Hematological Malignancies (Blood Cancer) Program


Premier Research Program

Program Leader(s): Edward Stadtmauer, MD and Nancy Speck, PhD

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 transplants 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.
  • Our doctors designed and oversaw trials sponsored by the National Cancer Institute and conducted by major centers nationwide. The results of these clinical trials prompted major changes in practice that reverberated throughout the nation.
  • In 1992, the Program was designated a Research Center for the International Bone Marrow Transplant Registry/Autologous Blood and Marrow Transplant Registry (now know as the CIBMTR). Our faculty hold high-level administrative positions in this NIH funded international research effort.
  • In 1999, the Program was awarded the designation of Transplant Center for the National Marrow Donor Program opening the potentially lifesaving therapy of bone marrow transplant to patients without sibling donors.
  • The Lymphoma Program started in 1974, under the direction of the former Abramson Cancer Center Director, John Glick, MD, is noteworthy for the national influence of the treatment of both Hodgkin’s Disease and Non-Hodgkin’s Lymphoma throughout the last two decades through his leadership and direction of numerous clinical trials.
  • The Lymphoma Program pioneered the use of innovative diagnostic procedures such as the positron emission tomography (PET) in the diagnosis and treatment planning in lymphoma.
  • We pioneered the use of chemotherapy (4HC) purged bone marrow transplantation both for non-Hodkin lymphoma (NHL) and acute myeloid leukemia (AML).

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.
  • In 2001, the Transplant Program was named by the National Institutes of Health as one of the original 14 Core Clinical Transplant Centers in the NHLBI-NCI funded Blood and Marrow Transplant Clinical Network. This national network of top Transplant Centers work together to develop and conduct clinical trials in order to evaluate innovations in transplant and improve the care of patients. Penn researchers have had key roles in the administration, protocol development and conduct of clinical trials performed within the network. Penn is among the top 3 centers for enrollment on these trials
  • In 2002, The Program was accredited by the Foundation for the Accreditation of Cellular Therapy (FACT) for autologous and allogeneic bone marrow and peripheral blood progenitor cell transplantation, including collection and laboratory processing. FACT accreditation is a voluntary process, which demonstrates the highest quality medical and laboratory practice for stem cell transplantation programs. The Program was the first adult center in the Delaware Valley to show such distinction in both quality of patient care and research. Our continued excellence was recognized by renewed accreditation in 2005.
  • The Program was a pioneer in the use of chemotherapy (4HC) purged bone marrow transplantation both for AML and NHL. This served as a basis of 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.
  • In 2002, the Abramson Cancer Center was one of three recipients worldwide of the prestigious Specialized Center of Research Award from the Leukemia and Lymphoma Society to support ongoing research to design the next generation therapies for leukemia and lymphoma.
  • Numerous clinical trials were conducted or initiated by Penn, innovating the field of autologous stem cell transplant in general by introducing such techniques as blood stem cell transplant, CD34+ selection and tandem transplants. 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.
  • The Lymphoma Program, under the direction of Stephen J. Schuster, MD, is one of the largest programs in the Delaware Valley.
  • A major focus of the Lymphoma research program is the development of immunologic approaches to the therapy for lymphoma. Pivotal clinical trials investigating combinations of monoclonal antibodies such as Rituximab and Epratuzumab, Rituximab and Galiximab, and radioimmunotherapy using Epratuzumab, Bexxar and Zevalin have been completed within the Program.
  • Dr. Schuster currently leads the largest NCI initiated multi-institutional trial investigating patient specific vaccination as therapy for follicular lymphoma. He has also developed strategies to decrease the immune suppressive toxicities of conventional therapies for lymphoma utilizing infusion of activated autologous T-cells.
  • Other areas of active investigation include pharmacogenomic analyses of the response to monoclonal antibody therapy and mechanisms of enhancement of cytotoxicity of monoclonal antibodies.
  • In 2001, the Transplant Program was named by the National Institutes of Health as one of the original 14 Core Clinical Transplant Centers in the NHLBI-NCI funded Blood and Marrow Transplant Clinical Network. This national network of top Transplant Centers work together to develop and conduct clinical trials in order to evaluate innovations in transplant and improve the care of patients. Penn researchers have had key roles in the administration, protocol development and conduct of clinical trials performed within the network. Penn is among the top 3 centers for enrollment on these trials.
  • Since the mid 1990’s, Multiple Myeloma has become the most common indication for autologous transplant in North America. This is reflected in our clinical and research activity. 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, the results of which were published in 2005, 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 seminal finding has the potential to lead to tumor specific vaccine therapies for many different diseases.

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.