Healthcare (Commonwealth Union) – Researchers at QIMR Berghofer have achieved a significant milestone with the development and approval of a new therapy for chronic Graft-versus-Host Disease (GVHD), a leading cause of severe complications and mortality in blood cancer patients undergoing life-saving stem cell transplants.
The severity of GVHD can vary greatly among patients. Several factors influence the risk of developing the condition, including the degree of tissue matching between the donor and recipient, the type of transplant (peripheral blood stem cells, bone marrow, or umbilical cord blood), and the use of immunosuppressive drugs to prevent the disease. Patients who receive transplants from unrelated donors are at a higher risk of GVHD compared to those who receive transplants from related donors, due to a lower degree of tissue compatibility.
Current treatment options for GVHD are limited and often involve the use of immunosuppressive drugs to reduce the activity of the donor cells. These medications can be effective in managing the symptoms of GVHD, but they also increase the risk of infections and other complications.
The U.S. FDA (Food and Drug Administration) has granted expedited approval for a drug stemming from the work of Dr. Kelli MacDonald, a QIMR Berghofer scientist whose groundbreaking discovery a decade ago identified an antibody capable of preventing chronic GVHD.
Researchers of the study highlighted the fact that chronic GVHD arises when transplanted cells attack the recipient’s organs and tissues, resulting in scarring that leads to debilitating, long-term health issues, particularly affecting the skin and lungs.
Extensive clinical trials have demonstrated the efficacy of Axatilamab, the FDA-approved therapy, in suppressing harmful immune cells and halting the progression of chronic GVHD. These findings have been featured in the esteemed New England Journal of Medicine.
This new treatment is expected to address the urgent needs of many patients with chronic GVHD who do not respond to standard steroid-based therapies.
“This is an important and exciting development for people with GVHD and has been over a decade in the making from our initial research,” explained Dr MacDonald.
“Until recently, there has been no satisfactory treatment for people affected by this debilitating condition which causes inflammation and fibrosis in tissues throughout the body. However, this new treatment has significantly reduced symptoms in up to 75 per cent of patients.
“I’m incredibly proud that a world-first pre-clinical discovery made right here in Brisbane is having such a tangible impact on the lives of patients across the world.”
The journey started in 2014, when Dr. MacDonald’s team at QIMR Berghofer uncovered the cellular mechanism responsible for chronic GVHD and identified an antibody capable of inhibiting this process, thereby preventing the disease from developing.
Healthcare professionals in the USA will soon have access to comprehensive prescribing details on the FDA’s website as the medication becomes available. However, it remains unclear when this treatment will be accessible in Australia.
Researchers pointed out that bone marrow and stem cell transplants are vital, life-saving options for patients with blood cancer. However, addressing the challenge of chronic GVHD as a complication has long been a significant unmet medical need.
Dr. MacDonald described the approval as a significant milestone for individuals managing the challenges of chronic GVHD, providing a much-needed option for those who have struggled to find effective treatments.
She further indicated that it is incredibly rewarding to see research evolve from the lab to directly improve patient outcomes. Dr. MacDonald also pointed out that this achievement underscores the value of two decades of transplant immunology expertise developed at QIMR Berghofer. It also highlights the dedication and lengthy efforts required to tackle clinical challenges through laboratory research. Dr. MacDonald’s work was supported by grants from the Rio Tinto Ride to Conquer Cancer, the National Health and Medical Research Council (NHMRC), and Cancer Council Queensland.
