National Heart, Lung, and Blood Advisory Council October 2017 Meeting Summary

DEPARTMENT OF HEALTH AND HUMAN SERVICES
NATIONAL INSTITUTES OF HEALTH
NATIONAL HEART, LUNG, AND BLOOD ADVISORY COUNCIL

MEETING SUMMARY OF THE
NATIONAL HEART, LUNG, AND BLOOD ADVISORY COUNCIL

October 24, 2017

The 275th meeting of the National Heart, Lung, and Blood Advisory Council (NHLBAC) was convened on Tuesday, October 24, 2017, in Building 35A, the Porter Neuroscience Center Conference Center, National Institutes of Health (NIH), Bethesda, Maryland. It was open to the public from 8:06 a.m. until 12:51 p.m. Closed session began at 1:16 p.m. and adjourned at 3:17 p.m. Dr. Gary H. Gibbons, Director of the National Heart, Lung, and Blood Institute (NHLBI), presided as Chair.

Council Members attending

Dr. Dale E. Abel 
Dr. Donna K. Arnett 
Dr. James D. Crapo 
Dr. George Q. Daley 
Dr. Michael R. DeBaun
Dr. Serpil C. Erzurum 
Dr. Karen Glanz
Dr. M. Luisa Iruela-Arispe
Dr. Fernando D. Martinez
Dr. Diane J. Nugent
Dr. Sally E. Wenzel
Dr. Phyllis C. Zee 

Council Members attending via teleconference

Dr. Nancy J. Brown
Dr. Michael R. DeBaun
Dr. Kim M. Smith-Whitley
Dr. Richard S. Schofield (ex officio)

Council Members unable to attend

Dr. Bradford C. Berk
Dr. Robert C. Robbins
Dr. Pilar N. Ossorio

Public attending

Dr. Lauren Brood, American Association of Immunologists
Ms. Anne Berry, Association of American Medical Colleges
Ms. Meg Calabro, RTI International
Ms. Diane Ceban, SAIC
Ms. Michele Givens, Decision Lens
Ms. Nuala Moore, American Thoracic Society

NHLBI employees attending 

A number of NHLBI staff members were in attendance.

Other NIH Institute employees attending

Dr. Matt Gillman, NIH OD
Dr. Delia Olufokunbi Sam, CSR

OPEN SESSION

I. CALL TO ORDER AND OPENING REMARKS

Dr. Gary H. Gibbons, Director of the National Heart, Lung, and Blood Institute (NHLBI), welcomed members and other attendees and called the 275th meeting of the National Heart, Lung, and Blood Advisory Council (NHLBAC) to order.

II. ADMINISTRATIVE ANNOUNCEMENTS

Dr. Laura K. Moen, Director, Division of Extramural Research Activities (DERA), NHLBI, made the required announcements for the Council meeting, which included: that a notice of the meeting was published in the Federal Register, Council members are required to absent themselves from the room if their presence would constitute a conflict of interest, Council members may not engage in lobbying activities while attending Council meetings or sponsored events, and portions of the meeting are closed to the public in accordance with the provisions set forth in sections 552b(c)(4) and 552b(c)(6), Title 5, U.S. Code and Section 10(d) of the Federal Advisory Committee Act, as amended.

III. REPORT OF THE DIRECTOR

Fiscal Year (FY) 2018 Appropriations and Update. Dr. Gibbons reported that he accompanied Francis Collins, M.D., Ph.D., Director of the National Institutes of Health (NIH), to congressional appropriations hearings. Early draft budgets from the Senate and House of Representatives have increases for the NIH, and this support is bipartisan. The federal government is operating under a continuing resolution that maintains FY 2017 funding levels. This continuing resolution expires in December 2017.

FY 2017. Preliminary estimates are that NHLBI will have awarded 708 competing R01 awards in FY 2017, exceeding its goal of 700. Of these, 145 were for early-stage investigators (ESIs). The preliminary R01 application success rate at the NHLBI is 23 percent, one of the highest rates of all NIH Institutes and Centers. The preliminary success rate for ESIs is 29 percent. The Institute’s funding format includes a zone of consideration (range of priority scores for considering applications for funding) for meritorious R01 applications and provides opportunities to prioritize certain programmatic emphases.

Regenerative Medicine. The NHLBI is part of a trans-NIH effort, the Regenerative Medicine Innovation Project, that is funded by the 21st Century Cures Act. Congress appropriated $30 million for this project, which supports clinical research on adult stem cells, and the NHLBI is helping to develop a strategy to obtain the required matching funds from partners. Several of the initial awards, which were in the form of supplements, were relevant to the NHLBI’s interests, including studies of therapeutic revascularization by endothelial cell transplantation, idiopathic pulmonary fibrosis treatment using induced pluripotent stem cell therapy, and engineered red blood cell and platelet production. A workshop sponsored by the NIH and the U.S. Food and Drug Administration will explore the state of regenerative medicine science involving adult stem cells, with a focus on collaborative approaches to develop safe and effective products.

