Breakthrough Immunotherapy

Graft-versus-host disease (GvHD) is a condition that can develop in response to an allogeneic stem cell transplant or a reduced-intensity allogeneic transplant. It can be both acute and severe, and has the potential to be life-threatening. This condition has been a major stumbling block in advancing stem cell research, as life-threatening consequences of transplants are not exactly ideal. But this all could change with a phase-2 clinical trial, presented by Seattle Children's Research Institute at the 59th American Society of Hematology (ASH) Annual Meeting, that shows how an immunotherapy drug can almost entirely eliminate severe acute GvHD.

Dr. Leslie Kean, the trial's principal investigator and associate director of the Ben Towne Center for Childhood Cancer Research at Seattle Children's, said that "Given the serious threat of graft-versus-host disease, new approaches to make stem cell transplants safer for patients remain a critical unmet need. To see such striking results in patients at extremely high risk for graft-versus-disease is incredibly encouraging."

This trial tested the drug abatacept (orencia) in patients who developed GvHD from hematopoietic stem cell transplants. When the drug was used in addition to standard GvHD treatment, it reduced the occurrence of acute, grade III-IV GvHD from 32 to 3 percent. These incredible results were for both adults and children. The group who received abatacept alongside their normal treatment experienced increased survival and recovery.

Advancing Stem Cell Research

Kean stated that "As a transplant physician, it's beyond heartbreaking to witness a patient develop severe acute graft-versus-host disease after having their leukemia cured through bone marrow transplant. To have a therapy at our disposal that safely targets just the T cells causing graft-versus-host disease would represent a major step forward in stem cell transplantation. It not only offers new hope that we can prevent graft-versus-host disease upfront, but that we can also significantly improve outcomes for patients requiring high-risk transplants."

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After these successful results, the second cohort finished enrollment in November, with 140 total patients. Data from this next step in this study will likely be available within the next six months.

This continuing study will not only provide necessary improved treatment for those battling life-threatening complications through this condition — it will also advance stem cell research. Blindness, non-healing wounds, HIV, and even aging are among the seemingly countless conditions that stem cell treatments could alleviate. But, among the many roadblocks in this type of research is this very serious disease. Until now, it was something that was a clear and life-threatening reality of stem cell transplants. But thanks to this study, there is better treatment and there could be better prevention of this heartbreaking disease.

This could allow research to push forward without patients having to risk their lives. So, while it could save lives through treatment with the immunotherapy drug, it could also save lives by allowing advanced medical research using stem cells to move forward. This development is a win-win for the future of medicine, the future of stem cell research and its life-saving potential.

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