Goal 3: Advance Translational Research

To facilitate innovation and accelerate research translation, knowledge dissemination, and implementation science that enhances public health.

Goal 3: Advance Translational Research

Submitted by (@nhlbiforumadministrator)

A Pipeline for Investigator- Initiated Translational Science

How might the NHLBI effectively encourage and support its investigators to collaborate with strategic partners to pursue the early translation of their HLBS discoveries into new diagnostics and therapeutics? The critical challenges to effective early translation of discovery science experienced by the investigator community include: 1. The need for translational skills development, training and guidance 2. Need for ...more »

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Goal 3: Advance Translational Research

Submitted by (@jnoel0)

DEVELOPMENT OF BIOMARKERS FOR SLEEP INSUFFICIENCY, CIRCADIAN DISRUPTION AND SLEEP DISORDERS

There is an urgent need to develop quantifiable biomarkers for acute sleep loss, chronic sleep insufficiency, circadian disruption and sleep disorders such as obstructive sleep apnea. These problems are highly prevalent but currently we do not have biomarkers to use for case identification, prognosis, or assessing response to therapy. There are currently small studies that indicate the feasibility. A recent workshop ...more »

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179 net votes
240 up votes
61 down votes
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Goal 3: Advance Translational Research

Submitted by (@rjjones)

Allogeneic transplantation as a safe and universally available therapeutic strategy for treating non-malignant blood diseases

Can new advances in allogeneic blood or marrow transplantation (BMT) make the procedure a safe and universally available therapeutic strategy for treating non-malignant blood and immune disorders such as sickle cell anemia, thalassemia, aplastic anemia, and severe combined immune deficiency?

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164 net votes
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Goal 3: Advance Translational Research

Submitted by (@jnoel0)

NEW INFRASTRUCTURE FOR CLINICAL RESEARCH IN SLEEP AND CIRCADIAN DISORDERS

Much of the current clinical research on sleep and circadian research depends on cohorts designed for other purposes. While this has been helpful, such studies have limitations. These limitations are related to availability of in-depth phenotyping data and questions as to whether individuals identified in population studies are equivalent to those who present clinically with specific disorders. These concerns could ...more »

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126 net votes
186 up votes
60 down votes
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Goal 3: Advance Translational Research

Submitted by (@giralts)

Can we leverage exisiting registries to perform prospective trials and advance reduce the cost of doing research?

Current costs for multicenter randomized or non randomized trials are astronomical, and a major obstacle to rapid implementation of potential lifesaving discoveries. In the field of hematopoietic cell transplantation (HCT) their is a federal mandate to have a treatment outcome registry. Funds should be made available to leverage that registry to perform prospective trials either randomized or not since HCT programs need ...more »

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123 net votes
154 up votes
31 down votes
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Goal 3: Advance Translational Research

Submitted by (@ewong0)

Novel Cell Apheresis Technologies to Treat Hematologic Diseases

Current FDA approved apheresis technology uses elutriation/centrifugation or filtration separation techniques to remove pathologic cellular and/or plasma elements. Currently these techniques are non-specific, limited by inefficient removal kinetics and often require considerable blood product exposure. Despite tremendous improvement in our understanding of the pathophysiology of a variety of disease, our ability to ...more »

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118 net votes
139 up votes
21 down votes
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Goal 3: Advance Translational Research

Submitted by (@zbigniew.m.szczepiorkowski)

ESTABLISHMENT OF APHERESIS MEDICINE CONSORTIUM TO ADVANCE DEVELOPMENT OF EVIDENCE BASED THERAPIES

The apheresis medicine encompasses treatment of numerous diseases many of which are directly related to blood, lung and heart. There is a need to establish consortia for Apheresis Medicine to facilitate networking, information exchange and research collaboration among investigators, including junior investigators. These consortia would perform basic science as well as translational research and investigate the best pathways ...more »

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112 net votes
131 up votes
19 down votes
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Goal 3: Advance Translational Research

Submitted by (@kkomanduri)

How can we develop more selective immunosuppression for allogeneic hematopoietic cell transplantation?

Graft versus host disease (GVHD) remains the most significant complication of allogeneic hematopoietic stem cell transplantation (HCT). While the use of HCT has grown significantly safer and has demonstrated broad efficacy in the setting of a broad range of blood diseases, immunosuppressive therapy has not dramatically evolved since the introduction of calcineurin inhibitor-based approaches decades ago. The availability ...more »

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106 net votes
129 up votes
23 down votes
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Goal 3: Advance Translational Research

Submitted by (@nhlbiforumadministrator)

Better disease models

Many diseases of the heart, lung and blood systems are studied using animal models, often with genetically engineered mice. However, while mice get models, humans get diseases. Too many grants are devoted to curing models, a practice encouraged by many high profile journals who want to see “proof” in a standard model of disease. Much less time, effort and money will be wasted on developing ineffective therapies if focus ...more »

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97 net votes
126 up votes
29 down votes
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Goal 3: Advance Translational Research

Submitted by (@zbigniew.m.szczepiorkowski)

TREATMENT OF SEPSIS-MULTIPLE ORGAN DYSFUNCTION SYNDROME (MODS) UTILIZING APHERESIS BASED STRATEGIES

Sepsis, a systemic inflammatory response to infection, is the most common cause of death in non-cardiac intensive care units. The incidence and severity of sepsis have increased over the last two decades. With advances in supportive care, sepsis carries a mortality that averages 17%, however, this figure increases to 50 - 80% in Multiple Organ Dysfunction Syndrome (MODS), defined as failure of 3 or more organ systems. ...more »

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97 net votes
115 up votes
18 down votes
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Goal 3: Advance Translational Research

Submitted by (@ewong0)

Development of Novel Apheresis Adsorption Technologies to More Effectively and Safely Treat Hematologic Diseases

Current FDA approved apheresis technology currently uses elutriation/centrifugation separation techniques to remove pathologic cellular and/or plasma elements. These techniques are non-specific, limited by inefficient removal kinetics and often require considerable blood product exposure. Despite tremendous improvement in our understanding of the pathophysiology of a variety of disease, our ability to treat many of ...more »

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93 net votes
112 up votes
19 down votes
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