Goal 3: Advance Translational Research

Adaptive trial design for clinical research

Will adaptive trial design improve clinical research for acute lung injury?

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 : American Thoracic Society member

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1 net vote
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Goal 3: Advance Translational Research

Implementation of checklist to improve outcomes of COPD patients with acute exacerbations

Implementation of checklist to improve outcomes of COPD patients with acute exacerbations

 

• Does implementation of a checklist designed to improve the care of patients with COPD exacerbations result in greater adherence to guideline recommendations?

 

• Does implementation of a checklist designed to improve the care of patients with  COPD exacerbations results in improved patient outcomes?

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 member

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3 net votes
3 up votes
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Goal 3: Advance Translational Research

Supporting large cohort studies over many years

How do we support large cohort studies over many years in terms of retention of subjects, manpower, funding, and appropriate outcome tools?

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 : Research Advocacy Committee, American Thoracic Society

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3 up votes
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Goal 3: Advance Translational Research

Translational research supporting stem cell therapy for cardiovascular disease

Translational research supporting stem cell therapy for cardiovascular disease, including: core laboratories for preclinical IND-enabling studies (e.g., PACT), and clinical trials networks for evaluating promising new treatments (e.g., CCTRN).

Submitted by (@judith.l.bettencourt)

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 most cost effective scientific procedure ever utilized to answer the risk benefit question posed by a new intervention to be used in humans is a clinical trial. Major clinical trials are their most effective when planted in controversial ground (MRFIT, CAST, ALLHAT). Like these studies, which were caught in a controversial dynamic of uncertainties and disparate sets of expectations, a clinical trial network to assess cell therapy is precisely what is needed.

Experienced researchers recognize the current inimical environment of cell therapy. Now - as before - some forces argue that new therapy offers no benefits, while other equally vehement constituents contend that the benefits of therapy are so great, and the risks so small, that the treatment requires little if any regulation and should be available at once to the US public. Each side provides thunder, but little light.

It is precisely in this contentious environment where passions argue beyond the data that clinical trials are required. Their construction of the most objective view of the strengths and weaknesses of the intervention comes at a cost, but the answers these well designed and concordantly executed studies provide is the clearest illuminations of the benefits and risks of human cell therapy.

Feasibility and challenges of addressing this CQ or CC :

Based on the unmet clinical needs in the treatment of cardiovascular disease and the compelling early evidence for the promise of cell therapy, NHLBI created the Cardiovascular Cell Therapy Research Network in 2007. Now in its ninth year, the Network has completed three major clinical trials in cell therapy. It has published 35 manuscripts in prestigious clinical journals including JAMA, Circ, and Circ Research. Its biorepository has published two manuscripts relating baseline phenotype findings to measures of left ventricular function. A fourth clinical trial is underway assessing the effect of cell therapy on peripheral vascular disease. The Network is also proceeding with the largest effort to assess the effect of CSC cells in patients with heart failure - the first clinical trial that will assess the effect of combined cell therapy in heart failure patients. In addition, CCTRN will study the effect of allogeneic mesenchymal stem cells in patients with anthracycline-induced cardiomyopathy. Each of these protocols is NHLBI and FDA approved.

CCTRN’s reputation of conducting and then promulgating the results of high quality clinical trials makes it the most effective mechanism to assess the benefits of cell therapy in cardiovascular disease. It is important to continue to fund the infrastructure already in place to ensure its continued high quality operation and its place as the cornerstone of cardiovascular clinical cell therapy research in the United States.

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149 up votes
34 down votes
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Goal 3: Advance Translational Research

Translation of scientific information into clinical and public health asthma and allergies practices and programs

More evidence-based, scientifically proven interventions to ensure that scientific information is translated into clinical and public health practices and programs to reduce the burden of asthma and allergies on individuals, families and society

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 : Asthma and Allergy Foundation of America (AAFA)

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1 net vote
1 up votes
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Goal 2: Reduce Human Disease

Effect of early mobilization

Does early mobilization, i.e. as soon as mechanical ventilation begins, improve long term outcomes in ALI survivors??

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 : American Thoracic Society member

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2 net votes
2 up votes
0 down votes
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Goal 2: Reduce Human Disease

Fibrosis Care Center Network and Patient Registry

Complex diseases such as interstitial lung disease and pulmonary fibrosis requires a collaborative effort to effectively characterize, appropriately diagnose, and efficient evaluate novel therapies. Similarly, basic, translational and clinical research in this field requires the integration of clinical phenotypes with biologic specimens. We propose the expanded development of the Care Center Network and Patient Registry ...more »

Submitted by (@gcosgrove)

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 envisioned impact of an integrated Care Center Network and Patient Registry is to create a resource that:

 

• Informs the understanding of interstitial lung disease (ILD), its epidemiology and natural history;

• Assists to understand treatment patterns associated with optimal outcomes that will inform an emerging standard of care and development of treatment guidelines;

• Facilitates patient and clinician engagement in support of future prospective studies;

• Furthers study of biomarkers and predictors of disease and severity;

• Documents patient experience of living with ILD as described through patient reported outcomes (PRO) including quality of life, functioning, and symptoms;

• Generates new hypotheses and new endpoints in support of future studies;

• Increases awareness of relevant issues and needs among the immediate ILD community;

