Thank you for participating!

Thank you to all who contributed to the National Heart, Lung, and Blood Institute (NHLBI) Strategic Visioning Forum. Ultimately, over 1,000 ideas were submitted, with more than 42,000 votes. This remarkable response exceeded expectations and provided a wealth of ideas to draw upon as NHLBI moves forward. Please visit the NHLBI Strategic Visioning page to find out more about the NHLBI Strategic Visioning process.


Welcome to the National Heart, Lung, and Blood Institute (NHLBI) Strategic Visioning Forum. The Institute is gathering ideas for the most compelling scientific priorities in the four NHLBI Strategic Goals to address over the next decade.

Goal 2: Reduce Human Disease

The Use of Therapeutic Apheresis to Reduce Circulating Levels of Galectin-3 and other Cancer and Inflammation Promoting Factors

Inflammation plays roles in cancer initiation, promotion, and progression. Elevated circulating galectin-3 (Gal-3) protein and other cancer and inflammation promoting factors (CIPFs) such as C-reactive protein and VEGF are associated with tumorigenesis and may play causative roles. Plasma Gal-3 is a biomarker, prognosticator, and pathogenic mediator of diverse cancers and is emerging as a therapeutic target. Preliminary ...more »

Submitted by (@elaine)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Details on the impact of addressing this CQ or CC :

Apheresis therapy in a clinical setting, both alone and in combination with conventional protocols, shows great potential to enhance treatment regimens, reduce dosage and side effects, improve drug deliver to target tissues, reduce long term treatment related morbidity and improve outcomes with significant benefits for patients with a broad range of cancer types and stages.

Feasibility and challenges of addressing this CQ or CC :

The need for well designed, randomized clinical trials would be readily feasible with the appropriate IND. Grant support will be needed for further development of this concept, as well as to develop columns with more optimized and specific capabilities, in addition to clinical trials demonstrating efficacy.

 

Apheresis is highly underutilized and underfunded in the US, while Apheresis research and development is much more advanced and widely utilized in Europe and Asia.

Name of idea submitter and other team members who worked on this idea : Isaac Eliaz, MD

Voting

33 net votes
40 up votes
7 down votes
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Goal 3: Advance Translational Research

Translational Bioinformatics Spanning Multiple Scales of Biologic Complexity to Implement Precision Pulmonary Medicine at the Po

What translational bioinformatics tools could be used in pulmonary medicine to allow multidimensional, multi-scale modeling of clinical and biomolecular data to assist clinical decision-making?

Submitted by (@nhlbiforumadministrator)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Compelling Question (CQ)

Details on the impact of addressing this CQ or CC :

Deployment of bioinformatics tools to construct multi-dimensional, multi-scale models of pulmonary (mal)functioning from large heterogeneous data sets spanning biological molecules, subcellular compartments, signaling pathways, cells, tissues, organs, organ systems and clinical therapeutics trials to predict actionable precision medicine for clinicians at the point of pulmonary care.

Feasibility and challenges of addressing this CQ or CC :

A variety of existing powerful informatics methods for integrating a vast wealth of clinical and high-dimensional data across DNA to organism compartments to develop multi-scale modeling approaches to improve point-of-care precision medicine. Consistent with a continuous learning healthcare system, precision medicine modeling is recursive, tentative pending better understanding and therefore continuously learning.

Fundamental to implementation of precision medicine is the ability to extract heterogeneous data from basic and clinical research to be integrated systematically into clinical practice in a cohesive and large-scale manner. Deployment of precision medicine models to predict (mal)functioning progression and response the treatment in daily practice relies strongly on the availability of an efficient bioinformatics platform that assists in the translation of basic and clinical science knowledge.

Name of idea submitter and other team members who worked on this idea : NHLBI Staff

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-2 net votes
10 up votes
12 down votes
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Goal 2: Reduce Human Disease

Prophylactic Platelet Transfusion Safety and Efficacy Before Invasive Procedures

What are the efficacy and safety of prophylactic platelet transfusions used to prevent bleeding before common invasive procedures?

