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Stem Cell Transplant Clinical Trials and Research

  • Corey Cutler, MD, MPH, FRCPC, and John Koreth, MBBS, DPhil

  • Improving Outcomes through Innovative Science

    For many patients with advanced/aggressive hematologic malignancies, allogeneic hematopoietic stem cell/bone marrow transplant (HSCT) offers the only opportunity for cure. However, some patients relapse, and many experience other complications their transplant. Our research efforts are focused on making stem cell/bone marrow transplant safer and more effective, reducing transplant-related complications, and improving immune system recovery.

    As one of the largest stem cell/bone marrow transplant centers in the world, we have the clinical volume and expertise to evaluate multiple approaches to improve transplant outcomes. Our robust research enterprise is focused on several key areas:

    • New treatments to better prevent or treat graft-versus-host disease (GvHD), a common and potentially severe side effect of allogeneic HSCT in which donor immune cells attack healthy patient cells and tissues
    • Novel immunotherapy and vaccine therapy strategies to stimulate the curative graft-versus-tumor (GvT) effect to control disease relapse
    • Therapies to address complications that arise due to the weakened immune system of stem cell transplant patients

    While advances have been made, there is more work to be done. Our program currently offers more than 15 clinical trials for stem cell/bone marrow transplant patients, many of which are available only at Dana-Farber Brigham Cancer Center. Key areas of research are featured below. See a full list of our stem cell/bone marrow transplant clinical trials.

    Graft-vs-Host Disease

    Dana-Farber is one of the world's leading research and treatment centers for acute and chronic GvHD, a challenging complication of allogeneic HSCT that can impact both quality of life and survival. Our scientists made the groundbreaking discovery of the role that both B cells and interleukin-2 (IL-2) can have in managing GvHD, and several studies examining these approaches and other novel therapies are ongoing. Some of these trials in GvHD management and prevention are highlighted below.

    Acute GVHD (aGvHD)

    For patients who receive stem cells from unrelated, mismatched donors, there is an increased risk of aGvHD and infections. Despite the use of drugs such as methotrexate, tacrolimus, and cyclosporine A, many patients still suffer from acute or chronic GvHD.

    • 20-522 - Preventing Acute GVHD in the Gastrointestinal Tract
      This is a phase 1b study evaluates UTTR1147A – a human interleukin (IL)-22 fusion protein – to prevent acute graft-versus-host disease (aGVHD) in patients undergoing HSCT. Pre-clinical studies of IL-22 show it can promote mucosal healing of the gastrointestinal tract, a key site of aGVHD.
    • 18-270 – T Cell Depletion in Mismatched Transplantation to Prevent aGvHD
      This phase 2 study evaluates selective graft engineering (i.e., depletion of alpha/beta T cell subpopulation) of the donor stem cell graft prior to transplantation as an alternative, yet effective, means of achieving GvHD prevention, while preserving immune recovery and the curative GvT effect.
    • 18-647 – Treating aGvHD with EQ001
      This phase 1b/2 study evaluates EQ001 in treating newly-diagnosed aGvHD. EQ001 targets CD6, a protein with an important immune system role. In previous studies we documented that depletion of CD6+ immune cells from donor stem cell graft was useful for GvHD prevention, suggesting that CD6+ immune cells are a useful target for aGvHD treatment.

    Chronic GvHD

    Chronic GvHD (cGvHD) remains a major complication of allogeneic HSCT, occurring in approximately 50 percent of transplant survivors and involving inflammation in multiple organs. Patients with cGvHD require prolonged immunosuppressive treatment, but some experience significant side effects and unsatisfactory outcomes, particularly patients with high-risk features of cGvHD.

    • 18-499 – KD025 for Chronic Graft-versus-Host Disease
      This phase 2 study evaluates KD025 for patients with chronic graft-versus-host disease (cGVHD). KD025 (also known as belumosudil) is an orally available Rho-associated coiled-coil kinase 2 (ROCK2) selective inhibitor. KD025 has been shown to downregulate pro-inflammatory T helper 17 cells and T follicular helper cells while upregulating anti-inflammatory regulatory T cells, which may potentially correct the immunological imbalance seen in cGVHD.
    • Read the study update from ASH 2020.