• Provides the opportunity to promote and inform policies in the larger health care community in support of those with ILD

Feasibility and challenges of addressing this CQ or CC :

With the establishment of collaborations between several partners, we initiated the PFF Care Center Network and Patient Registry in 2014. The Care Center Network and Patient Registry has since expanded to 21 centers regionally dispersed throughout the United States. The challenges of effectively and efficiently investigating the cause, care and treatment of pulmonary fibrosis are predominantly those of organization and integration of effort. Expertise is present throughout the United States. We suggest that with the continued expansion of the Care Center Network and Patient Registry, those challenges will be overcome and the focus of the fibrosis community efforts can be on diligently investigating the diseases that devastatingly affect patients. An integrated repository of well-phenotyped patients and biologic specimens is the first step in Precision Medicine for patients with interstitial lung disease and pulmonary fibrosis.

Name of idea submitter and other team members who worked on this idea : Gregory P. Cosgrove, MD, The Pulmonary Fibrosis Foundation

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Goal 1: Promote Human Health

Improving cardiovascular health

The scientific and clinical ramifications of the transulfurization systems are just beginning to be explored. Research methods and hypotheses should be explored.

Submitted by (@dnelson)

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 transulfuration pathway and H2S play key roles in cellular metabolism and are adversely affected by inflammation and the oxidative stress of chronic disease. Enhancing our knowledge of these systems and the various players would significantly benefit prevention and treatment.

Feasibility and challenges of addressing this CQ or CC :

This is a HOT area and breakthroughs are being announced daily.

Name of idea submitter and other team members who worked on this idea : Deanna Nelson

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-1 net votes
7 up votes
8 down votes
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Goal 2: Reduce Human Disease

Heart Failure with Preserved Ejection Fraction Needs better understanding

Effective treatment for Heart Failure with Preserved Ejection Fraction (HFpEF) currently does not exist. Lack of understanding of underlying mechanism(s) probably contributed to this lack of treatment. The well studied neural-hormonal blockade will not work for HFpEF because down stream kinase targets of adrenergic stimulation enhances myocardial relaxation. Consequently, sustain research outside current main stream thinking ...more »

Submitted by (@ctong0)

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

Details on the impact of addressing this CQ or CC :

Modulation of myosin-actin interaction, energetics, and post translational modification need to be studied in the context of HFpEF. Elucidating underlying mechanism(s) and translating the discoveries toward effective treatment can solve the HFpEF enigma. With aging population, finding effective treatment for HFpEF is sorely needed.

Name of idea submitter and other team members who worked on this idea : Carl Tong

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2 net votes
13 up votes
11 down votes
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Goal 2: Reduce Human Disease

Immunologic Treatment of Hematologic Malignancies

How can the use of CAR T-cell and checkpoint blockade strategies be optimized in order to cure hematologic diseases?

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

Details on the impact of addressing this CQ or CC :

As the body of evidence continues to grow on the potential applications for advanced immunotherapies, next-generation research must focus on addressing the possible curative effects that checkpoint blockades or adoptive CAR T-cell strategies can have for blood diseases including hematologic cancers. This will require specific research programs to fully understand the optimal role for these therapies within the continuum of care. To optimize these strategies for treatment of hematologic diseases, studies are needed to decipher specific hematologic diseases and circumstances under which these checkpoint blockers and CAR T-cell therapies may be employed as frontline approaches. Furthermore, while the optimal approach for these therapies is unclear, advanced studies are needed to elucidate the potential benefit in combining these promising approaches and whether patients can be better identified a priori for these therapies.

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

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13 net votes
28 up votes
15 down votes
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Goal 4: Develop Workforce and Resources

Moderating Positive Feedback between Research Funding and PhD/postdoc Numbers

A critical challenge will be to limit future destabilizing expansion of the number of PhD students and postdoc trainees. Because 75-80% of biomedical PhD students and postdocs now supported by NIH are funded by RO1 and other research funding, if/when research grant budget increase the number of PhD and postdoc “slots” would automatically expand--even if there is no expectation of comparable increases in demand for ...more »

Submitted by (@teitelbaum)

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

Details on the impact of addressing this CQ or CC :

Many including National Academies committees suggest that the 75-80% figure should be gradually reduced to around 50%, without increasing overall numbers, i.e. both limiting numbers supported under research grants and increasing numbers supported under training programs. Moderating the currently strong linkages of PhD/postdoc numbers to research funding should make future biomedical research careers more attractive than they have been in recent years.

Name of idea submitter and other team members who worked on this idea : Michael S. Teitelbaum

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5 net votes
15 up votes
10 down votes
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Goal 4: Develop Workforce and Resources

Training and mentor support in global health

Why can’t all training grants (T, K, etc) be opened out to all people working in US institutions, regardless of citizenship or green-card?

Why can’t we establish mechanisms for US junior investigators and mentors interested in global NHLBI areas?

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 :

Current policy does not allow non-citizens and green-card holders to apply for most K and T grants. However, given the changing nature of US workforce, this policy needs to change. Current mechanisms do not easily enable US investigators to get support for global work……training and/or research.

Feasibility and challenges of addressing this CQ or CC :

Why not?

Name of idea submitter and other team members who worked on this idea : K.M. Venkat Narayan

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4 up votes
17 down votes
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