez said the work reflects a broader shift in cancer research toward more personalised and precise therapies.

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