Goal 2: Reduce Human Disease

Disease Severity Biomarkers for Sickle Cell Disease

Can we identify biomarkers that can predict sickle cell disease severity?

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 :

Identifying reliable biomarkers that can predict severity of sickle cell disease could help doctors and patients make better decisions about a wide range of clinical decisions, including weighing the risks vs. benefits of bone marrow transplantation, chemotherapeutic manipulation of Hb F level, and gene therapy, all of which have potentially life-threatening complications.

Feasibility and challenges of addressing this CQ or CC :

Scientific advances make it feasible to identify biomarkers of disease severity. However, identifying biomarkers with good sensitivity and specificity is likely to be a challenge.

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

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

Developing tools/algorithms for objective evaluation of sleep health

What are the best tools/algorithms for robust and objective evaluations of sleep health biomarkers?

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 :

Sleep deficiency is pervasive in today’s society and associated with an array of threats to health and public safety. The availability of a biomarker(s) for sleep health would turn-the-curve on developing practical and feasible ways to identify individuals at risk for sleep deficiency and prevent/manage associated risks to health and public safety on a large-scale.

Feasibility and challenges of addressing this CQ or CC :

Sleep and circadian regulation is coupled to an array of behavioral, physiological and molecular/genetic processes to leverage in the development of biomarkers for sleep health.

Untreated sleep disorders and sleep deficiency pose a significant burden on health and public safety. There is currently no biomarker, or point-of-care technology available to objectively measure an individual’s level of sleep deficiency or susceptibility, a significant barrier to prevention and management.

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

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124 net votes
162 up votes
38 down votes
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Goal 2: Reduce Human Disease

Predictive biomarkers

What biomarkers of acute environmental exposure are predictive of disease onset or progression?

Submitted by (@nhlbiforumadministrator1)

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

Voting

18 net votes
27 up votes
9 down votes
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Goal 2: Reduce Human Disease

Predicting and monitoring atherosclerosis progression and vulnerable plaque

How to develop novel methods for predicting and monitoring atherosclerosis progression and vulnerable plaque including biomarkers and imaging?

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

Early COPD

What does early COPD actually look like. This is defined as severe COPD 30 years prior to its manifestation.

Submitted by (@davidmannino)

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

Details on the impact of addressing this CQ or CC :

Prevention programs in COPD either target smoking or those with established disease. Better understanding the factors that lead to the development of COPD (both in ever and never smokers) is critical to improved disease prevention.

Feasibility and challenges of addressing this CQ or CC :

We need to revisit long term studies in novel ways- and look at new cohorts. Better biomarkers need to be developed.

Name of idea submitter and other team members who worked on this idea : Dave Mannino

Voting

22 net votes
31 up votes
9 down votes
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Goal 2: Reduce Human Disease

Biomarkers and Response Predictors in Asthma

As the current chair of the Research and Training Division, I would like to convey that the AAAAI membership would like the NHLBI to consider the following in the development of its strategic plan: Given the development of specific biologic therapy for asthma, what are the biomarkers and predictors of response that will allow clinicians to choose the best therapeutic combination of medications (biologic and otherwise) ...more »

Submitted by (@wheeze)

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 : Mitchell Grayson on behalf of the American Academy of Allergy, Asthma, and Immunology

Voting

7 net votes
18 up votes
11 down votes
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Goal 2: Reduce Human Disease

Predictors of Sickle Cell Disease Severity

Can better predictors of disease severity such as specific biomarkers and/or genetic polymorphisms be identified so as to help understand the course and progression of sickle cell disease in various patients?

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 high clinical variability in sickle cell disease (SCD) and the lack of sufficient data to help understand and or predict the course of an individual’s disease warrants the identification of better predictors of disease severity. The identification of predictors of disease severity, such as biomarkers, will be vital in the management and treatment of SCD, especially since more recently several plasma biomarkers and certain genetic polymorphisms have been proposed to influence specific clinical outcomes, including stroke, sickle cell nephropathy, and survival. Furthermore, studies of biomarkers or genetic markers in the context of clinical drug trials may be helpful in predicting response rates, thus allowing for more personalized therapeutic decisions.

