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.

Strategic Goal: Goal 3: Advance Translational Research

Durable gene activity map at the individual level

A durable gene activity map of the individual to understand when certain gene sets are on vs off or dysfunctional over an individual’s lifetime as one way of guiding the precision of medicine for that patient. It would need to be person portable and universally exportable and interpretable across all of the EHRs.

Submitted by

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 of Critical Care Medicine Executive Committee/Council

Voting

2 net votes
3 up votes
1 down votes
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Strategic Goal: Goal 2: Reduce Human Disease

The 'other' circulation system - the lymphatic system needs the attention the blood circulation system receives.

Much is still unknown about the lymphatic system. No medical specialist owns it. NHLBI and NCI could work together on understanding lymphatic disease in the direction of understanding how metastasis spreads disease. NHLBI can help by establishing a unit to organize research interest in such an important body system in health and disease.

Submitted by

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

Details on the impact of addressing this CQ or CC :

Understanding the lymphatic system and how metastasis facilitates it as a highway to spread disease could save many lives.

Feasibility and challenges of addressing this CQ or CC :

There are several lymphatic diseases such as lymphangiomatosis - which are complex and systemic - through the study of which, we can learn the role the lymphatic system plays in disease - beyond the well-known essential functions of fat absorption, the immune response and in maintaining fluid balance.

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

New Targets for the Treatment of Heart Failure

Heart failure (HF) is one of the major health challenges in the 21st Century. Its prevalence is due a growing number of patients who survive heart attacks, who later develop heart failure; and the high incidence of diabetes leading to diabetic cardiomyopathy. Current treatments for HF only slow the progression of the disease; no treatment stops or reverses this adverse sequence. These limitations provoke the question ...more »

Submitted by

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

Details on the impact of addressing this CQ or CC :

The current treatment regimens for patients with heart failure (HF) focus on strategies to reduce cardiac work (by reducing heart rate, beta-blockers), afterload reduction (ACE inhibitors), and decrease in preload/blood volume (ACE inhibitors, aldosterone antagonists, diuretics). None of these treatments stop or reverse the progression of the disease. A recent gene therapy trial designed to improve cardiac contractility and calcium handling in HF has failed in clinical trials. Perhaps the reason that the current and experimental treatments have not produced an outcome of stopping or reversing the progression of the disease relates to the "cell" they are targeting. In this regard, the current treatments principally target cardiac myocytes, but there is evidence that vascular perfusion abnormalities may also be involved in the disease. One such piece of evidence relates to the diffuse fibrosis occurring in the failing hearts. Such fibrosis is often referred to as replacement fibrosis in that the fibrotic tissue has replaced cardiac myocytes which died. This death could be the result of a perfusion abnormalities caused by inadequate dilation of the coronary resistance vessels. Thus, is heart failure a pathology involving both the coronary circulation and cardiac muscle? In this regard, future investigations of heart failure consider cardiac-coronary interactions leading to perfusion abnormalities as a key factor in the progression of heart failure.

Feasibility and challenges of addressing this CQ or CC :

The challenge of addressing a coronary vascular contribution to heart failure would involve an interdisciplinary approach using genetic models with cardiac and/or vascular (smooth muscle and endothelium) expression or knock-out of key genes involved in cardiac function and vascular control. Sophisticated measurements of cardiac function and metabolic status of the heart using echocardiography, MRI, PET and a L-Band EPR would provide insight into flow-function-metabolism-oxygenation relations in the normal and failing heart.

Name of idea submitter and other team members who worked on this idea : William M Chilian

Voting

13 net votes
18 up votes
5 down votes
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Strategic Goal: Goal 3: Advance Translational Research

Cell-specific drug delivery

New paradigms are needed for developing both deliver systems AND drugs to achieve therapeutically effective cell-specific drug delivery.

Submitted by

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

Details on the impact of addressing this CQ or CC :

The proposed development is essentially needed for new effective therapies and personalized medicine.