Submitted by (@cjoseph)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Details on the impact of addressing this CQ or CC :

Platelets are commonly transfused prophylactically to thrombocytopenic patients before invasive procedures, but effectiveness in preventing bleeding and risks are largely unknown. Platelets are transfused for common procedures; liver biopsy, intravenous catheter insertion and removal, and lumbar puncture, among others. In the absence of evidence, decisions to transfuse are frequently based on litigation risk. Efficacy and safety data are mostly anecdotal and retrospective and guidelines are not evidence based, if institutional guidelines exist at all. The contribution of platelet transfusion to inflammation and organ injury may be considerable, particularly in patients with severe illness who are susceptible to further endothelial damage. Transfusion has immunosuppressive as well as antibody stimulatory effects. How these properties impact infection, organ dysfunction, and long term outcomes such as length of stay and survival, requires study.

 

RCTs of prophylaxis vs. no-prophylaxis with objective bleeding, adverse event and survival outcome measurements are necessary. Pre- and post- transfusion in vitro measurements of platelet function, hemostasis and markers of thrombosis performed in parallel could help determine bleeding risk and decision making. Measurements of inflammatory markers in vitro and in animal models, for example interleukin receptors and secreted cytokines, could help in understanding pathogenesis of adverse effects and identify patients at risk.

Feasibility and challenges of addressing this CQ or CC :

A randomized trial of different transfusion thresholds (e.g., 25,000/µl versus 50,000/µl) is feasible in the large target patient populations (ICU, liver disease, and hematologic malignancy) who are frequently transfused platelets for procedures. Important clinical outcomes include bleeding, thrombosis, and transfusion adverse effects. Hemostasis and platelet function assays, and tests to measure immunologic and inflammatory changes are currently available.

 

4. References

1. Platelet storage and transfusions: new concerns associated with an old therapy. Sahler J, Grimshaw K, Spinelli SL, Refaai MA, Phipps RP, Blumberg N. Drug Discov Today Dis Mech. 2011 Summer;8(1-2):e9-e14. Epub 2011 Jul 20.

2. Thrombocytopenia, Platelet Transfusion, and Outcome Following Liver Transplantation. Chin JL1, Hisamuddin SH2, O'Sullivan A3, Chan G2, McCormick PA Clin Appl Thromb Hemost. 2014 Nov 26. pii 1076029614559771. [Epub ahead of print].

Name of idea submitter and other team members who worked on this idea : Cassandra Josephson on behalf of the Platelet Committee of the State of the Science Meeting 2015

Voting

20 net votes
39 up votes
19 down votes
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Goal 3: Advance Translational Research

Using Social Media to Promote Healthy Behaviors

Since most people know that there are behaviors that they need to do to be healthy, can we leverage peer or family pressure or use social media to create a “grass roots” groundswell of health-promoting behaviors?

How might social media platforms such as Facebook and Meetup.com be leveraged for designing low-cost research studies and interventions that promote sustainable healthy lifestyle and behaviors?

Submitted by (@nhlbiforumadministrator1)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Details on the impact of addressing this CQ or CC :

Given the massive use of mobile devices and social media in our society, engaging this critical challenge would have a significant impact on our understanding how this technology can be used in disease prevention and health promotion.

Feasibility and challenges of addressing this CQ or CC :

It is feasible, timely, and cost-effective to study and incorporate into our interventions the use of social media because these applications are already so widely used.

Even if people haven’t memorized the American Heart Association’s seven factors related to heart health (get active, control cholesterol, eat better, manage blood pressure, lose weight, reduce blood sugar, stop smoking), most know that these are the behaviors that they need to do to be healthy. Despite this knowledge, heart disease is still the leading cause of death in the United States; about 1 in 3 U.S. adults has high blood pressure; diabetes affects nearly 26 million in the U.S.; and about 19% of U.S. adults are smokers. According to the CDC, in 2011 over 50% of those 18 years of age or older did not meet the recommended goal for aerobic exercise (150 minutes per week of moderate-intensity activity such as walking). Obesity is an epidemic: about one-third of American adults are obese (BMI ≥ 30 kg/m2). People know what to do, but why don’t they do it? Our built environments and culture do not intrinsically promote a healthy lifestyle. In the absence of a culture that promotes walking or biking over driving cars and that promotes fast and fattening food over more healthy food choices, can we use peer networks to promote healthy behaviors? For example, Meetup.com is a tool that people use to meet for activities that include hiking, tennis, and physical fitness boot camps.

