Australian researchers have developed a targeted therapeutic strategy that could improve treatment for myelofibrosis, a rare and serious blood cancer.
The study, published in Blood, explores using immunotherapy to attack the abnormal blood cells that drive the disease rather than simply managing symptoms, according to a Monday news release from the South Australian Health and Medical Research Institute.
Myelofibrosis disrupts the body’s ability to produce healthy blood cells, leading to fatigue, pain and an enlarged spleen. Current treatments can ease symptoms but do not eliminate the disease, the institute said.
The findings mark a significant step toward more precise, disease-focused therapies and show for the first time that Type 1 and Type 2 calreticulin mutations respond differently to treatment, said Daniel Thomas, director of SAHMRI’s Blood Cancer program. Thomas co-led the research with Angel Lopez, head of Human Immunology at SA Pathology.
Researchers identified two targets that effectively remove the abnormal cells.
“People with myelofibrosis are often treated with therapies that help control symptoms, but they don’t selectively target the abnormal cells driving the disease,” Thomas said. By focusing on what distinguishes those cells, he said, future treatments could become more effective and more targeted.
Lopez said the work underscores the promise of precision immunology, which uses the immune system to identify and attack disease-causing cells 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,” Lopez said.
Further research and clinical development are needed before the therapy can move to human trials, the institute said.