2008-09-01
Dr. Buchanan is Children's Cancer Fund Distinguished Chair in
Pediatric Oncology and Hematology, and Director of Pediatric
Hematology-Oncology at the University of Texas Southwestern Medical
Center.
Dr. Telen is Wellcome Clinical Professor in Medicine and Chief,
Division of Hematology, as well as Director of the Duke Comprehensive
Sickle Cell Center.
Drs. Buchanan and Telen were Co-Chairs of the 2007 ASH Workshop on Sickle Cell Disease.
In an article in the July/August 2008 issue of The Hematologist,
Susan Shurin, MD, Deputy Director of the National Heart, Lung, and
Blood Institute (NHLBI), outlined the Institute's realignment of its
sickle cell research program. Based on discussions during the 2007 ASH
Workshop on Sickle Cell Disease (SCD), the Society provided several
recommendations to NHLBI regarding the development of an SCD research
agenda. ASH's emphasis focused on developing new and improved
treatments, expanding funding for all types of research, and creating a
new model for clinical trials — essentially a comprehensive,
multidisciplinary approach for SCD research. The Society was pleased to
see many of its recommendations incorporated into NHLBI's new plan and
has commended the Institute for announcing a comprehensive and
innovative restructuring of its current program.
Clearly, a new era in SCD research, education, and patient care is
beginning. NHLBI's plan is extremely encouraging, even though change is
not always easy. Regardless of how wonderful the plan, there will be
many stumbling blocks on the road to success. The changes described by
Dr. Shurin have been painful to some investigators comfortable with the
prior research infrastructure and agenda promoted by the former
Comprehensive Sickle Cell Center program and its Clinical Trials
Consortium, especially since core funding for the former sickle cell
centers is being phased out. But the pain will only be short-lived,
since the doors will now hopefully be open to new ideas and input from
investigators in diverse disciplines. The changes in NHLBI's approach
to funding for SCD research, especially insofar as it encourages
involvement of specialists in other fields, should also promote renewed
vigor within the established research community.
Some excellent research ideas were not brought to fruition in the
past due to lack of resources and excessive bureaucracy, including at
NIH. It is hoped that the new research structure proposed by Dr. Shurin
will include mechanisms to assure agile and facile review and
implementation so that the new research programs meet their objectives.
Furthermore, the need to ensure that new therapies are clinically
tested quickly and appropriately (similar to models in cardiology) is
extremely important. This will require a responsive funding system and
an inclusive network of participants.
Of critical importance to the success of this effort will be the
ability to "ramp up" efforts by other branches of NIH, other government
agencies, industry, and the SCD community. In addition to the research
perspective, it is essential to understand the overall outcomes of the
clinical care provided. ASH will continue working with NHLBI through
the transition and implementation stages of its new program and
strongly encourages its members to do the same — pursuing the common
goal of improving the science and providing excellent individualized
care for patients with SCD.
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