Feasibility and challenges of addressing this CQ or CC :

The proposed approach is feasible but requires that new paradigms are adopted by organizations and researchers.

Name of idea submitter and other team members who worked on this idea : Karel Petrak

Voting

19 net votes
22 up votes
3 down votes
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Strategic Goal: Goal 2: Reduce Human Disease

To build on the information obtained from population based and epidemiological studies

What is the basis for relative resistance or vulnerabilities to obesity or related adverse health effects observed in various segments of population?

Submitted by

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

Details on the impact of addressing this CQ or CC :

Population based and epidemiological studies have identified segments of population that are resistant or susceptible to obesity and cardiovascular risks. Continued research is needed to identify additional such populations and their differences. Moreover, the next phase of research could be to understand the basis for this relative resistance or vulnerabilities, and exploit the information to improve obesity and cardiovascular disease risk at a community level.

Feasibility and challenges of addressing this CQ or CC :

Moving forward, strategies are needed that don’t yield only incremental benefits, but instead emphasize ‘high risk, high impact’ research to some extent. Given the enormous public health crisis obesity represents we urge continued support of basic, clinical, population, and translational obesity research.

Name of idea submitter and other team members who worked on this idea : The Obesity Society

Voting

7 net votes
13 up votes
6 down votes
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Strategic Goal: Goal 2: Reduce Human Disease

How can we improve strategies for weight loss maintenance

There is a need to improve and optimize strategies for weight loss maintenance to make them more effective for more individuals.

Submitted by

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

Details on the impact of addressing this CQ or CC :

Weight regain is the biggest challenge in obesity treatment. For many obese or overweight adults, losing weight over an initial period of time (usually 6 months) is achievable using a comprehensive lifestyle program, which includes a reduced-calorie diet, increased physical activity, and behavior therapy. Yet, participants in even the most successful weight loss programs regain weight after the first 6-12 months, especially if the intervention is discontinued or becomes less intensive.

Feasibility and challenges of addressing this CQ or CC :

recommendations from a recent working group meeting. We are ready to do more cross-collaborative work between physiologists and behavioral interventionists.

 

This question addresses both discovery science and clinical science goals.

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

Voting

67 net votes
97 up votes
30 down votes
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Strategic Goal: Goal 3: Advance Translational Research

Finding Why Apolipoprotein L1 Gene Variants Increase the Risk for Kidney Failure in African Americans

We plan to find the role of APOL1 in increasing the risk of kidney failure in African Americans and translate this knowledge into preventing end-stage kidney disease and the need for kidney replacement therapy, dialysis or kidney transplantation.

Submitted by

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

Details on the impact of addressing this CQ or CC :

Increase the quality of life for millions by preventing end-stage kidney disease and thus saving billions of dollars.

Name of idea submitter and other team members who worked on this idea : Carey Green MD, Dollie Green MD

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1 net vote
1 up votes
0 down votes
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Strategic Goal: Goal 3: Advance Translational Research

Translational Cardiovascular Medicine

There is a need for the NHLBI to catalyze the development of tools and shared data resources to facilitate mechanistic studies in a human model system. This includes the ability to culture human cardiac tissue, as well as generate a resource to systematically characterize and catalog the epigenome and histone marks associated with the transcriptome in normal and diseased heart tissues.

Submitted by

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

Details on the impact of addressing this CQ or CC :

The in-depth characterization of complex cellular systems is often beyond the scope and capability of individual investigators working in isolation. The NHLBI research community lacks a shared tissue-specific systems biology framework resource that would accelerate the progress of individual, investigator-initiated research.

 

The Human Genome Project and subsequent sequencing projects are characterizing the structural architecture of the human genome, but the biological significance of the human genome and the characterization of functional elements that mediate gene-environment interactions and influence tissue-specific gene expression remain to be further defined.