Name of idea submitter and other team members who worked on this idea : NHLBI Staff

Voting

17 net votes
52 up votes
35 down votes
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Goal 3: Advance Translational Research

To find specific medical therapies to treat the wide array of human vascular malformations and vascular tumors.

Vascular malformations and vascular tumors, together referred to as vascular anomalies, comprise a complex and wide array of diseases in which there is a fundamental disruption in blood and lymphatic vasculature. The lesions disrupt organ function, destroy tissue, cause bleeding, increase infections and can threaten life. At present, there are some medical therapies but none are specifically targeted to an underlying ...more »

Submitted by (@joyce.bischoff)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Details on the impact of addressing this CQ or CC :

Deciphering the cellular and molecular basis of human vascular anomalies will have a critical impact for patients with these lesions and it will also have a broad, far-reaching impact on cardiovascular research because the mechanisms and insights learned from these specific vascular anomalies will teach us the fundamental rules that are needed, and must be followed, to build and maintain a stable functional vasculature in humans. This will have an impact on a variety of areas of research including regenerative medicine.

Feasibility and challenges of addressing this CQ or CC :

With the enormous advances in next generations sequencing technologies, the time is ripe for a concerted push to find the gene mutations that cause human vascular malformations and vascular tumors, both the most common and the rare. Cellular models for human endothelial cells are vastly improved and far superior to murine endothelial models, making research on patient-derived cells highly feasible.

The challenges will be to develop animal models of the individual human vascular anomalies that reflect as closely as possible the critical and specific features of the vascular malformation or vascular tumor. Such animal models, as well as relevant cellular in vitro models, would then be ideal for screen drug libraries for ability to reverse or slow the formation of the malformation or tumor. Such drugs might then be candidates to test in pilot clinical trials.

Name of idea submitter and other team members who worked on this idea : Joyce Bischoff

Voting

18 net votes
20 up votes
2 down votes
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Goal 1: Promote Human Health

Basic research in glycobiology is urgently needed

There are two areas in which glycobiology has been very successful: technology development and disease correlation. Through efforts exemplified by the CFG, major strides in mass spectroscopy and the creation of novel technologies for probing glycan-protein interactions (e.g. glycan microarrays) have been seen. Likewise, the success of the Programs of Excellence in Glycoscience and those of us focused on disease have also ...more »

Submitted by (@nhlbiforumadministrator)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Details on the impact of addressing this CQ or CC :

If my experience as a standing member of a study section and as a PI who submits greater

than ten R01 applications for every one funded, the reason for this gap in our knowledge is not an inability to

pursue such projects, nor is it a lack of desire within the field. The reason is that these studies never make it

through study section. Why? I believe the explanation is found in the nature of glycosylation: heterogeneity.

The strong bias in modern molecular-based science is that if something is not isolatable in a pure pristine

homogeneity, it cannot be studied. Not only is this short-sighted, it fundamentally betrays the nature of biology.

Feasibility and challenges of addressing this CQ or CC :

Part of the solution for these issues is improving education in glycobiology. The other part of the solution is below:

1. Provide opportunities for R01 and individual PI-focused fundamental glycobiology studies. Promote the dissection of fundamental pathways of regulation and function within in vivo settings so that we might

begin to convert disease correlation into pathways ripe for therapeutic targeting. There is nothing to translate to the clinic without detailed knowledge of these fundamental pathways.

2. Provide opportunities to expand local expertise and infrastructure in glycosciences across the country, rather than focusing on a few centers of strength. My experience tells me that investigators outside of glycobiology are far more likely to engage a local collaborator and/or core facility to help explore a glycan-related topic than to deal with a large center in a far-off university – at which they know no one.

3. There should be a push to promote individual labs with diverse interests and systems to broaden the impact by integrating glycobiology across multiple fields, rather than on concentrating expertise in a small number of large centers.