Chronic obstructive pulmonary disease (COPD). This is a high priority for the NHLBI, which released the COPD National Action Plan in May 2017. This plan is a call to arms for the nation to address this devastating disorder. NHLBI-sponsored studies have shown that treatment responders have differential gene signatures, and developmental pathways are key to promote self-renewal in response to tissue injury instead of pathology. Dr. Gibbons hopes that studies like these can make a difference in treating this disease.

Sickle Cell Disease. With new technologies, including gene editing and transfer, the NHLBI continues to push toward a cure. One patient at the NIH Clinical Center has undergone an innovative gene transfer process that has transformed his life. Institute staff have worked hard to explore the use of genetic therapy technologies with hemoglobinopathies as a test case. The NHLBI is putting together the elements needed to progress with curative protocols.

Trans-Omics for Precision Medicine (TOPMed). The NHLBI supports research to understand the molecular basis of chronic diseases. TOPMed has sequenced more than 120,000 whole genomes. The NIH is exploring and pilot testing this cloud-based collection of user-friendly, searchable, accessible, and interoperable resources that are protected through controlled access.

Aligning NHLBI-Solicited Science With Its Strategic Goals and Objectives. The NHLBI Strategic Vision, developed with the help of Council input, guides the science that NHLBI supports. A concrete example of this effort is the recent release of a FOA aimed at addressing gaps in the K award pipeline (Objective 8 – workforce and resources). This effort was spearheaded by Rear Admiral Helena Mishoe, who has led the Institute’s training efforts for years and is retiring. Dr. Gibbons thanked Rear Admiral Helena Mishoe for her service to the NHLBI.

V. GABRIELLA MILLER KIDS FIRST PEDIATRIC RESEARCH PROGRAM

Dr. Jonathan Kaltman, Branch Chief, Heart Development and Structural Diseases Branch, NHLBI, explained that the NHLBI initiated the Gabriella Miller Kids First Pediatric Research Program under the 2014 Gabriella Miller Kids First Research Act. The Act, which allocated $126 million to this program, is named after a girl who developed a brain tumor at age 9 and who, before dying a year later, advocated with her family before Congress for pediatric research funding.

The program’s vision is to alleviate suffering from childhood cancer and structural birth defects by fostering collaborative research to uncover the etiology of these diseases and by supporting data sharing within the pediatric research community. The program will establish a resource for genomic and clinical data from patients with cancer and/or birth defects to facilitate data sharing and stimulate collaborative research that will accelerate discovery and ultimately drive innovation in diagnosis and treatment.

Dr. Kaltman summarized the program’s activities to date and finished up by stating that Kids First will be extending its work through collaborations with the Pediatric Cardiac Genomics Consortium, funded by the NHLBI, which is identifying genetic causes of congenital heart disease. The consortium successfully applied for whole-genome sequencing on 350 trios through the Kids First program, and this sequencing is helping identify the causes of congenital heart disease.

VI. INTRODUCTION TO THE DIVISION OF INTRAMURAL RESEARCH

Dr. Robert S. Balaban, Scientific Director, Division of Intramural Research (DIR), NHLBI, described the NHLBI DIR’s mission and it’s structure. Dr. Balaban also highlighted the NHLBI Orloff Science Award, which recognizes scientific or scientific infrastructure achievements that reached fruition within the last year, with an emphasis on teamwork within the DIR. Five researchers earned the award in 2017 and the NHLBI is creating videos featuring each winner to include with previous winners’ videos currently available on the award website.

VII. ADVANCING X-RAY IMAGING FOR BIOMEDICAL APPLICATIONS

Dr. Han Wen, Senior Investigator, Laboratory of Imaging Physics, DIR, NHLBI, explained that his team focuses on using physics discoveries to develop imaging technologies. Dr. Wen’s current work focuses on x-ray wave imaging. X-rays, the oldest imaging modality in medicine, are fast, their resolution is unmatched, they are relatively easy to operate, and their costs are not too high.

However, this imaging technique has limitations. In addition to the harmful effects of ionizing radiation, x-rays lack the contrast needed to examine soft tissues, such as the internal structures of the heart. Dr. Wen is therefore using x-ray wave imaging to enhance soft tissue contrast in x-rays. This technique leverages the characteristics of waves and uses low-dose radiation to increase soft tissue contrast without using iodine, which has its own complications.

