NHLBI Announcement: Institute to Realign its Sickle Cell Disease Research Program
March 10, 2008
The
National Heart, Lung, and Blood Institute (NHLBI) announced today a
comprehensive and innovative restructuring of its research program in sickle
cell disease (SCD).SCD is a serious
inherited blood disorder that most commonly affects people with origins in
Africa, Latin America, the Middle East, or the Mediterranean.Since 1972, the NHLBI has developed and
maintained a major research effort to improve the lives of individuals with SCD.The studies have led to a number of effective
approaches for the management and treatment of the disease, and today’s
patients live longer and have a better quality of life than was the case in
previous generations.However, there is
no cure for SCD, and therapies that exist do not benefit all patients.
The recent issuance of the Institute’s Strategic Plan (http://apps.nhlbi.nih.gov/strategicplan/)
and the scheduled renewal of the Comprehensive Sickle Cell Centers (CSCC)
program provided an opportune time to rigorously assess the NHLBI program
in SCD.The National Heart, Lung, and Blood Advisory Council conducted
an extensive review of the Institute’s research and training portfolio
in SCD, taking into account responses received to a public solicitation
for input from patients and lay and professional constituencies about
the top scientific and clinical priorities.A detailed report on the Council’s
findings and deliberations can be found at http://www.nhlbi.nih.gov/resources/docs/scd_program.htm.
Recommendations include the following.
- Basic
science — Research should focus on disease mechanisms; new treatment
approaches; genes and genetics; molecular biology and biomarkers; vascular
biology; erythropoiesis and red blood cell biology; and animal models.Progress in this area will require
stimulation of investigator-initiated research applications, with careful
attention to appropriate peer review, and expanded involvement of scientists
from areas other than hematology.
- Translational
and clinical research — Emphasis should be placed on pain pathophysiology and
management; fetal hemoglobin induction; trials of promising alternatives to
hydroxyurea; approaches targeting disease pathophysiology; curative therapies;
and prediction, prevention, and management of severe manifestations and
end-organ damage.The NHLBI should provide
research resources such as databases for genotype–phenotype data and
repositories for biological samples, encourage resource-sharing among
investigators, and assist investigators in bringing clinically relevant basic
science discoveries to the point where they are ready for human trials.
- Participation
in clinical research — The Institute should increase the pool of potential
participants for interventional and observational studies and to provide
opportunities for geographically broad participation by individuals and investigators
in research.
- Translation
and dissemination to the community — Timely and thorough application of research
findings should be promoted through development of evidence-based practice
guidelines and their widespread dissemination via educational programs for
physicians, other health-care providers, and patients.
Based on the NHLBAC
recommendations, the NHLBI is moving forward with the following innovations to
its SCD portfolio.
- Support
for basic research will be expanded through funding of investigator-initiated
grant applications and through NHLBI-initiated RFAs (requests for applications)
focused on the pathophysiology of SCD, the biology of pain in SCD, fetal
hemoglobin switching, and genetic modifiers of disease expression and
progression.
- The Institute
will reconfigure the CSCC program into a Basic and Translational Research
Program by funding meritorious projects submitted in response to the recent RFA
(http://grants.nih.gov/grants/guide/rfa-files/RFA-HL-06-008.html).The
program will emphasize fundamental investigations and their translation into
initial studies in humans, as well as community translation to promote
evidence-based clinical practice.SCD
Scholars programs for the career development of young investigators and
Summer-for-Sickle-Cell-Science programs for research training and mentoring of
high-school students also will be supported as part of a larger effort by the
Institute to prepare the next generation of scientists to advance the field of
SCD research.
- The
NHLBI has requested comments from its constituency regarding interest in
joining the RAID (Rapid Access to Interventional Development) program of the
National Cancer Institute, which provides contract services to aid in the
translation to the clinic of potential new therapeutic agents originating in
academia (http://grants.nih.gov/grants/guide/notice-files/NOT-HL-08-111.htm).The
RAID offers an avenue for swift progress in evaluation of promising therapeutic
approaches such as innovative drugs to stimulate fetal hemoglobin production.
- The
NHLBI will develop a new Clinical Trials Research Network (CTRN) designed to
open participation in clinical research to a much larger number of
investigators and individuals with SCD than is currently possible.Structured similarly to the Children’s
Oncology Group (http://www.childrensoncologygroup.org/, which offers clinical trial participation
to all eligible subjects and investigators, the CTRN will, in time, subsume the
current SCD Clinical Research Network (http://grants.nih.gov/grants/guide/rfa-files/RFA-HL-05-006.html).
- The
NHLBI will expand its support of genomic research in SCD beyond the boundaries
of current efforts being conducted within the framework of the CSCCs (http://grants.nih.gov/grants/guide/rfa-files/RFA-HL-05-006.html) by developing a program like the Framingham
SHARe (SNP Health Association Resource— http://www.ncbi.nlm.nih.gov/projects/gap/cgi-bin/study.cgi?study_id=phs000007.v2.p1) with contributions of genotypic and
phenotypic data from many investigators and their patients and free access to
qualified researchers.
- Finally,
the Institute will undertake a focused effort to develop evidence-based
guidelines for the care of individuals with SCD across the life-span that can
be used by health-care practitioners throughout the world.An educational campaign will be launched, in
partnership with the Sickle Cell Disease Association of America and other
patient advocacy groups and professional organizations, to raise awareness
about SCD and bring nationwide attention to its diagnosis and treatment.
By
implementing these recommendations, the NHLBI intends to take advantage of
existing scientific opportunities and make SCD resources more widely available,
better serving both the SCD research and the patient communities.
Inquiries may be
directed to:
Susan B. Shurin,
M.D.
Deputy Director
National Heart, Lung, and Blood Institute
Building 31, Room 5A48
9000 Rockville Pike
Bethesda, MD 20892- 2486
Telephone: (301) 496-1078
Fax: (301) 402-0818
Email: Shurinsb@nhlbi.nih.gov
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