Name of idea submitter and other team members who worked on this idea : Brian Cobb

Voting

3 net votes
3 up votes
0 down votes
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Goal 2: Reduce Human Disease

A parasite?

We know Alzheimer’s Disease, Parkinson’s Disease and Luey Body Dementia are all related. Could it be they are all caused by a parasite (Luey bodies) that gives different symptoms depending on the area of the brain they infest? They would be electrically active and shorting out different parts of the brain. They might also be transmissible through intimate contact between human beings.

Submitted by (@nhlbiforumadministrator1)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Compelling Question (CQ)

Details on the impact of addressing this CQ or CC :

If the root cause of these “diseases” is actually parasites and a way can be found to kill them, we could cure all 3 diseases.

Feasibility and challenges of addressing this CQ or CC :

Live parasites can be recovered from patients with any of these diseases and experimented upon to see what could kill them without harming a patient.

Name of idea submitter and other team members who worked on this idea : Joseph C. Mullally

Voting

-24 net votes
3 up votes
27 down votes
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Goal 1: Promote Human Health

Crowdsource NIH Funding

Congress doesn't seem to "get it" regarding the return on investment for biomedical research, but maybe the public does. Create a mechanism whereby individuals can donate directly to the NIH to support research in specific areas (or provide general support).

Submitted by (@jhagood)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Details on the impact of addressing this CQ or CC :

Small efforts in specific diseases, multiple foundations, etc. lead to a lot of different "nickel and dime" funding avenues. If some of these funds flowed to NIH, they would be more likely to support meaningful research. People might feel more invested if they felt their $ were going to support the "best and the brightest"

Voting

21 net votes
29 up votes
8 down votes
Active

Goal 2: Reduce Human Disease

Biology of Red Blood Cell Alloimmunization

What determines which individuals will develop RBC alloimmune responses resulting in clinically meaningful sequelae? This question encompasses: 1) the generation of alloantibodies that limit the availability of compatible blood or cause hemolytic disease of the fetus or newborn (HDFN); 2) the distinction between clinically significant and insignificant alloantibody responses, especially within alloantibody specificities ...more »

Submitted by (@nareg.roubinian)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Compelling Question (CQ)

Details on the impact of addressing this CQ or CC :

Humans exposed to RBC alloantigens, following therapeutic transfusion or in pregnancy following maternal exposure to fetal RBCs, can generate humoral alloantibodies capable of leading to hemolytic transfusion reaction, (HTR), or of leading to HDFN. RBC transfusions can also induce autoantibodies, and can lead to hyperhemolysis. It is poorly understood why some patients mount a detectable alloantibody response (“responders”), whereas others do not (“non-responders”). Within the responder population, alloantibodies may be categorized as “clinically significant” or “clinically insignificant”, based upon whether the resultant specific alloantibodies have been previously reported to cause HTR or HDFN. However, incompatible transfusion will only result in meaningful in vivo hemolysis in some patients, even with antibody specificities classified as clinically significant.

 

The ability to define responder/non-responder status before initial RBC exposure has the potential to: 1) decrease rates of RBC alloimmunization in responders through the provision of extended matched RBCs for initial and subsequent RBC exposure; 2) conserve valuable antigen negative RBC units for patients who will derive the greatest benefit; 3) conserve transfusion service resources in terms of time spent identifying antibodies and procuring antigen negative RBC units; and 4) decrease rates of HTR and HDFN.

Feasibility and challenges of addressing this CQ or CC :

Patients with hemoglobinopathies, especially those with SCD and thalassemia, have high rates of RBC alloimmunization and thus are important target populations for these studies. The impact of methods to reduce RBC antigen exposure or pathogen inactivation on neoantigen development remains unknown. The health impact of addressing the question of RBC alloimmunization is that the discovery of mechanistic underpinnings will provide a rational basis for the development and translation of novel diagnostic and therapeutic approaches, with a goal of increasing transfusion safety.