Dr. Wen is also using high-resolution imaging in humans to bring out more details than conventional computed tomography scanning. This dose-focusing technique can, for example, show the texture of a cyst and its wall, which is useful for determining the stage of lung disease.

VIII. NHLBI INITIATIVES

NHLBI staff presented 15 initiatives, all of which had been reviewed in September by the Board of External Experts (BEE), a working group of Council. First, extramural program staff develop ideas and potential initiatives, which they present to the trans-NHLBI Idea Forum. Sufficiently developed initiatives are subsequently considered by the BEE, which provides advice to Council.

Title: Secondary Participation in the NHGRI Genomic Resource Grant for the PhenX Toolkit (U41)

Objectives: The objective of this NHGRI competitive renewal award, funded in May 2017, is to expand the scientific scope and functionality of the PhenX (consensus measures for Phenotypes and eXposures) Toolkit. (https://www.phenxtoolkit.org/) By participating in this biotechnology resource cooperative agreement, NHLBI will support the addition or updating of HLBS-related standard measures to the Toolkit, and support development and dissemination of bioinformatics tools to help investigators improve the quality and consistency of their data and prepare their data for integrated databases such as the NIH Commons and the Precision Medicine All of Us Research Program.

Title: Career Pathway to Independence in Blood Science Award for Physician Scientists (K99/R00)

Objectives: The goals of the pilot physician-scientist-specific K99/R00-like program are to 1) attract a critical number of physician scientist career award applicants focused on blood science to this and other career mechanisms, and 2) provide career awardees in blood science with sustained support through the ‘K to R period’ that is commensurate with effort and includes a phased transition to research independence and retains ESI status at the time of the first R01 application.

Title: The Framingham Heart Study (FHS) Renewal Initiative (N01)

Objectives: The overall objective of the FHS renewal is to support novel epidemiologic research on heart, lung, blood, sleep (HLBS) and other diseases. The specific objectives are to:
1) continue participant retention, follow-up, and event adjudication for all FHS cohorts; 2) continue core study functions, including maintenance of existing data and biospecimen repositories; and 3) perform a basic research examination as a platform for independently funded ancillary studies to implement new hypothesis-driven exam procedures in the FHS Offspring, Generation 3, New Offspring Spouse, Omni 1, and Omni 2 Cohorts.

Title: The National Health and Nutrition Examination Survey (NHANES) 24-Hour Urine Sodium Component (Y01)

Objectives: To collect 24-hour urine excretion in a nationally representative sample of U.S. adults 70 years and older to monitor 5-6-year changes in sodium intake among U.S. adults 20-69 years, and for the first time, to estimate sodium intake among U.S. adults aged 70 years and older. In addition, a second (repeat) 24-hour urine specimen will be collected in a subsample among participants who provide an initial complete 24-hour urine specimen to account for within individual variation in intake.

Title: Cardiothoracic Surgical Trials Network Renewal (U10)

Objectives: The objectives of this initiative are to: (1) conduct collaborative randomized clinical trials (RCTs) and studies that evaluate cross specialty team-based care, interventions and novel therapies in the context of cardiac surgery but with broader relevance to cardiology and neurology; (2) utilize these findings to improve patient outcomes, and influence clinical practice, (3) conduct implementation research (4) train the next generation of clinical and implementation researchers.

Title: Molecular Atlas of Lung Development Program (LungMAP) Phase 2 (U01)

Objectives: The objective of the program is to build a comprehensive molecular atlas of late stage lung development in humans to serve as a critical reference platform for understanding both normal human biology and disease pathogenesis. LungMAP is an open access resource integrating multi-scale information to define lung cells with gene expression, physiological states, developmental trajectories and 3-dimensional location.

Title: Disparities Elimination through Coordinated Interventions to Prevent and Control Heart Disease Risk (DECIPHeR) (UG3/UH3/U24)

Objectives: The purpose of this initiative is to stimulate community-engaged, community-based implementation research to promote community health and reduce or eliminate cardiovascular disparities in high burden communities. Research supported by this initiative will employ proven effective evidence-based interventions in implementation research to determine optimal and sustainable strategies for delivering interventions to reduce or eliminate cardiovascular disparities and improve population health.

Title: Summer Institute for Research Education in Biostatistics (R25)

Objectives: The objective of this initiative is to support up to six awards to teach summer courses in biomedical statistics for advanced undergraduates and recent graduates to encourage them to pursue careers in biostatistics. The term “biostatistics” is used in the broad sense, including biomedical informatics and data science.

