Bone Marrow Transplant and Stem Cell Transplant Program

Premier Research Program

Bone Marrow and Stem Cell Transplant research at Penn is accomplished though our NCI approved and funded Hematologic Malignancies Program

Program Leader(s): Alan Gewirtz, MD and Edward Stadtmauer, MD

Since Penn's discovery of the Philadelphia Chromosome, which revolutionized cancer treatment, we've had a history of groundbreaking research.

Every day our scientists learn more about the causes of hematologic malignancies (leukemia, lymphoma and myeloma) , as well as, better ways to prevent and detect them. Penn continues to be on the cutting edge in the development of new therapies and the use of bone marrow and stem cell transplant to treat these cancers.

All of which can make a difference to patients and their families, both today and in the future.

Making History

  • Started in 1987, the autologous (an individual's own marrow saved before treatment) transplant program has been under the direction of Edward Stadtmauer, MD since 1992.
  • We pioneered the use of chemotherapy purged bone marrow transplantation both for acute myeloid acute myeloid leukemia (AML) and non-Hodgkin lymphoma (NHL). This served as the basis for major national clinical trials comparing autologous stem cell transplant to conventional chemotherapy for AML. This led to the use of autologous transplant for AML as the standard of care.
  • By the mid 1990's, the primary indication for autologous transplant was high risk and metastatic breast cancer. Though ultimately very beneficial for patients with other diseases, the use of stem cell transplant for breast cancer did not result in incremental survival over conventional dose therapies. A high priority national clinical trial lead by Penn has held up as a model for other comparison trials and altered the course of breast cancer therapy throughout the nation and the world.
  • Penn investigators have been at the forefront of surmounting the barrier to successful donor transplant from graft-versus-host disease and transplant related morbidity. Throughout the 1990's, Penn clinicians were pioneers in the revolution regarding the availability of alternate donors both from mismatched sibling and unrelated volunteers as well as the demonstrated efficacy of blood derived stem cell transplant and the use of less toxic non-myeloablative preparatory regimens which have markedly improved the outcome and decreased the toxicity of allogeneic transplant.

Shaping the Future of Cancer Care

  • With over 50 available clinical trials, at any given time, our active clinical trials research program includes trials sponsored by national cooperative groups, industry and grants.
  • Penn investigators pioneered the use of donor lymphocyte infusions as adoptive immunotherapy for patients with poor prognosis or relapsed malignancies. The technique of activated donor T-cell infusions developed at Penn is under investigation. Less toxic non-myeloablative regimens have been developed and are showing great promise in a number of malignancies. Non-myeloablative regimens are allowing patients who otherwise would not be eligible for allogeneic transplant to successfully undergo the procedure.
  • Since the mid 1990's, Multiple Myeloma has become the most common indication for autologous transplant in North America. Penn investigators have been in the forefront of the study of this modality in myeloma including CD34+ purging, adoptive immunotherapy with activated autologous T-cell infusions and the development of comparative trials of autologous and non-myeloablative allogeneic transplant for Multiple Myeloma. In a clinical trial, Penn investigators demonstrated for the first time the ability of activated T-cell infusions to re-establish immune function after transplant for Multiple Myeloma. This decisive finding has the potential to lead to tumor specific vaccine therapies for many different diseases.
  • Penn investigators helped design and are active participants in national trials comparing the non-myeloablative approach to autologous transplant in Multiple Myeloma and Non-Hodgkin's Lymphoma. This work has lead the Leukemia and Lymphoma Society to fund a prestigious five year multi-million dollar Specialized Center of Research Award to investigators within the Program to further these innovative trials.
  • The next few years will include investigation into novel approaches to further improve aspects of autologous transplant such as stem cell mobilization, detection and reduction of minimal residual disease and further reduction in the toxicity of this procedure to further benefit our patients.
  • A number of our junior faculty were recently awarded Leukemia and Lymphoma Society career development awards designed to foster the next generation of leaders in this field.

As one of the nation's largest cancer research centers our investigators benefit each day from collaborations and interactions with over 400 scientists and physicians involved in the Abramson Cancer Center.