Goal 2: Reduce Human Disease

Combination Iron Chelator Trials in Thalassemia and other transfusion-dependent anemias

Three chelators are presently available in the US and much of the world: parenteral deferoxamine, and oral deferasirox, as well as oral deferiprone. Monotherapy is unsuccessful in a significant minority of patients, due to side effects or inadequate response at tolerable doses. Taking a page from enormously successful strategies for combination oral therapy in hypertension, led by NHLBI and others over the past four ...more »

Submitted by (@gcioffi)

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

Feasibility and challenges of addressing this CQ or CC :

NHLBI should lead the way for these trials, which will be of enormous benefit to reduce morbidity and mortality in thalassemia, a terribly common problem worldwide, as well as in the rare, transfusion-dependent congenital anemia.

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54 up votes
15 down votes
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Goal 3: Advance Translational Research

Enhancing Translational Returns With Better Animal Models and the Basic Science Needed to Support Such Efforts

Can we improve on the preclinical development of therapies through more informed choices on new animal models by linking basic science at the R01 level with national resource centers?

Submitted by (@johnengelhardt)

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 development of effective therapies for heart, lung, and blood require the appropriate animal models for testing. Mouse models have been the mainstay and for the most part very effective. But for those diseases where mice fall short, humans have become the testing ground. With the massive push for translation at NIH, clinical trials often lack proof of efficacy in animal models or are wrongly based on biology in rodents that does not apply to humans. These clinical efforts that don’t effectively translate are exhausting resources to maintain a robust RO1 pipeline on basic research. Recognizing that we must push for translation and also keep basic research funded at a high level, NIH and NHBLI needs to get more creative in taping the best animal models for the disease.

Feasibility and challenges of addressing this CQ or CC :

With new technologies rapidly expanding for transgenesis in embryos, picking the appropriate species for modeling a given disease is now becoming a reality. However, there are several barriers to growth in this area: 1) we often do not know organ physiology and stem cell biology well enough in non-rodent species, 2) the average researcher typically does not have the expertise to utilize non-rodent models in their research or to generate new genetic non-rodent models for study, 3) the costs of non-rodent disease models is high and must be strategically utilized. One potential solution is to maintain resource centers in particular key species that collaborate with basic scientists to both better understand non-rodent organ biology and work selectively to translate basic discovery into therapies. NHLBI recently had an RFA for this type of work that was discontinued. If a new RFA was designed that links funded research (and/or new research applications) through NHLBI to selected target mission diseases and the use of strategic resource centers with expertise in alternative non-rodent models, this might productively transition appropriate use of new models for the next generation of scientists. Such an RFA for example, could provide supplements to existing R01s for projects linked to resource centers and/or have specific R01 RFAs to enter into studies in new animal models or to create new models for a given purpose.

Name of idea submitter and other team members who worked on this idea : John Engelhardt

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31 net votes
48 up votes
17 down votes
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Goal 2: Reduce Human Disease

Coconut oil and the Effect on Lipids

What is the effect of coconut oil (high in saturated fat) on blood lipids and cardiovascular disease (CVD) risk?

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 :

Coconut oil is a highly saturated fat yet it is being promoted heavily in the popular media as a healthy fat. It is touted as a cure all for several chronic conditions including CVD risk factors. It is important to determine how coconut oil impacts blood lipids for public health.

Feasibility and challenges of addressing this CQ or CC :

A feeding trial would provide clear results on the effect of coconut oil on the effect of lipids.

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

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-14 net votes
7 up votes
21 down votes
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Goal 2: Reduce Human Disease

Molecular determinants of pulmonary failure in sepsis

Respiratory failure in sepsis is almost universal and leads to worse clinical outcomes, yet it is poorly understood. Recent epidemics of pulmonary failure from respiratory viruses (e.g. influenza, SARS, MERS, etc) makes understanding molecular determinants of respiratory failure and the associated inflammatory and physiologic responses, critical for improving the health of our nation and potentially mitigating future ...more »

Submitted by (@greg.martin)

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

Details on the impact of addressing this CQ or CC :

Exploring and understanding the molecular determinants of pulmonary failure will impact not only the predictable complications of acute respiratory illnesses such as influenza, but also inform our understanidng and treatment of myriad other common respiratory illnesses resulting in pulmonary failure, such as pneumonia, chronic obstructive pulmonary disease, asthma, obesity hypoventilation, etc.

Feasibility and challenges of addressing this CQ or CC :

Patients receiving critical care services in the United States are among the most close monitored, including continuous monitoring of cardiorespiratory physiology. This highly monitored population is a nature source for studying longitudinal changes in molecular patterns and respiratory physiology.

Name of idea submitter and other team members who worked on this idea : Society of Critical Care Medicine Executive Committee/Council

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

Investigator-Initiated Early Translation

What changes are needed to facilitate investigators independently recognizing and pursuing the early translation of their discoveries towards clinical applications through competitive peer reviewed models?

Submitted by (@nhlbiforumadministrator)

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 : NHLBI Staff

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35 net votes
44 up votes
9 down votes
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Goal 4: Develop Workforce and Resources

Research training to support population-focused obesity research in ethnic minority populations

NIH is already facing a challenge in increasing the number and viability of researchers of color. Obesity research in black (or other high risk minority) populations can be used to explore how research training programs that focus on specific issues of importance to populations of color might contribute to the recruitment and success of ethnic minority researchers in the NIH system.

