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

Follow-up care for newborns diagnosed with sickle cell trait or disease

• There is a need to develop and support formal programs to provide follow-up care for newborns who test positive for the sickle cell trait or sickle cell disease upon screening. While newborn screening programs exist nationwide, healthcare providers report that often, screening is conducted only upon request (likely related to cost) and there is usually no follow-up afterwards. Interventions are also needed further ...more »

Submitted by (@coretta.jenerette)

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 : International Association of Sickle Cell Nurses and Physician Assistants, Inc.

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14 net votes
17 up votes
3 down votes
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Goal 3: Advance Translational Research

Xenotransplantation is the future and ideal treatment for end stage organ failure

A genetically engineered and humanized pig organ expressing human genes and devoid of antigens immunogenic to humans may prove to be an ideal solution to overcome the acute organ shortage for solid organ transplantation. Recently, a remarkable progress has been made in this direction with reports of graft survival approaching 3 yrs in a pig to primate cardiac transplantation model.The issues of viral transmission and ...more »

Submitted by (@mmohiud2)

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

Details on the impact of addressing this CQ or CC :

Over 150,000 patients are currently waiting for organ transplantation in USA alone. Unfortunately most of these patients will die waiting. The current options available to prolong life in these patients are limited and are not perfect. Despite of all the awareness for organ donation we will never be able to match the overwhelming need fir the organs. Using organs from a non human source seems to a very valid solution. Pigs have been chosen due to our ability to genetically modify them, their short breeding cycle, similar anatomy to humans, no known transmission of disease from pig to human and their widespread consumption in our diet diminishing many ethical concerns. We have made a tremendous progress in xenotransplantation field and with enough support this treatment for end stage organ disease can become a clinical reality very soon.

Feasibility and challenges of addressing this CQ or CC :

Due to advances in the field of genetic engineering it has now become possible to produce humanized pigs which can be used as organ donors for humans and their organs do not induce a strong immunogenic response . The reaction to remaining xeno antigens could be easily suppressed by minimal immunosuppression. Perhaps with even lesser immunosuppression than what is used for human allotransplantation. The main challenges for this treatment are mostly related to the ethical concerns which are also being addressed by many in the field.

Name of idea submitter and other team members who worked on this idea : Muhammad M Mohiuddin

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20 net votes
29 up votes
9 down votes
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Goal 3: Advance Translational Research

Palliative and hospice care for COPD patients

Does palliative care and/or hospice care as practiced across communities improve end-of-life care for COPD – specifically, does it reduce the burden of symptoms, improve HRQoL and satisfaction, reduce utilization in last 6 months of life (i.e. hospital visits, cost, invasive ventilation use, etc), improve the end-of-life experience, and increase the concordance of place of death to expressed patient preferences?

Submitted by (@k.willard)

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

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12 net votes
16 up votes
4 down votes
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Goal 3: Advance Translational Research

Spinal Cord Injury: hype, unmet promises, and misery which does not need to be

Research to "fix" spinal cord injury in humans, has been insanely hyped, rare in reality, and very disappointing in its clinical applicability to human patients. After a parade of rat models, mouse models, cat models, dog models, African green monkey models, pig models, guinea pig models, hamster models, rabbit models, gerbil models, etc. one wonders whether most researchers or funders will ever have any interest in ...more »

Submitted by (@mgwmgw)

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

Details on the impact of addressing this CQ or CC :

So, how about we put a moratorium on FDA approval of all research related to a cure for spinal cord injury when done by any persons or organizations who have not published every single one of their past experiments in the time required, and for any research which involves other species than humans.

 

Also, how many different ways of creating stem-like cells do we need? Let's stop creating stem-like cells and start applying the ones we have to human patients.

 

How about spending the animal model money instead on improving the quality of life for people living with disabilities. Let us start with actually enforcing the ADA on all new enough buildings.

