Trial: Preventing Graft-versus-Host Disease

Name of the Trial
Randomized Pilot Study of Donor Th2 Cells Generated In Vitro by Sirolimus Treatment with or without Oral Sirolimus versus Oral Sirolimus Alone for Prevention of Graft-versus-Host Disease after Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Hematologic Malignancies (NCI-04-C-0055). See the protocol summary at

Principal Investigator
Dr. Daniel H. Fowler, with Dr. Michael R. Bishop (Protocol Chair), NCI Center for Cancer Research

Why is This Trial Important?
Patients with hematologic malignancies, such as leukemia, lymphoma, and multiple myeloma, can be cured with allogeneic hematopoietic stem cell transplantation (HSCT). In HSCT, T lymphocytes and peripheral blood stem cells from a sibling donor are infused into a cancer patient's bloodstream after the patient has received preparatory chemotherapy. The donor's T lymphocytes can recognize the patient's cancer cells as foreign and attack them, leading to a potentially curative graft-versus-tumor (GVT) effect.

However, donor T lymphocytes, in addition to mediating beneficial GVT effects, may also attack the patient's normal tissues, causing graft-versus-host disease (GVHD). GVHD is the major life-threatening complication of allogeneic HSCT. Cyclosporine, a drug that suppresses immune system function, is usually given after HSCT to prevent GVHD. Nonetheless, moderate-to-severe GVHD can develop in approximately 50 percent of transplant patients who receive cyclosporine.

Researchers are investigating whether another immunosuppressive drug, sirolimus, can work with cyclosporine to prevent GVHD more effectively. Sirolimus is thought to prevent GVHD in part by stimulating the formation of a class of immunosuppressive cells, called Th2 cells, in donor T lymphocytes. Sirolimus can be used to generate donor Th2 cells in vitro before transplantation.

In this randomized trial, each patient who receives HSCT is treated with cyclosporine and one of the following additional treatments: 1) sirolimus tablets, 2) sirolimus-generated donor Th2 cells, or 3) sirolimus tablets and sirolimus-generated donor Th2 cells.

Who Can Join This Trial?
Researchers will recruit 30-76 patients aged 18 to 75 who have been diagnosed with hematologic malignancies or related conditions. See the complete list of eligibility criteria at

Where Is This Trial Taking Place?
The study is taking place at the NIH Clinical Center in Bethesda, Md.

Who to Contact
For more information, contact the NCI Clinical Studies Support Center at 1-888-NCI-1937. The toll-free call is confidential.

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