By George R. Buchanan, MD
2009-01-01
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.
Readers of The Hematologist who are particularly
interested in sickle cell disease will be fully aware of the recent
climate changes apparent on the sickle cell disease clinical research
weather map. ASH, recognizing that changes were in the air some time
ago, sponsored a workshop in May 2007 focusing on sickle cell disease
that I was privileged to co-chair. A number of recommendations were
made by ASH to NHLBI aimed at making it more responsive, efficient, and
inclusive by creating a new research framework. As a result of ASH’s
leadership — and with input from many other organizations and
individuals — a series of decisions regarding sickle cell disease
infrastructure was made by NHLBI during the past six to nine months.
These include termination of the Sickle Cell Disease Center Program,
creation of a new Basic and Translational Research Program (BTRP), and
continued engagement with multiple constituencies to garner advice
about future research paradigms.
All of these recent deliberations culminated in a comprehensive
NHLBI-sponsored workshop held in Bethesda on October 22-24, 2008,
titled "Clinical Priorities/Clinical Trials." The 250 or so attendees
represented the entire spectrum of the clinical and translational
investigative community. The agenda was guided in large part by the
recommendations of the prior ASH workshop on setting research
priorities for sickle cell disease. Prior to the meeting, eight
subcommittees were created to address issues related to
pulmonary/cardiac complications, renal complications, stem cell
transplantation, pain, epidemiology/patient outcomes, fetal hemoglobin,
neurology, and vascular biology/pathophysiology. Each subcommittee met
one or more times by conference call prior to the workshop to define
critical priorities, opportunities, and knowledge gaps. At the
workshop, each subcommittee chair made a brief presentation and then
breakout sessions were held to vet and fine-tune the working groups’
preliminary recommendations. The refined recommendations were then
presented to workshop participants the next day, and robust discussion
followed.
Another feature of the workshop was a series of presentations about
how other NIH-funded collaborative clinical research groups (involved
with asthma, HIV/AIDS, and cancer) execute their multi-center trials. A
special workshop highlight was keynote speaker Dr. David Dilts from
Vanderbilt’s Business School, who gave an energetic and highly
thought-provoking speech on building networks, especially in these lean
financial times.
The workshop fostered an open and constructive dialogue among
investigators and other stakeholders (including patients with sickle
cell disease and advocacy group representatives). A summary of the
workshop proceedings and findings will be published on the NHLBI Web
site.
NHLBI has now heard what the clinical research community desires and
appears ready to act on the advice that it has received to build a
foundation for future discoveries that will benefit patients with
sickle cell disease from around the world.
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