Early trial results show promising results for preventing GVHD
CANCER DIGEST – Dec. 8, 2024 – A new drug is showing promise of significantly reducing one of the most serious complications of stem cell transplants in results of an early clinical trial.
Researchers at Washington University School of Medicine in St. Louis published the early results online Nov. 22, 2024 in the journal Blood showing that the drug itacitinib drastically reduced graft-versus-host disease (GVHD) for stem cell transplant patients who do not have a fully matched donor.
Stem cell transplantation is the standard treatment of several types of blood cancers, including leukemias and lymphomas. Blood stem cells are the cells produced in the bone marrow that develop into all the different types of cells that make up the blood and immune system. The treatment involves eradicating a person’s diseased immune system and replacing it with stem cells from a healthy a donor.
One of the most difficult challenges of such transplants is the problem of GVHD, a complication in which the new donor immune system recognizes the patients tissues as "foreign" and attacks them. Severe GVHD can be fatal to the patient. To minimize that complication, doctors have sought to use donors whose immune cells most closely matched those of the patient. Unfortunately, finding identically matched donors is difficult. Using stem cells from the patient’s siblings or other close family members is the best option, but these donors are usually only half-matched.
Half-matched stem cell transplants are now common as treatments to reduce GVHD have improved to make the treatment safer and less arduous. The research into reducing GVHD has been the focus of a team led by John DiPersio, MD, PhD of the Siteman Cancer Center at Washington University.
The Phase I trial involved 42 patients diagnosed with acute myeloid leukemia, acute lymphoblastic leukemia or myelodysplastic syndrome at high risk of GVHD. All were treated with the new drug itacitinib before stem cell transplantation and four to six months after transplant. After six months, none of the 42 patients had developed severe GVHD. That compared to historical data showing a 10% to 15% rate of severe GVHD for such patients.
After one year, 89% of patients had experienced no chronic GVHD, and 80% survived, compared to a historical average of 60% to 80% overall survival.
“We saw no severe GvHD, and the rates of relapse were lower than expected in these high-risk patients," DiPersio said in a press release. "Low GvHD rates and low relapse resulted in very encouraging survival for the patients in this study. These early results are compelling, and we hope to conduct a bigger randomized controlled clinical trial to be able to evaluate efficacy.”
Itacitinib is one of a class of new drugs called JAK inhibitors used to reduce GVHD. Some of these drugs have already been FDA approved for treatment of GVHD after stem cell transplants. Itacitinib is the first such drug being tested to prevent GVHD.
Sources: Washington University School of Medicine press release, journal Blood
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