A drug that is currently approved by the FDA to treat a certain cancer is now under investigation to help prevent a deadly side effect of bone marrow transplantation known as graft-versus-host-disease (GVHD).
Researchers at the University of Michigan Comprehensive Cancer Center combined the oral drug vorinostat with medications that are normally administered after transplant. With the addition of vorinostat, 21 percent of patients developed GVHD, compared with the typical rate of 42 percent for the standard regimen.
Vorinostat is currently approved to treat patients with cutaneous T-cell lymphoma who have progressive, persistent, or recurrent disease. Senior study author Pavan Reddy, MD, hypothesized that the drug's anti-inflammatory properties could be useful in preventing GVHD, an immune response in which donor cells attack other cells in the transplant recipient's body.
Lead study author Sung Choi, MD, assistant professor of pediatrics at the University of Michigan Medical School, said in a press release:
Graft-vs.-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.
Participants in this study were older adults who underwent a reduced-intensity bone marrow transplant with cells donated from a relative. Patients received the standard post-transplant medication regimen in addition to vorinostat. The researchers found that vorinostat was safe and tolerated well by participants, with manageable side effects.
What makes vorinostat different from other anti-rejection medications is that it targets histone deacetylases. "Vorinostat has a dual effect as an anti-cancer and an anti-inflammatory agent," Choi said in the release. "That's what's potentially great about using it to prevent graft-vs.-host, because it may also help prevent the leukemia from returning."
The results of this study were presented at the American Society of Hematology's annual meeting Dec. 9 in Atlanta. This clinical trial continues to enroll patients, and researchers hope to test vorinostat soon in patients receiving bone marrow from unrelated donors.
For those of you who treat bone marrow transplant recipients, have you witnessed the unfortunate event of GVHD? If so, what was the outcome, and what treatment regimen did you use?