Name of idea submitter and other team members who worked on this idea : Alice Kuaban on behalf of the American Society of Hematology (ASH)

Voting

58 net votes
76 up votes
18 down votes
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Goal 2: Reduce Human Disease

Biomarkers of Pulmonary Hypertension

What are informative and clinically relevant biomarkers of pulmonary hypertension (PH)?

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 :

This research emphasis would help identify novel pulmonary hypertension biomarkers of disease risk and progression that can be used for early detection or as outcome measures in prevention trials or treatment of PH, which is a disease currently still not curable with high mortality rate.

Feasibility and challenges of addressing this CQ or CC :

NHLBI Division of Lung Diseases just launched the multi-center PVDOMICS research program last September that will enroll ~1,500 patients in the next 5 years for deep phenotyping PH. PVDOMICS will provide a perfect foundation and platform for this proposed featured study about informative and clinically relevant biomarkers of PH, and make answering this proposed question more feasible in the next 5-10 years.

Although significant advances in the treatment of pulmonary hypertension have been made in the past two decades, currently pulmonary hypertension remains a devastating disease without many clinically relevant and specific biomarkers available. Novel new informative and clinically relevant pulmonary hypertension biomarkers would greatly help advance the subtype-specific early diagnosis and precision treatment of this disease that could potentially decrease the mortality of PH.

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

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75 net votes
87 up votes
12 down votes
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Goal 2: Reduce Human Disease

Biomarkers and phenotypic characteristics of asthma patients

Are there biomarkers or phenotypic characteristics that will allow us to identify the patients with asthma who will experience a more rapid decline in lung function?

Submitted by (@nhlbiforumadministrator)

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

Name of idea submitter and other team members who worked on this idea : American Thoracic Society

Voting

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

Drug Hypersensitivity Databases

As the current chair of the Research and Training Division, I would like to convey that the AAAAI membership would like the NHLBI to consider the following in the development of its strategic plan: Drug Hypersensitivity is a growing concern for patients who are unprotected against potentially severe and lethal reactions. It would be important to generate databases to characterized the different drug reactions, their ...more »

Submitted by (@wheeze)

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

Name of idea submitter and other team members who worked on this idea : Mitchell Grayson on behalf of the American Academy of Allergy, Asthma, and Immunology

Voting

-7 net votes
11 up votes
18 down votes
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Goal 3: Advance Translational Research

Detection of inflammatory monocytes as a biomarker of CVD

Coronary artery disease (CAD), of which atherosclerosis is a major contributor, costs the United States $108.9 billion each year. While a number of conventional risk factors such as smoking, diabetes and hypertension have been associated with CAD, their predictive performance is poor in the prevention of acute coronary syndrome (ACS). Despite the prevalence of ACS in our society, there are currently no molecular biomarkers ...more »

Submitted by (@scottsimon)

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

Details on the impact of addressing this CQ or CC :

Need more opportunities to put forth innovative diagnostic and therapeutic solutions than currently vetted through the CTSC process.

We have developed the Artery-on-a-chip (A-chip), currently in a research tool format, which accurately measures the extent of inflammatory monocyte activation in a whole blood sample by shearing it along a molecular sensor and enumerating monocyte capture.

Name of idea submitter and other team members who worked on this idea : Scott I Simon, Greg Foster, Ehrin Armstrong

Voting

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

Identifying Biomarkers of Chronic Lung Diseases

What are the biomarkers that identify expression and progression of specific subtypes of chronic lung disease?

Submitted by (@jdc000)

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

Details on the impact of addressing this CQ or CC :

Most chronic lung diseases, including COPD, do not have biomarkers that reflect disease pathogenesis accurately. Readily measurable biomarkers, especially in peripheral blood, would be key tools for clinical trials and studies of disease heterogeneity.

Feasibility and challenges of addressing this CQ or CC :

The most useful chronic lung disease biomarkers would be proteins, metabolites, or gene expression measurements in peripheral blood. Defining useful peripheral blood biomarkers that reflect lung disease pathogenesis will require validation that the lung disease process can be captured in peripheral blood measurements.

Name of idea submitter and other team members who worked on this idea : Ed Silverman, James Crapo and COPDGene Executive Committee

Voting

42 net votes
62 up votes
20 down votes
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