The U.S. Food and Drug Administration (FDA) has fast-tracked the approval of axatilamab, a novel treatment for chronic Graft-versus-Host Disease (GVHD), offering hope to blood cancer patients undergoing stem cell transplants. This approval follows a decade of research led by Dr. Kelli MacDonald at QIMR Berghofer, whose breakthrough discovery of an antibody paved the way for this new therapy. Chronic GVHD is a severe complication where transplanted cells attack the recipient's organs and tissues, leading to debilitating and often long-lasting health problems, particularly in the skin and lungs.
Clinical Trial Success
Rigorous clinical trials have demonstrated that axatilamab effectively suppresses harmful immune cells, preventing the progression of chronic GVHD. The results, published in the New England Journal of Medicine, indicate that axatilamab meets the urgent needs of a significant proportion of patients with chronic GVHD who do not respond to initial steroid-based treatments. According to Dr. MacDonald, the treatment has significantly reduced symptoms in up to 75% of patients.
Scientific Background
In 2014, Dr. MacDonald's lab at QIMR Berghofer identified the cellular process responsible for chronic GVHD and discovered an antibody capable of blocking this process, thereby preventing the disease's development. This foundational work has now translated into a clinically available therapy.
Impact and Availability
Axatilamab is poised to address a major unmet need in the treatment of chronic GVHD, a condition that affects many blood cancer patients who rely on bone marrow or stem cell transplants. Healthcare providers in the USA will soon have access to full prescribing information on the FDA's website. The availability of the treatment in Australia is yet to be determined. Dr. MacDonald emphasized the importance of this approval, stating, "This approval is a big step forward for people dealing with the challenges of chronic GVHD and offers a new option for those who have struggled to find effective treatment."
A Decade in the Making
Dr. MacDonald reflected on the journey from laboratory discovery to clinical application, noting, "It really is a dream to see your research progress from the laboratory to improve the outcome of patients." She also highlighted the expertise in transplant immunology developed at QIMR Berghofer and the persistence required to address clinical problems through research.
