FYI from the NHLBI Index
September 2011: Vol. 12, Issue 2
12th Annual PIO Meeting Recap
The NHLBI held its Twelfth Annual Public Interest Organization (PIO) meeting on Monday, May 23, and Tuesday, May 24, 2010, in Bethesda, Maryland.
The meeting opened with a presentation by Dr. Lawrence A. Tabak, Principal Deputy Director, NIH. He discussed the NIH budget, noting that
despite the doubling of the NIH budget between Fiscal Years 1998 and 2003, the NIH’s purchasing power has declined to about what it was in 2001
due to subsequent flat budgets and inflation.
Dr. Tabak briefed the audience on creation of the new National Center for Advancing Translational Sciences (NCATS). The purpose of
NCATS is to improve and catalyze the development, testing, and implementation processes for diagnostics and therapeutics. The NCATS will
facilitate, but not duplicate, other translational research activities supported by the NIH, complement the private sector, and reinforce
commitment of the NIH to basic research.
The program continued with a presentation by Dr. Susan B. Shurin, Acting Director of the NHLBI. She discussed components of the
NHLBI strategic plan, specifically how the Institute’s priorities are driven by scientific opportunities and public health needs; how
basic, clinical, and population research complement each other; and how data from population studies are informing basic science and
clinical translational research.
Dr. Shurin also discussed participation by the NHLBI in the NIH Therapeutics for Rare and Neglected Diseases (TRND) Program, a Congressionally
mandated effort to speed development of new drugs for rare and neglected diseases that is a collaboration between NIH-intramural and extramural
labs with appropriate expertise. Projects can enter TRND at a variety of developmental stages and will be taken to the appropriate phase needed
for external organizations to adopt them for clinical development. It will not duplicate PhRMA (Pharmaceutical Research and Manufacturers of
America) projects, but will encourage creative partnerships and novel approaches to intellectual property rights.
Following Dr. Shurin’s presentation, PIO representatives had the opportunity to meet NHLBI extramural staff members. This portion of the
meeting has always been well received, but this year’s version exceeded all expectations because of a new twist: each PIO representative was
individually matched to a specific NHLBI staff member with expertise in the disease relevant to the PIO’s mission.
Next was a presentation about the NIH Undiagnosed Diseases Program (UDP). The first speaker in this session was Dr. Cynthia Tifft,
Deputy Clinical Director of the National Human Genome Research Institute. She discussed the UDP, which is a trans-NIH effort focused
on diagnosing the most puzzling medical cases referred to the NIH Clinical Center, and highlighted some of the program’s intriguing cases.
For more information on the UDP, visit http://www.genome.gov/27544402.
Following Dr. Tifft’s remarks, Dr. Robert Balaban, Scientific Director, NHLBI, discussed some of the NHLBI intramural research efforts
related to rare and undiagnosed diseases.
A session on dietary supplements was presented by Dr. Paul Coates, Director of the NIH Office of Dietary Supplements, and Ms.
Caroline Kruse, Executive Director of the Platelet Disorder Support Association.
Dr. Coates gave an overview of his office’s mission, which is to strengthen knowledge and understanding of dietary supplements in
order to enhance quality of life and health for the U.S. population by evaluating scientific information, stimulating and supporting
research, disseminating research results, and educating the public. The Office of Dietary
Supplements web site is at http://ods.od.nih.gov/.
Ms. Kruse concluded the session on dietary supplements by discussing immune thrombocytopenia (ITP) and the complementary therapies
frequently used by patients with ITP and other platelet disorders. For more information
about PDSA, please visit http://www.pdsa.org/.
The final session, a discussion of collaboration in sharing resources and information, was presented by Mr. James O’Leary, Chief
Innovation Officer of Genetic Alliance, Dr. Steve Groft, Director of the NIH Office of Rare Diseases Research (ORDR), and Ms. Audrey
Gordon, President and Executive Director of the Progeria Research Foundation.
Mr. O’Leary discussed the activities of his organization, a nonprofit health advocacy organization committed to transforming
health through genetics and promoting an environment of openness centered on the health of individuals, families, and
communities. Genetic Alliance’s network includes more than 1,000 disease-specific advocacy organizations, as well as
thousands of universities, private companies, government agencies, and public policy organizations. For more information
about Genetic Alliance, visit http://www.geneticalliance.org.
Dr. Groft spoke about collaboration between patient advocacy groups, industry, regulatory agencies, and others in the successful
development of therapeutics for rare diseases. He also briefed the audience on the International Rare Diseases Research Consortium,
which hopes to develop 200 new therapies for rare diseases by 2020 and develop diagnostic tests for most rare diseases. For more
information, visit http://rarediseases.info.nih.gov/.
Ms. Gordon spoke about the progress made in understanding progeria and described her foundation’s successful efforts in building a
patient registry to enhance research efforts. For more information about the Progeria
Research Foundation, visit
This year’s event drew participants from 20 states and the District of Columbia. In attendance were 81 PIO representatives,
representatives of two professional societies, and one National Heart, Lung, and Blood Advisory Council member.
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