Graft versus host disease happens when donor immune cells from a stem cell or bone marrow transplant attack the recipient's tissues. It affects roughly half of transplant recipients, ranging from mild skin rash to severe damage to skin, liver, and digestive tract. Treatment aims to suppress the attack without losing the transplant's benefit against cancer.
What's actually going on in research
Trials are testing JAK inhibitors like ruxolitinib (FDA-approved for steroid-refractory acute GVHD in 2019), Rho kinase inhibitors, monoclonal antibodies targeting specific immune pathways, and cellular therapies using regulatory T cells. Researchers are also studying ways to prevent GVHD while preserving graft-versus-tumor effect, and new treatments for chronic GVHD affecting skin and lungs.
JAK inhibitors
Ruxolitinib became the first FDA-approved treatment for steroid-refractory acute GVHD. Trials are now testing other JAK inhibitors and using them earlier in treatment.
Regulatory T cell therapy
Studies are testing infusions of regulatory T cells—immune cells that dampen immune attacks—to prevent or treat GVHD. Early trials show promise in reducing GVHD without increasing infection risk.
Rho kinase inhibitors
Drugs that target Rho kinase pathways show potential for chronic GVHD, especially skin and lung involvement. Several agents are in mid-stage testing.
What to know before you search
Eligibility depends on GVHD type (acute or chronic), severity, organs involved, prior treatments, and time since transplant.
What types of trials are currently open
- Prevention trials — Testing drugs or cellular therapies given at the time of transplant to prevent GVHD from developing, often comparing to standard approaches like methotrexate and tacrolimus.
- Acute GVHD treatment trials — Testing new drugs for people whose acute GVHD doesn't respond to steroids, the standard first treatment.
- Chronic GVHD trials — Testing treatments for GVHD that persists or develops months after transplant, affecting skin, mouth, lungs, or other organs.
- Cellular therapy trials — Studies of regulatory T cells or other cell products designed to control GVHD without suppressing the immune system broadly.
- Supportive care trials — Testing treatments for specific GVHD symptoms like mouth pain, dry eyes, joint stiffness, or lung scarring.
Recently added Graft Versus Host Disease trials
Low-Dose Liposomal Amphotericin B for Invasive Fungal Infection Prophylaxis in Neutropenic Children
This is a single-center, single-arm, observational clinical study evaluating the efficacy and safety of low-dose liposomal amphotericin B (1 mg/kg/day, intravenous, once daily) for the prevention of invasive fungal infections in children aged 3-17 years with hematological malignancies who develop prolonged neutropenia (absolute neutrophil count ≤ 0.5×10\^9/L, expected to last \> 7 days) and are at high risk for invasive fungal disease. Participants are those who, per the treating physician's routine clinical decision, have been initiated on liposomal amphotericin B prophylaxis at 1 mg/kg/day due to intolerance or toxicity to other antifungal agents. The primary outcome is the incidence of proven or probable invasive fungal disease. Secondary outcomes include incidence of pneumonia, persistent unexplained fever \>4 days, use of additional systemic antifungal therapy, and adverse events. A total of 30 participants will be enrolled. Data will be collected at baseline, during treatment, and within 7 days after treatment completion.
Study to Evaluate the Safety and Effectiveness of Withaferin A as a Treatment to Prevent GvHD in Transplant Patients
What is acute Graft versus host disease (aGvHD)? GvHD is a complication that can occur after an allogeneic stem cell transplant resulting in damage to some organs. The death rate of GvHD patients is 15-40%. In GvHD, the donated peripheral blood stem cells or bone marrow view the recipient's body as foreign, and the donated cells/bone marrow harm the body. aGvHD usually develops in skin, liver or gastrointestinal tract, and symptoms might appear within few weeks after transplant. Symptoms of acute GvHD are observed as skin rash or reddened areas on the skin, yellow discoloration of the skin and/or eyes, and abnormal blood test results, nausea, vomiting, diarrhea, or abdominal cramping. What is the current prevention used for GvHD? To prevent development of GvHD many standard drugs like cyclosporine (CSA), tacrolimus (TAC), methotrexate (MTX), mycophenolate mofetil (MMF), rapamycin and cyclophosphamide are given. What is standardized Withaferin-A (SWA)? Withaferin-A (WA) is the main active component of Withania somnifera (Ashwagandha). It has been shown in many studies to have properties of healing and immune-modulation (improving the immune system). Studies have been done in our Clinical Pharmacology Laboratory that have shown a significant beneficial effect of this drug, when added to the standard drugs used for prophylaxis of GvHD, on reducing the risk of acute GvHD. The drug has also been tested and proven to be very safe in humans. What is the rationale of this trial? As has been described earlier, GvHD is a difficult complication of allogeneic stem cell transplant which can lead to increased hospital stay and deaths post-transplant. The standard drugs for prevention of GvHD are cyclosporine (CSA), tacrolimus (TAC), methotrexate (MTX), mycophenolate mofetil (MMF), rapamycin and cyclophosphamide. These drugs also have some side effects during the course of transplant. This points out the need for new preventive drugs that are safe and effective. SWA is an oral formulation of WA which seems to be beneficial in the early studies done in the Clinical Pharmacology Laboratory, ACTREC. SWA has also been found to be safe at very high doses. SWA will be given along with the standard drugs given to prevent GvHD. Despite of consuming these drugs, about 40 - 60% patients still develop GvHD. The investigators aim to add SWA to these standard drugs during transplant to reduce significant aGvHD. How will SWA be given? Participants who agree to participate in this trial and are found to be eligible will be given SWA as a capsule at a dose of 500 mg/day (2 capsules of 250 mg) to 3000 mg/day (6 capsules of 250 mg) as per the dose level allotted to the Patient. The drug will be given for a total duration 90 days starting from Day +1 of transplant. All other standard treatments which are part of a transplant procedure will be carried out without any change. Participants will be monitored clinically for any adverse events and followed up as per standard protocols post-transplant. What additional tests will be carried out? Additional blood sampling to study the levels of the drug WA blood samples will be collected at 0, 1, 2, 4, 8 hours on the day of start of SWA (Day +1) and Day +7. Checking immune cell profile and cytokines (which are markers of - immunity levels) will be done at Day+30, Day +90, Day+180, Day+365 from the start of SWA which is also the part of routine care. Blood sample of 5 ml will also be taken at Day 0, Day +14, Day +30, Day +60 and Day +90 after start of drug to see level of some special protein called JAK2 STAT3 protein. What are the risks involved in participation? According to available literature and information, SWA is a safe and well tolerated drug. In a phase 1 study, Standardized Withaferin-A was administered to Patients with advanced stage osteosarcoma. The drug was well tolerated by Patient up to a dose of 4800 mg. No severe side effects were observed. Increase in liver enzymes and skin rash were the most common side effects. Other side effects included fatigue, fever, swelling, and diarrhea. What is the possible impact of this trial? If indeed SWA works and prevents GvHD effectively then this could be a breakthrough in treatment. It would help many Pateints to prevent GvHD post allogeneic stem cell transplant and would be a safe, easily available, inexpensive and oral drug for the same. This possibly could benefit and help future Patients who undergo bone marrow transplant (BMT) to have better chances of survival and reduce their financial burden.
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