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New Australian Findings Offers Hope for Rare Blood Cancer Patients

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—Australian researchers say they have identified a new, highly targeted way to attack myelofibrosis, a rare and serious blood cancer, offering fresh hope for treatments beyond symptom management.

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

The findings were published in the leading international journal *Blood* on Monday, January 12, 2026.

The research centres on eliminating the cells that cause myelofibrosis, rather than depending on therapies that mainly relieve symptoms such as fatigue, pain, enlarged spleen, and reduced quality of life.

While existing treatments can help ease symptoms, there are currently no therapies that remove the abnormal cells driving the condition.

Details of the work were outlined in a statement published on Monday on the SAHMRI website titled “New targeted treatment offers hope for people with myelofibrosis.”

The study was co-led by Professor Daniel Thomas, Director of SAHMRI’s Blood Cancer Programme, and Professor Angel Lopez, Head of Human Immunology at SA Pathology, with key contributions from Dr Denis Tvorogov of the University of Adelaide and Cancer Council SA research fellow Dr Chloe Thompson-Peach.Professor Thomas described the findings as a major advance and a world-first discovery demonstrating that Type 1 calreticulin mutations differ from Type 2 mutations in how they respond to treatment.

“People with myelofibrosis are often treated with therapies that help control symptoms, but they don’t selectively target the abnormal cells driving the disease.

“Our research shows that by focusing on what makes these cells different, it may be possible to develop treatments that are both more effective and more targeted. This is part of a major paradigm shift in the treatment of myelofibrosis and related diseases,’’ Prof Thomas said.

The researchers identified two distinct targets capable of optimally removing the disease-causing cells. A critical part of the discovery involved the use of patient samples donated for research and stored at the South Australian Cancer Research Biobank, supported by the Health Services Charitable Gifts Board.

The study underscores the potential of precision immunology, which uses the immune system to identify and destroy harmful cells while largely preserving healthy tissue.

The findings suggest that different biological forms of myelofibrosis may respond best to different targeted approaches.Professor Lopez said the work reflects a broader movement in cancer research toward more personalised and precise treatment options.

“This work shows the power of precision immunology, where treatments are designed to recognise disease-causing cells with extraordinary specificity while sparing healthy tissue.

“The future of cancer treatment lies in understanding disease at a molecular and immune level and then translating that knowledge into therapies that are, potent, long-lasting and precise,” Prof Lopez said.Researchers cautioned that despite the promising results, additional research and clinical development are needed before the approach can be tested in patients.

The team will now undertake further studies required to safely progress the treatment toward clinical trials, with the goal of converting the findings into safer and more effective therapies for people living with myelofibrosis.

Myelofibrosis (MF) is a rare blood cancer in which scar tissue accumulates in the bone marrow, disrupting normal blood cell production and leading to anaemia, infections, and bleeding disorders. It often causes an enlarged spleen and liver, fatigue, and weight loss, with treatment typically focused on symptom control, improving blood counts, and, in severe cases, stem cell transplantation.

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