 

When we make technology for doctors to use, we consult doctors. When we make technology for teachers to use, we consult teachers. When we make technology for disabled people to use… we consult insurance companies, and medical professionals who are not and have never been disabled. We fail to apply the most basic usability testing to the tools which disabled people must use. For example, has any wheelchair designer tried to propel a manual wheelchair uphill on wet grass? How about across a cobblestone street? or down a normally bumpy sidewalk? Now imagine that your butt has atrophied and you are sitting on your hip bones. How painful would that be? Now remember that pressure sores resulting from this bad design can be fatal, and then tell me why we do not take this more seriously.

Feasibility and challenges of addressing this CQ or CC :

The misery which does not need to be is not a new idea: http://badcripple.blogspot.com/2015/01/obsession-with-walking.html

 

Let's get the price of tools for disabled people down to the point where most patients can really afford them, or where the insurance can actually cover them. Let's get exoskeletons price-competitive with wheelchairs, for example, instead of using them to make soldiers able to carry heavier packs in war.

Name of idea submitter and other team members who worked on this idea : Mary-Anne Wolf (inspired by the Bad Cripple blog of William Peace and by the Wheelchair Driver website forum)

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-7 net votes
5 up votes
12 down votes
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Goal 3: Advance Translational Research

Develop an Effective and Functional Biological Pacemaker

There is a need to develop a biological pacemaker for pediatric patients that would react to neurohumoral factors that normally modulate heart function, as well as adapt to the growing heart.

Submitted by (@nhlbiforumadministrator)

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

Details on the impact of addressing this CQ or CC :

Reduce risks associated with the increasing use implantable pacemakers. Increase reliability of artificial electrical pacemakers.

Feasibility and challenges of addressing this CQ or CC :

Animal studies have already demonstrated feasibility of cell- and gene-based as well as hybrid approaches.

The introduction of implantable medical devices using electrical impulses through electrodes placed in the heart to regulate its beating in patients whose native cardiac pacemakers fail— i.e., implantable electronic pacemakers— have permitted hundreds of thousands of individuals to live extended, relatively normal lives. Many advances since the introduction of implantable pacemakers into medical practice during the latter half of the 20th century have improved reliability, but their use still carries significant risks; e.g., lead fracture, infection, malfunction, and the need for replacement.

To date experimental cell therapy, gene therapy, and hybrid approaches have been used to create biological pacemakers in animal models. These incorporate the use of human embryonic stem cells or induced pluripotent stem cells or overexpression of the transcription factor, TBX18, to produce functional biological pacemakers in large animal models. Other gene therapy approaches have also been used to generate functional biological pacemakers in animals. These include overexpression of ion channels impacting diastolic membrane depolarization and excitability in non-pace making regions of large animal hearts. Beta-2 receptor or adenylyl cyclase overexpression represent other strategies that have been employed. Finally, a hybrid approach has used human mesenchymal stem cells loaded with the pacemaker gene HCN2is to induce pacemaker activity in large animals. Thus multiple approaches exist and collaboration is needed between investigative groups to overcome the challenge of creating and testing an effective and reliable biological pacemaker in humans.

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

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8 net votes
23 up votes
15 down votes
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Goal 3: Advance Translational Research

Build a National Surveillance of Chronic CV and Lung Diseases

There is a need to build a robust coordinated surveillance system on the incidence and prevalence of chronic diseases. Surveillance data are needed to: •Describe and monitor the burden, trends, and patterns of these diseases •Set parameters and metrics of research priorities •Identify where to target resources for prevention, treatment, and delivery of care •Track and monitor progress toward public health disease ...more »

Submitted by (@nhlbiforumadministrator)

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 high prevalence of chronic cardiovascular and lung diseases has created burden in increasing healthcare costs and high mortality rates in the US compared to other developed countries. Even so, they remain among the most preventable health problems. A national surveillance system for chronic cardiovascular and lung diseases would enable data-driven decision-making about public health strategies for prevention, management, and cost containment.