Submitted by (@skumanyi)

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

Details on the impact of addressing this CQ or CC :

To say the least, not all researchers of color study disparities related issues and not all disparities research is done by researchers of color. That is the way it should be. However, I suspect that research focusing on populations of color would attract a greater than average proportion of researchers of color (NIMHD might have data on this but NIMHD funding alone would be grossly insufficient as the only relevant funding stream. It would also be inappropriate and ineffective to silo the entire burden as an NIMHD responsibility).

Feasibility and challenges of addressing this CQ or CC :

The infrastructure for such training might not exist. Isolated minority researchers attached to various centers and programs would not necessarily work; some sort of networking would have to be done based on an infrastructure devoted to population-oriented obesity research and with a critical mass of obesity researchers focusing on the black (or other) population..

Name of idea submitter and other team members who worked on this idea : Shiriki Kumanyika, Melicia Whitt-Glover, Debra Haire-Joshu

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6 net votes
6 up votes
0 down votes
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Goal 1: Promote Human Health

Qigong and Tai Chi for Chronic Disease Prevention

Non-pharmacological interventions for pain and stress have gained tremendous momentum. Mind-Body Practice -- Qigong and Tai Chi -- are group based and inexpensive to implement. The evidence base suggests that these practices are safe and effective for a multitude of preventable chronic disorders.. THE QUESTION: Given safety and efficacy, should there be vigorous research on implementation of Qigong and Tai Chi and ...more »

Submitted by (@rogerjahnke)

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

Details on the impact of addressing this CQ or CC :

What can we do to assure that safe, effective, inexpensive non-parmacological approaches like Qigong and Tai Chi become widely diffused into communities, agencies, organizations, schools, health systems and businesses.

Feasibility and challenges of addressing this CQ or CC :

We have participated in a number of studies that have contributed to the evidence base for Mind-Body Practice as a safe and effective non-pharmacological programming.

 

The key -- group based. For the financing, group based is inexpensive. For the efficacy group based supports compliance.

Name of idea submitter and other team members who worked on this idea : Dr Roger Jahnke, http://IIQTC.org

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2 net votes
33 up votes
31 down votes
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Goal 2: Reduce Human Disease

Improve vascular healing and extend long term benefit of interventions

How can we develop new approaches to improve vascular healing and extend the long term benefits of vascular interventions for more patients?

Submitted by (@societyforvascularsurgery)

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 response to vascular injury, whether it be catheter interventions, bypass surgery, or chronic implants, is a reactive process characterized by inflammation, cell proliferation, and fibrosis leading to failure. Better understanding of the mechanisms of vessel remodeling, and restoring homeostasis, is needed to improve prediction, develop and translate new treatments. This remains the leading scientific problem in vascular medicine and surgery. New approaches such as proteomics, lipidomics, molecular imaging offer new opportunities in this realm.

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

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0 net votes
1 up votes
1 down votes
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Goal 1: Promote Human Health

Prevention of Obesity

What are the behavioral factors that predispose to excessive weight gain and development of obesity? And, which intervention strategies can effectively prevent excessive weight gain and obesity? NHLBI, other NIH institutes and the society at-large have invested heavily in research and clinical practice aimed at treatment of obesity (i.e, weight loss in those who are already overweight). However, much less research ...more »

Submitted by (@rpate0)

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

Details on the impact of addressing this CQ or CC :

Reducing the prevalence of obesity is one of the great public health challenges of the 21st century. Research should be focused, first and foremost, on prevention, not treatment, of this problem. It seems highly likely that improving the behaviors that can prevent obesity would produce a wide range of important public health benefits.

Feasibility and challenges of addressing this CQ or CC :

Two generations ago the prevalence of obesity was much lower than it is today. The prevalence was lower then, not because overweight people were better at losing weight; rather rates were lower because far fewer people became overweight in the first place. It is high time that the scientific community, clinicians, and public health practitioners invested their efforts in prevention first, where there is every reason to believe we could be successful. These efforts should be informed by a robust body of knowledge, and it is recommended that NHLBI lead the effort to expand the body of knowledge on primary prevention of obesity.

Name of idea submitter and other team members who worked on this idea : Russell Pate

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6 net votes
8 up votes
2 down votes
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Goal 3: Advance Translational Research

Diagnosing Risks to Sleep Health and Therapeutic Responses

What are practical point-of-care diagnostic biomarkers that could be used for assessment of sleep/circadian health, sleep disorders, and the risk of sleep-related heart, lung, and blood diseases?

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 :

Existing assessments based on intensive overnight physiological measurements of sleep interpreted by medical specialists are impractical for the goal of diagnosing a spectrum of sleep-related health risks and the need to protect the safety of the public at large. Sleep/circadian-related biomarker panels are needed to enable the development of practical diagnostic tests for point of care implementation, and determining whether the response to therapy has been successful.

Feasibility and challenges of addressing this CQ or CC :

Mature high-throughput genomic technologies combined with recent advances in our knowledge of sleep-coupled pathways provide a rich foundation for systematic investigation and the application of computational modeling strategies.

Discovery research advances implicate an array of cellular sleep and circadian mechanisms in pathophysiological pathways leading to cardiometabolic and pulmonary disease. Irregular and disturbed sleep impairs cellular biological rhythm in all tissues and organs leading to oxidative stress, unfolded protein responses, and impaired cell function. These pathophysiological changes are accessible to existing high-throughput quantitative technologies facilitating systematic study and the identification of candidates and panels of candidate correlating with sleep health status.

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

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81 net votes
107 up votes
26 down votes
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