 

Though these questions are unlikely to be completely answered within the next 3-10 years, existing and emerging immunohematology and genomics tools, evolving sophistication of animal models, and existing and novel systems for human studies including donor-recipient repositories have the potential to increase the understanding of when and how alloimmunity to RBCs evolves, in what contexts it is clinically significant—even life-threatening—and how this important, but currently challenging and poorly understood condition, might be prevented and/or mitigated.

Name of idea submitter and other team members who worked on this idea : Nareg Roubinian, MD and Naomi Luban, MD for the 2015 NHLBI State of the Science in Transfusion Medicine

Voting

44 net votes
58 up votes
14 down votes
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Goal 2: Reduce Human Disease

Childhood Interstitial Lung Disease

What is the natural history of the best characterized ChILD disorders (surfactantrelated sequence variants, neuroendocrine cell hyperplasia of infancy (NEHI),pulmonary interstitial glycogenosis (PIG),idiopathic pulmonary hemosiderosis)?

Submitted by (@nhlbiforumadministrator)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Compelling Question (CQ)

Details on the impact of addressing this CQ or CC :

We know little about the natural history of many of the child entities, and their relative rarity makes it difficult for any one center to answer the major questions they pose. The children has begun a patient registry that will begin to address the issue of natural history and disease tracking.

 

a. To improve the power of such a registry, we suggest that support be provided to find novel methods to link this data base to available electronic medical records of participating centers in order to assess physiologic and other clinical associations with specific diseases.

 

b. Support for a biomarker repository holding serum, frozen and fixed lung tissue, patient DNA, RNA, and proteomic and metabolomic materials, and bronchoalveolar lavage effluent and cell pellets, will allow for genome wide analysis as well as proteomic and metabolomic analysis.

Feasibility and challenges of addressing this CQ or CC :

This question is best addressed in the context of a multicenter data registry, ideally linked to a clinical sample.

Name of idea submitter and other team members who worked on this idea : ATS Member

Voting

2 net votes
2 up votes
0 down votes
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Goal 3: Advance Translational Research

Use of symptoms vs spirometry in increasing patient and provider adherence to guidelines

What is the comparative effectiveness of using symptoms vs. spirometry in increasing patient and provider adherence to COPD treatment guidelines and patient-reported outcomes (symptom frequency, activities of daily living, quality of life, sleep quality, exacerbations)?

Submitted by (@hgussin)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Compelling Question (CQ)

Details on the impact of addressing this CQ or CC :

COPD is underdiagnosed. The lack of recognition of COPD risk by physicians and patients themselves is well known, with many undiagnosed COPD patients presenting for the first time with late stage COPD. Currently used cut-points based on a fixed ratio of FEV1/FVC may overestimate the number of elderly patients with COPD, particularly with mild disease, because of changes in lung volumes with aging. It has been suggested that using a cut-point based on the normal distribution of FEV1/FVC values may decrease the misclassification rate. Other strategies have been proposed for risk assessment as adjuncts to diagnostic classification (e.g., Fragoso et al. J Am Geriatr Soc 2008, 56:1014-1020). Pertinent references: Guideline #1 in Qaseem et al., strong recommendation, moderate-quality evidence; GOLD, 2008 and the 2005 American Thoracic Society/European Respiratory Society Task Force Report, standards for the diagnosis and management of patients with COPD.

Although there are ample guidance to help providers identify and evaluate patients likely to have earlier stage COPD, few are referred to spirometric testing. Subsequent spirometry provides a good working yield of true positives, which is frequently superior to pre-test probabilities of other, more complex and expensive medical tests commonly ordered for other conditions (colonoscopy,lung cancer), why is it so much more difficult to provide spirometry? COPD will remain undertreated as long as it remains underdiagnosed.

Name of idea submitter and other team members who worked on this idea : Helene Gussin, PhD

Voting

9 net votes
12 up votes
3 down votes
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Goal 1: Promote Human Health

Screening for asymptomatic vascular disease

Does screening for asymptomatic vascular disease increase awareness, promote compliance with lifestyle interventions, and improve overall health?

Submitted by (@societyforvascularsurgery)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Compelling Question (CQ)

Name of idea submitter and other team members who worked on this idea : Society for Vascular Surgery

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0 net votes
2 up votes
2 down votes
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