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 3: Advance Translational Research

Accelerating Translational Research

NHLBI should define a strategy to promote collaborative research between clinician-scientists who perform patient-oriented research, and basic scientists who focus on the preclinical realm. There is not enough cross-talk between these two groups, and yet much to be gained from increasing interactions between the two (e.g. accelerating the translation of bench science findings into the clinic). In particular, funding strategies ...more »

Submitted by (@golan0)

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

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Goal 4: Develop Workforce and Resources

Bridge “translational gap”

Provide resources and training to improve the ability of scientists to bridge the “translational gap”. Continue and expand the VITA program.

Submitted by (@societyforvascularsurgery)

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 : Society for Vascular Surgery

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Goal 4: Develop Workforce and Resources

Training for radiologist researchers for effective translational research

Critical Challenge

Submitted by (@str0001)

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

Details on the impact of addressing this CQ or CC :

As targeted therapy and molecular mechanisms of disease are emerging, a mechanism to improve the ability of radiologists to perform translational research is crucial. Such knowledge is essential for collaborative multidisciplinary research that ultimately leads to imaging as disease-specific diagnostic and therapeutic tools to combat pulmonary and cardiovascular disease.

Feasibility and challenges of addressing this CQ or CC :

Knowledge in the molecular mechanisms of disease and the potential for imaging technology to advance via targeted imaging agents, positron emission tomography (PET), functional MR methods, PET/computer tomography, and PET/MR is increasing. The radiologist has in depth expertise within imaging technology, performance of studies, and diagnostic abilities of imaging techniques. A program directed towards developing imagers towards translational imaging research will include in-depth education and training in lung physiology, pulmonary disease mechanisms, multimodality imaging bridging CT, PET/CT, MR and PET/MR, and the molecular techniques. With such knowledge and training, radiologists will be prepared to serve as principal investigators and collaborators in multidisciplinary teams. An understanding of imaging technologies and their capabilities, the clinical challenges, and molecular techniques will enable imagers to provide innovative solutions to diagnostic dilemmas in pulmonary and cardiovascular disease.

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

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Goal 3: Advance Translational Research

Infrastructure for human translational research

With the reduction in NCAT support for human translational research, infrastructure support will need to come from the NHLBI. This will increase the cost of most human, mechanistic based RO1 studies by 20-30%. This will exceed the current cap of $500K in many circumstances. The cap will need to be raised or NHLBI and other institutes need to determine how NIH can continue to provide this critical infrastructure.

Submitted by (@gwilliams)

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

Details on the impact of addressing this CQ or CC :

With the pending loss of infrastructure support by NCAT for human translational, mechanistic studies, a contiued decline in resources to support this critical resources for N of 1 studies. With appropriate support there will be increased capacity to determine which pre-clinical data is applicable to humans and to design more percise, mechanism based clinical trials to increase the likelihood of precision, personalized medicine for many of NHLBI's targeted diseases, e.g, hypertension, stroke, cardiovascular disease with diabetes and hypertension, asthma, and sleep apnea.

Feasibility and challenges of addressing this CQ or CC :

The template for addressing this challenge is already available. The specific funding mechanism(s) will need to be addressed.

Name of idea submitter and other team members who worked on this idea : Gordon Williams, Gail Adler, Charles Czeisler, Ellen Seely, Lindsey Baden

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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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Goal 4: Develop Workforce and Resources

Establishment of an independent study section on Pulmonary Vascular Biology and Translational Research

The research on pulmonary vascular biology including smooth muscle cell biology and endothelial cell biology and related pulmonary vascular diseases such as pulmonary hypertension and related right heart failure, and endothelial dysfunction in lung vascular inflammation and acute lung injury, as well as pulmonary embolism and lung transplantation has been rapidly expanding. The field is attracting an ever increasing ...more »

Submitted by (@yyzhao)

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

Details on the impact of addressing this CQ or CC :

Establishment of a study section on Pulmonary Vascular Biology and Translational Research will provide adequate funding to stimulate innovative research on this rapidly expanding field and promote translational research and thereby promote human health by providing potential novel therapeutic strategies for the devastating diseases such as pulmonary hypertension and acute lung injury.

Name of idea submitter and other team members who worked on this idea : Youyang Zhao, Kurt Denmark, Asrar B. Malik, Mark Gladwin, Jahar Bhattacharya, Michael Matthay, Sharon Rounds, Jason Yuan

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Goal 4: Develop Workforce and Resources

Training of Clinical & Translational Scientists

Although the NCRR and NIGMS used to have a mechanism to train new generation of clinical & translational scientists, this program was stopped. Why?

What is the possibility of other institutes to come up with the priority of funding resources in this regard?

Submitted by (@dkagr0)

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

Details on the impact of addressing this CQ or CC :

In view of the health care models, strong control of insurance companies in determining the remuneration, lack of protective time for qualified clinicians to continue their research, no incentive to the institute for promoting such activities, lack of available tenure-track jobs, pool of effective and well-trained clinical & translational researchers is decreasing rapidly. Even though NIH invests resources to train MD-PhD students, a very minor pool of these graduates continue curiosity and passion in advancing new knowledge and discovering newer approaches.

