Goal 2: Reduce Human Disease

To extend our knowledge of the pathobiology of heart, lung, blood, and sleep disorders and enable clinical investigations that advance the prediction, prevention, preemption, treatment, and cures of human disease.

Goal 2: Reduce Human Disease

Research priorities: Sleep disorders in women

Women are more likely to have insomnia and they develop the condition at an earlier age than men. Hormone changes and menopause are two key factors but little is known about the mechanisms, which will inform the treatment. This is an understudied area of scientific focus with vast potential public health impact.

Submitted by (@hjoffe)

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Goal 2: Reduce Human Disease

Mitigating risks due to the RBC storage lesion and vulnerable patients

What are the underlying dependencies (genomic, metabolic, disease) in individual donors that either accelerate or delay the changes to red blood cells during refrigerated storage? What methods of preparation might protect patients from the risks posed by the accelerated degradation of RBCs provided by "poor storers"? What characteristics of individual patients make them particularly vulnerable to transfusion of red ...more »

Submitted by (@andrew.dunham)

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Goal 2: Reduce Human Disease

Clinicial studies of Azithromycin for COPD Treatment

Azithromycin is very effective at reducing exacerbations in COPD, but we do not understand the biological mechanism, i.e. anitbiotic effect versus antiinflammatory effect. Because we do not understand how it works (and because of the potential for side effects), azithromycin is not yet widely used. Clinical studies designed to answer this question would help us refine our therapeutic approach and may allow us to minimze ...more »

Submitted by (@nhlbiforumadministrator)

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Goal 2: Reduce Human Disease

Iron Metabolism in thalassemia syndromes, and other rare and common anemias

Thanks to elegant work since the 1990s, many details of the role of iron regulation and metabolism have been elucidated. Recent efforts in academic and pharmaceutical laboratories aim to translate these discoveries into therapies that may alleviate anemia in iron-refractory states (including anemia of inflammation and congenital disorders), and may have important therapeutic effects in non-transfusion-dependent thalassemia, ...more »

Submitted by (@gcioffi)

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Goal 2: Reduce Human Disease

Challenge

Genetic or biologic makers that predict outcomes in pulmonary fibrosis are needed.

Validated animal models of lung fibrosis that better resemble the human condition are needed to speed up the drug development process.

An international patient registry is needed to help promote understanding of the natural history of pulmonary fibrosis and real-world impacts of interventions.

Submitted by (@swigrisj)

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Goal 2: Reduce Human Disease

Systems Approaches to "Phenosimilar" Diseases

There are diverse diseases that share similar features. For example, many chronic airways diseases (chronic aspiration, ciliary dyskinesia, some COPD) "phenocopy" cystic fibrosis in terms of infectious agents, mucus clearance problems, progressive loss of lung function, etc. Use multiplatform "omics" approaches and Big Data bioinformatics to identify common nodes for therapeutic targeting. Other examples: emphysema and ...more »

Submitted by (@jhagood)

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Goal 2: Reduce Human Disease

Psychological & Behavioral Phenotyping to Improve Weight Control

What information is needed to improve our understanding of individual characteristics and processes that explain or predict dietary intake patterns, initiation or sustained engagement in physical activity, and sedentary behavior so that we can improve treatment matching and identify novel targets for more efficacious individual- and population-level approaches for weight management?

Submitted by (@nhlbiforumadministrator)

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