Australian team develops targeted therapy for rare blood cancer

Alsharq Tribune-AFP 

Researchers in Australia have developed a new targeted therapeutic approach that could improve treatment for myelofibrosis, a rare and serious form of blood cancer.

Published in the journal Blood, the research focuses on finding smarter ways to treat myelofibrosis by targeting the abnormal blood cells that drive the disease using immunotherapy, rather than just managing symptoms, said a news release from the South Australian Health and Medical Research Institute on Monday.

Myelofibrosis disrupts the production of healthy blood cells, causing fatigue, pain and an enlarged spleen, reducing quality of life, the release said, adding that current treatments ease symptoms but cannot eliminate the disease.

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, according to Daniel Thomas, director of SAHMRI's Blood Cancer program, who co-led the study with Angel Lopez, head of Human Immunology at SA Pathology.

The team found not just one, but two different targets that optimally remove the culprit 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," said Thomas, adding that the research showed that by focusing on what makes these cells different, it might be possible to develop treatments that are both more effective and more targeted.

He noted the research is part of a major paradigm shift in the treatment of myelofibrosis and related diseases.

Using patient-donated cells, the research highlights the potential of precision immunology, which harnesses the immune system to recognize and act on disease-causing cells with extraordinary specificity while sparing healthy tissue, Lopez said.

"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," said Lopez.

Further research and clinical development are needed before the therapy advances to human trials, potentially paving the way for safer, more effective treatments for myelofibrosis, according to the release.