Researchers in Australia have pioneered a novel therapeutic strategy that could transform treatment for myelofibrosis, a rare and aggressive blood cancer that affects the bone marrow.
The findings, published in the journal Blood, explore ways to tackle the disease by directly targeting the abnormal blood cells driving myelofibrosis using immunotherapy, rather than merely alleviating symptoms, according to a news release from the South Australian Health and Medical Research Institute (SAHMRI) on Monday.
Myelofibrosis disrupts the production of healthy blood cells, causing fatigue, pain, and an enlarged spleen, which significantly diminishes quality of life. Current therapies primarily manage symptoms but cannot eradicate the underlying disease.
The study marks a global first in demonstrating that Type 1 and Type 2 calreticulin mutations respond differently to treatment, according to Daniel Thomas, director of SAHMRI’s Blood Cancer Program, who co-led the research with Angel Lopez, head of Human Immunology at SA Pathology.
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The team identified not one, but two distinct targets that can selectively eliminate the disease-causing cells.
“People with myelofibrosis are often treated with therapies that control symptoms, but they do not selectively attack the abnormal cells driving the disease,” Thomas said. He added that by focusing on the unique characteristics of these cells, it may be possible to develop treatments that are both more effective and more precise.
Thomas noted that the work represents a major shift in the approach to treating myelofibrosis and related blood disorders.
Lopez highlighted the role of patient-donated cells in the study, emphasising the potential of precision immunology, which leverages the immune system to recognise and target disease-causing cells while sparing healthy tissue.
“The future of cancer treatment lies in understanding disease at the molecular and immune level, then translating that knowledge into therapies that are potent, long-lasting and highly targeted,” Lopez said.
The SAHMRI release stated that further research and clinical development are required before the therapy can progress to human trials, potentially opening the door to safer and more effective treatments for patients with myelofibrosis.
