Download For Immediate release - Aplastic Anemia and MDS International

For Immediate Release:
Contact: Alicia Moran, 410-867-0242
Aplastic Anemia and MDS International Foundation (AA&MDSIF) to Be
Patient Support Group for Bone Marrow Failure Clinical Research Center
Cleveland Clinic Receives NIH Grant to Coordinate Center, Seek New Approaches
To Treatment; AA&MDSIF Medical Board Chairman Selected as Principal Investigator
(Annapolis, MD ) – November 5, 2003 – The Cleveland Clinic and Jaroslaw P.
Maciejewski, MD have been awarded $4.5 million over 5 years to create a Bone Marrow
Failure Clinical Research Center. The AA&MDSIF will serve as the patient group
supporting the Center’s activities and will lead efforts to educate patients and health care
professionals about treatment options.
“We are proud to be a part of this five-year study and to lend support to this effort to
help find better preventions, treatments and cures for these bone marrow failure
disorders,” said Bob Carroll, President of AA&MDSIF and a 14-year MDS survivor.
“For more than 20 years our foundation has been working with the medical community
and the patient community to create awareness and improve treatments. The Center’s
creation and our collaborative efforts should lead to major advances in fighting bone
marrow failure diseases.”
The mission of the Bone Marrow Failure Clinical Research Center will be to study
rare bone marrow disorders and identify new approaches to treatment. Dr. Maciejewski,
who will be the principal investigator on the grant, is Chief of the Hematopoiesis and
Experimental Hematology Section at the Cleveland Clinic and serves as chairman of the
AA&MDSIF medical advisory board.
Bone marrow failure syndromes and cytopenias are a group of serious diseases that
include aplastic anemia, myelodysplastic syndrome (MDS), paroxysmal nocturnal
hemoglobinuria and single lineage cytopenias (e.g., large granular lymphocyte leukemia,
pure red cell aplasia). Despite the progress achieved in recent decades, the mortality rate
for these diseases remains high. Some of these diseases are still incurable and result in
chronic morbidity throughout the remainder of the patients’ life.
“The low prevalence of bone marrow failure syndromes, has inhibited progress in
medical care of these patients, making it difficult to conduct systematic clinical studies
that address their treatment, natural history, epidemiology and specific causes” said Dr.
Maciejewski. “Yet, the importance of these diseases is far greater than their limited
prevalence would suggest, because they represent unique models to study stem cells,
immunity and defects in human DNA.”
“The creation of such a dynamic collaboration has been a vision of our Foundation
and thousands of patients for several decades, “said Marilyn Baker, Executive Director of
the AA&MDSIF. “We are grateful to Dr. Maciejewski, the Cleveland Clinic, and NIH
for making this important consortium a reality. We are honored to have been asked to
play such a vital role in this significant scientific endeavor and know that together we
will be successful in furthering patient care and medical research.”
The Center, with support from AA&MDSIF, will conduct laboratory and clinical
research directed towards improvement of patient care including, longitudinal
observational and therapeutic trials, which would not be possible in a single, center
setting. The award will also provide recourses to facilitate access of patients to
specialized care and to clinical research studies and enhancing the availability,
accessibility, and quality of information to patients and their physicians. Moreover, the
new center at the CFF will develop systematic and uniform diagnostic tests and apply
data mining tools to promote a new understanding of bone marrow failure syndromes.
The other institutions collaborating with the Clinic include the Division of Hematology
and Oncology at UCLA, Los Angeles, the Penn State Cancer Institute, University of
Pennsylvania in Hershey and the H. Lee Moffitt Cancer Center and Research Institute at
the University of South Florida Tampa, FL, and the National Institutes of Health.
In response to a recent Congressional mandate, the National Institutes of Health
(NIH) has made a $51 million, 5-year commitment to fund the Rare Disease Clinical
Research Network, which will expand clinical research across a broad spectrum of rare
diseases. The Bone Marrow Failure Clinical Research Center is one of seven centers
receiving grants to focus on a specific medical area in which more research is needed. All
centers were required to integrate the resources and perspectives of a non-profit voluntary
patient advocacy group as part of their grant application and 5-year plan.
Approximately 25 million people across the United States are affected by an
estimated 6,000 rare diseases or conditions. In addition to the Bone Marrow Failure
Clinical Research Center, other centers in the NIH Rare Disease Research Network will
be studying urea cycle disorders; rare lung conditions; nervous system disorders; rare
genetic steroid disorders; and vasculitis.
The Aplastic Anemia and MDS Foundation is a non-profit patient advocacy group
founded in 1983. Its mission is to serve as a resource for patient assistance, advocacy, and
support; provide educational materials and medical information; and to fund medical
research to find effective treatments and cures for Aplastic Anemia, Myelodysplastic
Syndromes, and related bone marrow diseases such as PNH. The Foundation’s is
headquartered in Annapolis, MD. Its website is www.aamds.org
The Cleveland Clinic Foundation, located in Cleveland, Ohio, is a not-for-profit
multi-specialty academic medical center that integrates clinical and hospital care with
research and education. U.S. News & World Report consistently names The Cleveland
Clinic as one of the nation’s best hospitals in its annual “America’s Best Hospitals”
survey. The Cleveland Clinic website is www.clevelandclinic.org.