Clinician-to-Clinician Update Clinician-to-Clinician Update

New Clinical Trials Aimed at Improving Bone Marrow Transplant Results

April 2015

Cancers originating in the bone marrow often require blood or marrow transplants (BMT) to cure patients with advanced disease. University of Minnesota Cancer Care is invested in developing new approaches, combining cellular and molecular therapy, with existing treatment options to help improve survival rates for these patients.


For patients with high-risk MDS:

An active Phase II clinical trial at University of Minnesota Medical Center, led by Erica Warlick, MD, shows promise in using cellular therapy to treat patients with high-risk myelodysplastic syndromes (MDS). Patients in this trial receive Decitabine, a hypomethylating agent widely used as standard care to treat MDS, in combination with Vorinostat, a histone deacetylase inhibitor. Interleukin-2 (IL-2) activated natural killer (NK) cells from a haploidentical (partially-matched) family donor are then given to the patient—all planned as outpatient therapy. The goal of this trial is to improve blood counts, reduce transfusion needs and achieve remission in MDS patients more efficiently than standard therapies alone. Indeed, patients have already responded to this treatment with cytogenetic remission, stable disease, and one in complete remission. Similar studies using combination therapy with the hypomethylating agent, azacitidine, are scheduled to open soon.

For patients with acute leukemia:

University of Minnesota Cancer Care has been a national leader using Natural Killer (NK) cell therapy in patients with AML. Researchers, led by Jeff Miller, MD, have been investigating NK cells in different stage of AML treatment, including patients whose disease is refractory/resistant to therapy or for patients who achieve remission but cannot tolerate more chemotherapy. Other cellular therapies, including the use of genetically engineered T-cells will soon be in clinical trials.

In addition, studies for elderly patients with AML will open to patients who do not benefit much from standard therapies. Today, cellular therapies, are also being investigated not only for leukemia therapy but also for supportive therapy when anti-leukemia therapy is given. One example is a Phase II cellular therapy trial for leukemia patients, which showed promise after completing a Phase I national study. Patients in this trial will receive a transplant of unrelated donor myeloid progenitors following induction chemotherapy, with the expectation that these cells will mature and improve patient’s neutrophil counts for 10-14 days, thereby reducing risks of infection. If successful, it will limit post-transplant infection risks and increase the safety of leukemia treatment, allowing more patients to move on to an allogeneic BMT.

Recent recognition of novel molecular and genetic abnormalities is also advancing leukemia research. Mutations in the FMS-like tyrosine kinase 3 (FLT-3) gene are present in over 30% of all acute myeloid leukemia (AML) patients and are associated with poor prognosis. University of Minnesota Cancer Care, through the work of Celalettin Ustun, MD, is involved in a national study investigating the effects of FLT-3 inhibitors on leukemia patients who are unresponsive to conventional chemotherapy. These targeted inhibitors provide a better safety profile than traditional chemotherapy and may allow patients with refractory AML, who are not responding to chemotherapy, the opportunity to achieve the remission necessary for a successful and potentially curative BMT. Future studies will focus on the use of these FLT-3 inhibitors as maintenance therapy to prevent relapse in patients, even after BMT.

Search for all current, open clinical trials

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