Title: Secondary Participation in the Ethical, Legal, and Social Implications of Genomics Research Program Announcements (R01, R21, R03)

Objectives: To support research on the ethical, legal, and social implications (ELSI) of human genome research. NHLBI’s approval of this request for secondary participation would stimulate ELSI research on topics specific to our large portfolio of genetic/genomic research on heart,
lung, blood, and sleep (HLBS) disorders.

Title: The Mechanistic Role of the Microbiome in the Pathobiology of Heart, Lung, Blood, and Sleep Diseases (R61/R33)

Objectives: To support functional microbiome research focused on understanding the molecular, immunological and physiological mechanisms by which the microbiota (gut, lung, oral, including bacteria, viral and fungal microflora) and its derived factors modulate heart, lung, blood and sleep (HLBS) biology and physiology to promote health or contribute to disease.

Title: NHLBI SBIR Phase IIB Bridge Awards to Accelerate the Commercialization of Technologies for Heart, Lung, Blood, and Sleep Disorders and Diseases (R44)

Objectives: The purpose of the NHLBI SBIR Phase IIB Bridge Award program is to accelerate the development and eventual commercial launch of innovative biomedical products to prevent, diagnose, and treat heart, lung, blood, and sleep-related diseases and disorders. The program facilitates the capital-intensive steps that are required to advance SBIR Phase II projects to commercialization by promoting partnerships between Phase II awardees and third-party investors or strategic partners. In addition to funding, it is expected that these third-parties will provide due diligence on the small businesses prior to the award, project management during the award, and assistance with obtaining further investment capital after the award ends. This unique public-private partnership aims to encourage investors to make earlier investments in NHLBI mission-related technologies and to ensure that the Institute only funds the best technologies with the greatest chance of reaching the market.

Title: Small Market Awards: SBIR Phase IIB Competing Renewals for Heart, Lung, Blood, and Sleep Technologies with Small Commercial Markets (R44)

Objectives: The purpose of the NHLBI SBIR Phase IIB Small Market Award is to support technology development for rare HLBS diseases and/or pediatric populations. There are two primary goals: to fill a gap in early stage funding for technologies with these small commercial markets; and to create public-private partnerships that enhance the return on the NHLBI investment in the small business program.

Title: T32 Training Program for Institutions That Promote Diversity (T32)

Objectives: The primary goals of the T32 Training Program are to: (1) contribute to the expansion of the future pool of individuals from diverse backgrounds underrepresented in the biomedicine science enterprise, (2) enable trainees to increase their competitiveness for peer review research funding, (3) strengthen publication record of trainees, and (4) foster institutional environments conducive to professional development in the biomedical science.

Title: Short-Term Research Education Program to Increase Diversity in Health-Related Research (R25)

Objectives: The goal of the Program is to support short-term research educational activities that enhance the diversity of the biomedical and behavioral research workforce. To this end, this funding opportunity announcement encourages the development of creative educational
activities with a primary focus on research experiences.

Title: Mentored Career Development Award to Promote Faculty Diversity/Re-Entry in Biomedical Research (K01)

Objectives: The objective of this initiative is to increase the number of highly trained junior faculty, from diverse backgrounds. It is targeted toward individuals whose basic, clinical, and translational research interests are grounded in the advanced methods and experimental approaches needed to solve problems related to cardiovascular, pulmonary, and hematologic diseases and sleep disorders in the general and health disparities populations. For the purpose of this announcement, “translational research” includes both early-stage (T1 and T2) as well as late-stage (T3 and T4) translational research.


CLOSED PORTION 

This portion of the meeting was closed to the public in accordance with the determination that it concerned matters exempt from mandatory disclosures under Sections 552b(c)(4) and 552b(c)(6), Title 5, U.S. Code and Section 10(d) of the Federal Advisory Committee Act, as amended (5 U.S.C. appendix 2).

IX. REVIEW OF INTRAMURAL RESEARCH

Reports prepared by the Board of Scientific Counselors (BSC), NHLBI, on the NHLBI intramural laboratories reviewed during FY 2017 were presented to the Council by Dr. Robert Balaban, Director, Division of Intramural Research, NHLBI and Dr. Collin M. Stultz (BSC Chair), Massachusetts Institute of Technology.

X. REVIEW OF APPLICATIONS

The session included a discussion of procedures and policies regarding voting and confidentiality of application materials, committee discussions and recommendations. Members absented themselves from the meeting during discussion of and voting on applications from their own institutions, or other applications in which there was a potential conflict of interest, real or apparent. Members were asked to sign a statement to this effect. The Council considered and recommended 2,705 applications requesting $5,976,951,103 in total costs. For the record, it is noted that secondary applications were also considered en bloc. There were applications considered for early concurrence during the August council round. 

ADJOURNMENT 
The meeting was adjourned at 3:17 p.m.