Feasibility and challenges of addressing this CQ or CC :

1. Additional resources must be developed by NHLBI, NIAID, NIDDK and other major institutes to support this endeavor.

2. Institutes/medical schools who provide protective time to their faculty to continue their efforts in clinical & translational research, must be acknowledged and incentivized.

3. There has been no effective way of measuring outcomes from such investments. All of us must take ownership in utilizing the resources more effectively and more productively.

Name of idea submitter and other team members who worked on this idea : Devendra K. Agrawal, PhD

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38 up votes
7 down votes
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Goal 3: Advance Translational Research

Incentivizing Translational Research

Support for scientific research depends on making a compelling case that we contribute to the health of Americans and the health of the US economy. This idea is to address the critical challenge of making basic research relevant to the lives of Americans by incentivizing NHBLI researchers to engage meaningfully in translational research.

Submitted by (@tomtherramus)

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 specific proposal is to give a 5 to 10 percentile bump (similar to that given to junior investigator) to researchers whose NIH funding has led to translational outcomes that are of tangible benefit to the health of Americans and/or the US economy.

 

Categories that would meet the translational bump might include:

1. A clinical trial based on their basic or clinical research;

2. Generation of a device, drug or other therapy that has entered cllinical testing;

3. Granting of a patent that has been licensed by a company,

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

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Goal 1: Promote Human Health

Transformative Impact of Proteomics

The proteomics field has dramatically progressed over the past 20 years, with advancements and improvements in experimental designs and sample preparation protocols, as well as mass spectrometry equipment, approaches, and analysis. This has resulted in substantial forward progress towards a proteomic pipeline to establish cause and effect mechanisms of cardiovascular disease. There is a need for CV proteomics that resolve ...more »

Submitted by (@mllindsey)

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 necessary tools have been assembled, and managing implementation will reduce the time required for completion of larger scale projects.

Feasibility and challenges of addressing this CQ or CC :

high feasibility; the challenge will be managing communication across groups to maximize impact

Name of idea submitter and other team members who worked on this idea : Merry Lindsey

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Goal 4: Develop Workforce and Resources

Promoting Translational Research training using the R03 mechanism

We in Wisconsin have developed a robust training program for mixed MD and PhD postdocs in a clinical dept and yet their future in research is blocked by few faculty positions, poor funding and over complicated NIH applications. One of the simplest grants is the R03 and we have already had graduates with only a few years postdoc experience succeed in gaining R03 funding. The R03 is a very simple very flexible mechanism. ...more »

Submitted by (@ianbird)

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

Details on the impact of addressing this CQ or CC :

More MD and PhD Postdoc trainees will spend more time focused on work and less writing grants - and the efficiency of expenditures by NIH would go up in terms of paper generated per dollar. MDs who may have a desire to dip in and out of funded research as their clinical path demands would be able to do that. R03 funding does not at any time change your status as a new or early career investigator. Its perfectly reasonable to hold more than one even without a faculty title but as a PhD scientist or practicing clinciian.

Feasibility and challenges of addressing this CQ or CC :

Its easy to do - just decide to do it. Also writing an R03 is the best possible training for writing an R01. I run a T32 for predocs and I mentor K12 postdocs. I know what NIH INTENDS in its training grants. But also recognize just to take on the longer and longer and more demanding K application after already being well trained is a real disincentive to everyone, even if those pages in training plan are well intentioned. IF omeone has not had the chance to train or is cross training to a new area then a K99 is perfect. But someone who is well trained, may stay in their area and has already proven themselves needs a fast submission fast review process to get funding NOW that may be the difference between continued employment though this funding crisis or not. This could provide that for them. Especially with expedited review!

Name of idea submitter and other team members who worked on this idea : Ian Bird

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Goal 4: Develop Workforce and Resources

Translational training programs

The strategic vision to enhance translation and to enhance the workforce both require training that spans the scope of basic science, pre-clinical development, clinical trials. We lack coherent mechanisms for training the next generation of translational researchers, some of whom may be MDs, and some PhDs. A program should provide cross-training of Clinical Fellows and Postdocs to reflect the needed interactions between ...more »

Submitted by (@wjones7)

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 impact will trainees with more comprehensive exposure and involvement in translation of science from the bench to bedside. MDs will spend more time in labs or involved in pre-clinical work, PhDs will become CITI certified and assist with enrollment of clinical trials and trial design. Journal clubs will span the sciences, the clinical practice and the translational realm including regulatory and industry considerations. Trainees can use this background whether they go on in medicine, science, translation, or industry to fit and contribute to an increasingly translational medical bioscience field.

Feasibility and challenges of addressing this CQ or CC :

Feasibility must include a academic medicine environment active in translational biomedical science such that the mentors can include scientists, physicians and physician/scientists, some of whom are translators. Some of the scientists should be from industry and perhaps projects and funding can involve industry/Pharm as well these will benefit from an educated workforce. Challenges involve individuals at the sites putting the right teams together, but many Universities are doing this with incubators and translational units at present. This will further the clinical involvement to include Fellows in Fellowship programs in Cardiology, Medicine and Surgery.

Name of idea submitter and other team members who worked on this idea : Keith Jones

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