Goal 3: Advance Translational Research

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

Goal 3: Advance Translational Research

Animal Models for Translational Research and Drug Development

There is a need to identify and develop suitable animal models (e.g. larger, non-primate animal models) that faithfully predict the outcomes of new medicines and treatments in heart, lung, blood, and sleep (HLBS) disorders prior to human clinical trials.

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73 net votes
92 up votes
19 down votes
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(@nhlbiforumadministrator)

Goal 3: Advance Translational Research

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 »

Voting

196 net votes
226 up votes
30 down votes
Active
(@nhlbiforumadministrator)

Goal 3: Advance Translational Research

Bone Marrow Stem Cell Transplant in Peds sibling matched SCD

There is a need to improve accessibility of Bone Marrow Stem Cell Transplantation (BMSCT) for Sickle Cell Disease patients who are most likely to benefit from this treatment option. 1. Building a culture of trust between and among primary care providers, specialists, patients/families, and other stakeholders 2. Consensus building around BMSCT as an acceptable treatment alternative (as opposed to another research endeavor)... more »

Voting

52 net votes
80 up votes
28 down votes
Active
(@nhlbiforumadministrator)

Goal 3: Advance Translational Research

Application of data science to extract information from EHR/EMR

There is a need to utilize bioinformatics to analyze electronic health records / electronic medical records (EHR/EMR) documentation of practice-based pulmonary and sleep medicine to better understand longitudinal care delivery patterns and outcomes, identify changes in evidence, inform practice, and foster reverse translation to integrate basic and clinical research.

Voting

54 net votes
81 up votes
27 down votes
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(@nhlbiforumadministrator)

Goal 3: Advance Translational Research

Expediting Gene and Cell Therapies to the Clinics

What methodologies will best enhance the translation of technologies for gene and cell therapies into potential products for clinical application and commercial development? In considering a strategy for NHLBI investment in gene therapy, it is important to note that we are only at the beginning of a revolution that will eventually impact biomedical research across a broad range of specialties. NHLBI/NIH needs to create... more »

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66 net votes
97 up votes
31 down votes
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(@nhlbiforumadministrator1)

Goal 3: Advance Translational Research

Public-Private Partnerships to Improve Nutrition & Reduce Obesity

How can we best encourage and support collaborations between academic researchers and industry partners to test strategies for changing nutritional choices and eating behaviors to healthier patterns that can improve obesity rates? Subquestions include issues around adopting healthier food choices in a variety of environments, effects of diet alteration, the methods and effects of enhancing healthier food flavors, understanding... more »

Voting

54 net votes
96 up votes
42 down votes
Active
(@nhlbiforumadministrator1)

Goal 3: Advance Translational Research

Arrhythmia Therapies Based on Understanding Mechanisms

There is a need to translate these new insights of genetic, molecular, cellular, and tissue arrhythmia mechanisms into the development of novel, safe, and new therapeutic interventions for the treatment and prevention of cardiac arrhythmias.

Voting

51 net votes
86 up votes
35 down votes
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(@teresabarnes)

Goal 3: Advance Translational Research

Sustainability and Continuation of Research

While many challenges to advancing research efforts exist, a particularly confounding one for patient advocates is when promising research efforts are funded by NHLBI and the research is completed and published (leading to more questions) and is seemingly forgotten. This lack of continuity leads to further questions and is absent a solid path forward applying what has been learned to next steps in the research effort.... more »

Voting

87 net votes
106 up votes
19 down votes
Active
(@nhlbiforumadministrator)

Goal 3: Advance Translational Research

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 »

Voting

97 net votes
126 up votes
29 down votes
Active
(@rjjones)

Goal 3: Advance Translational Research

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?

Voting

164 net votes
214 up votes
50 down votes
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(@giralts)

Goal 3: Advance Translational Research

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 »

Voting

123 net votes
154 up votes
31 down votes
Active
(@akuaban)

Goal 3: Advance Translational Research

Genome Editing and Gene Therapy

There is a critical need for the establishment of strategies that will determine the efficacy, safety, and toxicity of genome editing techniques specifically in hematologic diseases.

Voting

69 net votes
87 up votes
18 down votes
Active