FDA approves new treatment for graft-versus-host disease

The US FDA has fast-tracked approval of a drug based on findings by QIMR Berghofer researcher Dr Kelli MacDonald, a decade after her breakthrough discovery of an antibody that could prevent chronic graft-versus-host disease (GVHD).

Chronic GVHD — a major cause of complications and death in blood cancer patients who receive vital stem cell transplants — occurs when the transplanted cells attack the recipient’s organs and tissues. This causes scarring, which can lead to debilitating and often long-lasting health problems — particularly in the skin and lungs.

Back in 2014, MacDonald’s lab at QIMR Berghofer identified the cellular process causing chronic GVHD as well as the antibody that could block this process and prevent the disease’s development. The resulting drug, axatilamab, has now been shown in clinical trials to suppress harmful immune cells and prevent the development of chronic GVHD (as described in The New England Journal of Medicine).

“This is an important and exciting development for people with GVHD and has been over a decade in the making from our initial research,” MacDonald said, with the FDA-approved drug expected to meet the urgent needs of a large proportion of patients with chronic GVHD who fail to respond to initial steroid-based therapy.

“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% of patients.”

Healthcare providers will soon be able to access full prescribing information on the FDA’s website as the drug becomes available in the USA. It is not yet known when the treatment will become available in Australia.

“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,” MacDonald concluded.

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