New treatment for potentially deadly graft-versus-host disease

A discovery by QIMR Berghofer researchers has led to the development and approval of a new treatment for chronic graft-versus-host disease (GVHD), the major cause of serious complications and death in blood cancer patients who receive vital stem cell transplants.

The United States Federal Drug Administration (FDA) has fast-tracked approval of a drug based on QIMR Berghofer researcher Dr. Kelli MacDonald's findings, a decade after her breakthrough discovery of an antibody that could prevent chronic GVHD.

Chronic GVHD occurs when transplanted cells attack the recipient's organs and tissues and cause scarring which leads to debilitating and often long-lasting health problems, particularly in the skin and lungs.

Rigorous clinical trials have shown the FDA-approved treatment known as Axatilamab, successfully suppresses harmful immune cells, preventing the development of chronic GVHD. Those results were published in the New England Journal of Medicine.

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

"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.

"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 work began in 2014, when Dr. MacDonald's lab at QIMR Berghofer identified both the cellular process causing chronic GVHD, and the antibody that could block this process and prevent the disease's development.

Health care providers will soon be able to access full prescribing information on the FDA's website as the drug becomes available in the U.S. It is not yet known when the treatment will become available in Australia.

Bone marrow or stem cell transplants are critical, life-saving treatments for blood cancer patients, but helping those who suffer the complication of chronic GVHD has been a major unmet need.

"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," Dr. MacDonald said.

"It really is a dream to see your research progress from the laboratory to improve the outcome of patients. The ability of our research to lead to new drug approval really emphasizes the importance and quality of the expertise in transplant immunology, developed over two decades at QIMR Berghofer. It also highlights the persistence and timeframes needed to understand and address clinical problems in the lab."

Provided by QIMR Berghofer Medical Research Institute