Although many patients are diagnosed with earlier phases (also known as "precursor conditions") of blood cancers, in most cases, there are no effective, disease-modifying therapies available. This means that most patients do not receive treatment until their cancer progresses.
Precursor conditions include early cases of chronic lymphocytic leukemia called monoclonal B cell lymphocytosis (MBL); early cases of multiple myeloma or Waldenström's Macroglobulinemia called monoclonal gammopathy of undermined significance (MGUS); and early cases of myelodysplastic syndrome and myeloproliferative disorders.
In order to understand why some patients with these precursor conditions go on to develop progressive disease, while others do not show progression — and to develop better targeted therapeutic agents to prevent progression, or even eliminate the disease before it leads to symptoms — the Dana-Farber Cancer Institute/Brigham and Women's Cancer Center (DF/BWCC) Blood Cancer Prevention of Progression Clinic (BCPC) has been created.
Although the last decade has seen the development of effective target therapies for patients with blood cancers, their clinical use has been hampered by drug resistance, clonal evolution, and disease progression — all of which limit the extent and duration of patients' response.
Of note, most blood cancers — including multiple myeloma, chronic lymphocytic leukemia, and acute myeloid leukemia — are preceded by clonal disease states that then progress into disease. However, the genomic, genetic, and epigenetic factors governing the process of progression and therapeutic resistance have not been systematically characterized.
The BCPC is a research initiative focused on understanding the progression and clonal evolution of blood cancers, with an ultimate aim to develop targeted therapeutic agents that can eliminate the early clones of disease.
Robert Soiffer, MDCo-Principal Investigator, Blood Cancer Prevention of Progression ClinicChief, DF/BWCC Division of Hematologic Malignancies and Co-Chief, DF/BWCC Adult Stem Cell Transplantation ProgramProfessor of Medicine, Harvard Medical School
Dr. Soiffer graduated from New York University School of Medicine in 1983, and trained in internal medicine at Brigham and Women's Hospital, where he also was chief medical resident.
He joined Dana-Farber/Brigham and Women's Cancer Center (DF/BWCC) in 1988, after completing a medical oncology fellowship. Dr. Soiffer has served as vice president (2006), president (2007), and immediate past president (2008) of the American Society of Blood and Marrow Transplantation.
Irene Ghobrial, MDCo-Principal Investigator, Blood Cancer Prevention of Progression ClinicDirector, Michele & Stephen Kirsch Laboratory, Dana-Farber Cancer InstituteMedical Oncologist, DF/BWCC Jerome Lipper Multiple Myeloma CenterAssociate Professor of Medicine, Harvard Medical School
Dr. Ghobrial is a physician-scientist who specializes in early precursor conditions of multiple myeloma and Waldenström's Macroglobulinemia, specifically MGUS and smoldering diseases. Her research interests are in the area of cell trafficking and cell metastasis in B cell malignancies and the malignant bone marrow niches. She is the principal investigator of several clinical trials in both multiple myeloma and Waldenström's Macroglobulinemia.
David P. Steensma, MDCo-Principal Investigator, Blood Cancer Prevention of Progression ClinicMedical Oncologist, DF/BWCC Adult Leukemia ProgramAssociate Professor of Medicine, Harvard Medical School
Dr. Steensma's primary area of clinical activity and research focus is the myelodysplastic syndromes (MDS) and related conditions. His research activity includes development of new therapies, as well as discovery of new somatic genetic mutations important in the pathobiology of this difficult and poorly-understood group of smoldering myeloid neoplasms.
Dr. Steensma is also involved in collaborations with several laboratory-based scientists, particularly Benjamin Ebert, MD, PhD and Mark Fleming, MD, DPhil, to try to discover new pathways disturbed in MDS and related disorders – with the goal of finding pathways that can be exploited therapeutically.
Benjamin Ebert, MD, PhDCo-Principal Investigator, Blood Cancer Prevention of Progression ClinicMedical Oncologist, DF/BWCCAssistant Professor of Medicine, Harvard Medical School
Dr. Ebert's laboratory studies the biology and genetics of hematologic malignancies and other hematologic disorders. His group employs a range of genomic and proteomic technologies, as well as classical cellular and molecular biology approaches, to investigate the molecular basis of specific human diseases.
Major focuses of the laboratory are myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). Dr. Ebert identified a gene that plays a central role in the pathophysiology of MDS with deletions of chromosome 5q. These findings revealed a molecular link between del(5q) MDS and congenital bone marrow syndromes such as Diamond Blackfan anemia. The Ebert laboratory has also definied sets of mutations in MDS that are associated with various disease phenotypes, including overall survival and response to specific therapies.
Irene Ghobrial, MDJacob Laubach, MDNikhil Munshi, MDRobert Schlossman, MD
Kenneth Anderson, MDClaudia Paba-Prada, MDPaul Richardson, MD
Jorge Castillo, MDIrene Ghobrial, MD
Steven Treon, MD, PhD
Benjamin Ebert, MD, PhDR. Coleman Lindsley, MD, PhDDavid Steensma, MD
Daniel DeAngelo, MD, PhDRichard Stone, MD
Ann Mullally, MDDavid Steensma, MD
Martha Wadleigh, MD
Matthew Davids, MD
Jennifer Brown, MD, PhDCatherine Wu, MD
Ann LaCasce, MD
Jennifer Brown, MD, PhDArnold Freedman, MDCatherine Wu, MD
Benjamin Ebert, MD, PhDAric Parnes, MD
Nancy Berliner, MD
An important note: Taking part in the Blood Cancer Prevention of Progression Clinic (BCPC) research study is voluntary. Instead of being in this research study, you may continue to obtain your clinical care without participating in the study. Your decision not to participate will not affect your clinical care in any way.
The research study seeks to perform research tests on specimens obtained during the course of clinical care, bank the specimens and material derived from the specimens, and link the specimen data to clinical data. It also seeks to obtain blood and a buccal swab or mouthwash for germline genomic testing, bank this material and the DNA derived from it, share de-identified genomic data with other investigators or centralized repositories. Finally, it seeks to permit providers to see the results of these research tests and permit re-contact for potential treatment or clinical trial enrollment opportunities.
The following patients are eligible to participate in this study:
It is important to understand that this is not a clinical study being done to benefit you directly. Therefore, taking part in this research study may not directly benefit you. However, the information gained from your participation is likely to generate important information which will help future patients with blood and individuals at high risk of developing those diseases.
Blood cancers occur when the molecules that control normal cell growth are damaged. Many of these changes can be detected by directly examining parts of the cancer or cells in blood. Several alterations that occur repeatedly in certain types of blood cancers have already been identified, and these discoveries have led to the development of new drugs that target those alterations. More remain to be discovered.
The purpose of this research study is to perform these molecular analyses on your tissues (obtained from biopsies), blood, or other body fluids such as saliva or urine. Importantly, this study will use tissue specimens that have already been collected as part of your clinical care. Your tissue sample may be used to create a living tissue sample (called a "cell line") that can be grown in the laboratory. This allows researchers to have an unlimited supply of your cells in the future without asking for more samples from you.
These studies are being done to add to our knowledge of how genes and other factors affect cancer. We are gathering this knowledge by studying groups of people, and the study is not meant to test your personal medical status. For that reason we will not ordinarily give you the results of our research on your samples unless there is clear evidence of actions that could benefit your health.
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