A new class of drugs reduced the risk of patients contracting a serious and often deadly side effect of lifesaving bone marrow transplant treatments, according to a new study from researchers.

The study, the first to test this treatment in people, combined the drug vorinostat with standard medications given after transplant, resulted in 22% of patients developing graft-versus-host disease (GVHD) compared with 42% of patients who typically develop this condition with standard medications alone. Results of the study, conducted by researchers at the University of Michigan (U-M) Comprehensive Cancer Center, appear in The Lancet Oncology (2013; doi:10.1016/S1470-2045(13)70512-6).

“Graft-versus-host disease is the most serious complication from transplant that limits our ability to offer it more broadly. Current prevention strategies have remained mostly unchanged over the past 20 years. This study has us cautiously excited that there may be a potential new way to prevent this condition,” said lead study author Sung Choi, MD, assistant professor of pediatrics at the U-M Medical School.

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Vorinostat is currently approved by the US FDA to treat certain types of cancer. But U-M researchers, led by senior study author Pavan Reddy, MD, found in laboratory studies that the drug had anti-inflammatory effects as well, which they hypothesized could be useful in preventing GVHD. GVHD is a condition in which the new donor cells begin attacking other cells in the patient’s body.

The study enrolled 61 older adults from U-M and Washington University in St. Louis, Missouri, who were undergoing a reduced-intensity bone marrow transplant with cells donated from a relative. Patients received standard medication used after a transplant to prevent GVHD. They also received vorinostat, which is given orally as a pill. Fifty of the 61 participants completed the full 21-day course of vorinostat.

The researchers found vorinostat was safe and tolerable to give to this vulnerable population, with manageable side effects. In addition, rates of patient death and cancer relapse among the study participants were similar to historical averages.

The results mirror those found in the laboratory using mice. Reddy has been studying this approach in the lab for 8 years.

“This is an entirely new approach to preventing graft-versus-host disease,” Reddy said. Specifically, vorinostat targets histone deacetylases, which are different from the usual molecules targeted by traditional treatments.

“Vorinostat has a dual effect as an anticancer and an anti-inflammatory agent. That’s what’s potentially great about using it to prevent graft-versus-host, because it may also help prevent the leukemia from returning,” explained Reddy.

“We are encouraged by our findings,” Choi said. “Vorinostat combined with standard graft-versus-host disease prophylaxis after related-donor transplant appears to be safe and associated with lower than expected incidence of acute GVHD. Future studies are needed to assess the effect of vorinostat in broader transplant settings. We are currently investigating vorinostat plus standard therapies to prevent GVHD in transplants with an unrelated donor.”