Blood and Marrow Transplant Research Program

The Blood and Marrow Transplant Research Program within the Mayo Clinic Transplant Research Center focuses on improving outcomes of blood and marrow transplantation for patients by making the procedure safer, reducing the risk of relapse after transplant, and proactively managing long-term health issues.

Physician-investigators in the Blood and Marrow Transplant Research Program are leaders in blood and bone marrow transplant research, with expertise in graft-versus-host disease (GVHD), long-term follow-up, managing wellness, new clinical protocols and care delivery.

Research focus areas

The Blood and Marrow Transplant Research Program has several research focus areas, including:

  • Allogeneic transplant
  • Graft-versus-host disease
  • Alternative donor approaches
  • Prevention of infectious complications
  • Management of long-term issues
  • Immune-based therapies
  • Disease recurrence
  • Immunoglobulin light chain (AL) amyloidosis and POEMS syndrome

Here's a closer look at the research focus areas.

Allogeneic transplant

In the realm of allogeneic transplant, program investigators have performed studies assessing the role of family donors who are less than full immunologic matches with their family members to help extend lifesaving transplants to patients who would otherwise lack a suitable donor.

For myelofibrosis, investigators are pioneering a new form of allogeneic transplant called reduced-intensity conditioning transplant (sometimes called minitransplants), which has the potential for cure. Mayo Clinic is an internationally recognized center of excellence for the treatment of patients with myelofibrosis.

Graft-versus-host disease

The Blood and Marrow Transplant Research Program is participating in a variety of research studies related to graft-versus-host disease (GVHD), a potentially life-threatening complication of allogeneic stem cell transplant.

These include studies to:

  • Identify patients at the highest risk of developing severe GVHD
  • Refine the staging of acute GVHD in a multi-institutional setting to permit higher quality clinical trials
  • Examine a pre-emptive approach using biomarkers and intervention with such drugs as natalizumab
  • Develop novel treatment approaches of GVHD
  • Test established and state-of-the-art GVHD treatment regimens, such as anti-thymocyte globulin (ATG), post-transplant cyclophosphamide, CD34 lymphocyte depletion, proteasome inhibition (bortezomib) and antiretroviral agents (maraviroc)

Alternative donor approaches

Given that a significant number of patients, especially those from ethnic minorities, are unable to find matched donors, there's increasing interest in exploring alternative sources for donor cells.

The Blood and Marrow Transplant Research Program at Mayo Clinic has an active cord blood program that allows the use of umbilical cord blood stem cells for transplant. The team is increasingly performing transplants using haploidentical donors and has ongoing clinical trials comparing this approach to traditional transplant approaches.

Prevention of infectious complications

The transplant program team is examining vaccination approaches to reduce the burden of post-transplant infectious complications, such as cytomegalovirus.

Management of long-term issues

Transplant survivors can develop unique complications, including GVHD, neurocognitive defects, increased risk of certain cancers and psychosocial issues. Researchers at Mayo Clinic are investigating ways to reduce such long-term complications.

Immune-based therapies

The Blood and Marrow Transplant Research Program is investigating ways to enhance the immune function of patients after autologous transplant to help reduce recurrence of disease.

These include studies for:

  • Vaccination strategies
  • Modifying T cells to fight various cancers
  • Using new drugs that can enhance the immune surveillance of the body by restoring the function of the immune cells

Disease recurrence

Studies have determined that lymphocytes known as natural killer cells are the most important cells in reducing the risk of disease recurrence after transplant. This phenomenon has been shown to be the most powerful predictor of outcome after autologous transplant and has been shown to apply to each of the hematologic malignancies for which patients undergo transplant.

The more rapid recovery of these cells after transplant seems to relate to the number of natural killer cells contained in the peripheral blood stem cell product collected for transplant. Altering the way in which peripheral blood stem cells are mobilized may enhance the ability to collect these natural killer cells.

Ongoing laboratory research in animal models will allow Mayo Clinic investigators to unravel the mechanisms by which these natural killer cells function to prevent tumor recurrence. Clinical trials are examining how the proportion of these immune cells can be enhanced in stem cell collections, thus increasing the recovery of the immune cells after transplant and potentially improving outcomes.

Immunoglobulin light chain (AL) amyloidosis and POEMS syndrome

The Blood and Marrow Transplant Research Program at Mayo Clinic is a pioneer in the use of autologous peripheral blood stem cell transplant for the treatment of immunoglobulin light chain (AL) amyloidosis and POEMS syndrome, both of which are relatively rare plasma cell disorders.

Immunoglobulin light chain (AL) amyloidosis and POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal plasma-proliferative disorder, and skin changes) are progressive fatal illnesses for which no other effective therapy currently exists. Studies at Mayo Clinic have shown that autologous transplant can extend survival in patients with these diseases.

Program faculty

Here's a list of faculty in the Blood and Marrow Transplant Research Program by campus location.