    • 16-256 – Preventing Chronic GVHD with Obinutuzumab
      This randomized phase 2 trial evaluates obinutuzumab, a potent anti-B cell agent, as a preventative therapy for cGvHD after HSCT. Patients will be randomized to receive obinutuzumab or a placebo at 3, 6, 9 and 12 months after HSCT.
    • 18-700 – AMG592 for Steroid-Resistant cGvHD
      This study builds on our research on the role of low-dose IL-2 in enhancing the activity of natural anti-inflammatory regulatory T cells (Tregs) for improving GvHD symptoms. It evaluates the ability of AMG592 (a long-acting IL-2 molecule) to provide greater sustained expansion of Tregs to treat cGvHD that has not responded to traditional steroid treatment.

    Relapse Therapy and Prevention

    Although allogeneic HSCT is a potential curative therapy for patients with advanced hematologic cancers, disease relapse is the most frequent cause of treatment failure. Our team is pursuing novel strategies in immunotherapy and vaccine therapy to induce or restore curative GvT activity post-transplant.

    • 20-336 - Enhancing Anti-Tumor Response and Managing GVHD with T-reg Enriched Cells
      This pilot/phase 1 study for patients with relapsed AML or MDS explores the use Treg-enriched cells in haploidentical stem cell transplantation without post-transplant immune suppression to enhance graft-vs-leukemia effect while maintaining graft-versus-host disease control.
    • 18-283 – Adding Venetoclax to Transplant Conditioning
      This clinical trial aims to improve the ability of standard reduced-intensity conditioning (RIC) treatment (e.g. fludarabine plus busulfan chemotherapy) to further deplete cancer cells by adding a targeted agent, venetoclax, to the regimen. In lab studies, venetoclax has increased the anti-tumor effects of the RIC therapy. In this study, our goal is to use venetoclax to help eliminate remaining or residual cancer and reduce the risk of relapse in high-risk MDS or AML patients.
    • 19-142 – Treg Depleted DLI Plus Ipilimumab
      This study takes a novel approach to manipulating donor lymphocyte infusion (DLI) – a common therapy used to treat AML and MDS relapse after HSCT – by depleting the DLI product of immune suppressive Treg cells and combining it with an immunotherapy medication (ipilimumab), each of which has been shown to be effective in treating patients who relapse after transplant. We hypothesize that the combination therapy will be safe and will enhance our ability to cure patients with myeloid disease relapse after HSCT.
    • 19-265 – CIML NK Cell Therapy with IL-2
      This innovative study uses cytokine-induced memory-like (CIML) NK cells in combination with IL-2 to activate an anti-leukemic response in patients with AML, MDS, or MPN who have relapsed after haploidentical HSCT. Earlier studies have shown NK cells to be effective in fighting myeloid diseases, but only for short durations. Our goal is to use CIML NK cells and IL2 to help sustain the NK cell activity against the AML, MDS, or MPN.

    Infectious Disease and Supportive Care

    An estimated 75 percent of patients who undergo stem cell transplantation each year develop an infectious complication. Our team has many active studies to prevent and address such complications, including the one described below targeting cytomegalovirus (CMV), the most common viral infection in transplantation.

    • 18-348 – Treating Cytomegalovirus
      CMV is associated with end-organ disease and poorer outcomes in transplant patients. In 2017, based on research by our team, the FDA approved letermovir as a preventive therapy for CMV in allogeneic stem cell transplant patients who are CMV seropositive (who have evidence of CMV infection prior to stem cell transplant). This study will evaluate using letermovir for treatment of active CMV infection, an area of unmet clinical need given the lack of effective non-toxic CMV treatment options for treatment.
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