Feasibility and challenges of addressing this CQ or CC :

Advances in the ability to culture adult human cardiac tissue for prolonged periods while maintaining normal electrical and mechanical function will enable testing of novel therapeutics to treat cardiovascular disease. Tissue-specific epigenetic modification of nucleic acids and dynamic changes in the histone code and chromatin remodeling are important determinants of the transcriptome within heart tissue. Advances in the ability to perform genome-wide analyses in human cardiac tissues to assess dynamic changes in the histone code and chromatin remodeling will enable comparison between normal and disease states.

Name of idea submitter and other team members who worked on this idea : Stacey Rentschler and colleagues, Washington University in St. Louis

Voting

6 net votes
10 up votes
4 down votes
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Strategic Goal: Goal 2: Reduce Human Disease

Determining the significance of variants of unknown significance

Variants of unknown significance (VUS) confound the value of genetic testing. We need to improve our understanding of the clinical significance of VUS so we can more definitively re-classify them as pathologic or benign variants.

Submitted by

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

Details on the impact of addressing this CQ or CC :

Next gen sequencing and clinical genetic testing are becoming a more frequent component of clinical care. Not infrequently a VUS result is returned providing little clinical value to the patient. Determining the significance of these variants will be extremely valuable to patients undergoing genetic testing.

Feasibility and challenges of addressing this CQ or CC :

Bioinformatics and big data infrastructure will mature over the next several years to allow us to record VUS results, follow patients longitudinally, aggregate data, and ultimately determine the significance of VUS.

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

Voting

-9 net votes
6 up votes
15 down votes
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Strategic Goal: Goal 3: Advance Translational Research

Need to Determine the Basis for Difference in Response to Weight Management Approaches

Why does the response to weight loss regimen in terms of weight loss and its sustainability, and improvement in health outcomes vary considerably?

Submitted by

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

Details on the impact of addressing this CQ or CC :

Past research focused on using variations of diet and exercise has revealed important information about the health benefits of weight loss. The limitations of such approaches in producing biologically meaningful and sustained weight loss for the majority have also been recognized. Even within a study population, compliance to weight loss regimen, weight loss and its sustainability, and improvement in health outcomes vary considerably. Research is needed to identify the basis for this variation, which may lead to enhanced outcome and applicability of such approaches.

Feasibility and challenges of addressing this CQ or CC :

Identifying factors that enhance weight management response may lead to translational studies that yield more effective results. A strong support of promising clinical translational research may promote a conducive environment for developing more practical applications.

Name of idea submitter and other team members who worked on this idea : The Obesity Society

Voting

8 net votes
14 up votes
6 down votes
Active

Strategic Goal: Goal 2: Reduce Human Disease

Blood Pressure Recommendation

What should be the systolic blood pressure goal for pharmacological treatment, and should it vary by age or by cardiovascular disease (CVD) risk category?

Submitted by

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

Feasibility and challenges of addressing this CQ or CC :

Despite fifty years of clinical trial research and forty years of national guideline activity, important clinical questions remain under intense scientific debate. The importance of these questions are underline by the scientific consensus that hypertension is most important cardiovascular risk factor globally, in fact, more important than even tobacco use.

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

Voting

-1 net votes
13 up votes
14 down votes
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Strategic Goal: Goal 2: Reduce Human Disease

Identifying Epistatic Genes in Sickle Cell Disease

What genes are involved in the modulation of phenotype in sickle cell disease?

Submitted by

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

Details on the impact of addressing this CQ or CC :

The pathophysiology of sickle cell disease results from cellular defects caused directly by the Hb S mutation interacting with the environment and many other gene products—some known, but most yet unidentified–a typical example of epistasis. How normal tissue perfusion is interrupted is complex and why the phenotype of sickle cell disease differs from patient to patient is poorly understood. Answers to this question will provide additional insight into their biological and functional relationships.

Feasibility and challenges of addressing this CQ or CC :

Scientific advances make it feasible to identify additional epistatic genes, which will provide additional insight into their biological and functional relationships.

Name of idea submitter and other team members who worked on this idea : The Sickle Cell Association of New Jersey

Voting

6 net votes
6 up votes
0 down votes
Active