Feasibility and challenges of addressing this CQ or CC :

A 2011 Institute of Medicine (IOM) report concluded that a coordinated surveillance system is needed. It proposed a framework for such a system that would integrate existing information through collective efforts of multiple stakeholders. The time is right to gain from and build upon numerous ongoing broad initiatives in biomedical Big Data, including growing health IT adoption mandated by the HITECH Act, ONCHIT efforts to achieve health IT interoperability, the NIH BD2K initiative, and the multiorganizational network participating in FDA Mini-Sentinel, HCS Collaboratory, and PCORnet, among others. The NHLBI is well-positioned to lead, develop and implement the IOM’s recommended framework and system. (IOM report - http://www.iom.edu/Reports/2011/A-Nationwide-Framework-for-Surveillance-of-Cardiovascular-and-Chronic-Lung-Diseases.aspx)

Existing data sources (i.e., population surveys, registries, cohort studies, administrative data, and vital statistics) do not individually provide nationally representative data, cannot be linked, and are not currently readily accessible to all levels of users. One potential way to build such a system is to integrate and expand existing data sources.

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

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5 net votes
13 up votes
8 down votes
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Goal 3: Advance Translational Research

Early prediction of cardiovascular disease by primary-care assessment

Tools for early assessment of cardiovascular disease have become available but not adopted in primary-care settings. Increased arterial stiffness is a well-known marker for advanced cardiovascular disease (CVD) and has been shown to be an independent predictor of cardiovascular mortality. In addition, arterial pulse wave velocity (PWV) has been readily accepted as a measure of arterial stiffness. Despite significant ...more »

Submitted by (@roy.wallen)

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

Details on the impact of addressing this CQ or CC :

In the US, 84 million adults will see their primary care physician for treatment of cardiovascular disease (CVD). CVD is responsible for an average of one death every 40 seconds. The direct and indirect costs of cardiovascular disease and stroke are approximately $315 billion, including the cost of health care services, medications to treat high blood pressure, and missed days of work. The World Health Organization states that 80% of premature heart disease and stroke is preventable. Focusing on assessing risk factors for cardiovascular disease, screening for individuals at risk, and then providing effective and affordable treatment to those who require it can prevent disability and death and improve quality of life.

 

In Europe, the European Society of Cardiology (ESC) has issued guidelines based on the weight of evidence in favor of the usefulness of screening for CVD by assessing arterial stiffness. These guidelines are supported by nonrandomized trials and suggest the development of randomized trials or meta-analyses. However, no guidelines exist in the US for screening for arterial stiffness from such organizations as the American Heart Association (AHA) and the American College of Cardiology (ACC). Existing guidelines to include assessment of cholesterol, lifestyle, obesity, and factors for risk are important. However, a simple, low-cost, objective measurement could be implemented at the point of primary care to improve early detection and treatment of CVD.

Feasibility and challenges of addressing this CQ or CC :

Screening capabilities and some level of clinical evidence exist for early detection of CVD. Therefore, implementation of a practice guideline in the US is very feasible. Studies and assessment from existing data such as have been completed by ESC can be replicated in the US and promulgated by AHA and ACC. This effort will require support from public and private entities, including universities, in order to see practice standards implemented.

 

Challenges to date include funding and the application of clinical protocols to support randomized studies or meta-analyses that will provide evidence for benefits of early screening. Further, public policy and current funding are focused on treatment rather than prevention. Existing reimbursement established by the Centers for Medicare & Medicaid Services (CMS) is focused on treatment rather than prevention and private insurance carriers have followed this same policy. Broader clinical study will support both the adoption of screening tools in primary care and broader reimbursement policy.

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

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5 net votes
8 up votes
3 down votes
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Goal 3: Advance Translational Research

Better preclinical models that model phenotypes/endotypes of human disease.

There is an urgent need for better preclinical models that model phenotypes/endotypes of human disease.

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 : Research Advocacy Committee, American Thoracic Society

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

Predicting COPD exacerbations and relapse

What measures other than PFT data can be used to predict risk of 1) COPD exacerbations (e.g., hospitalization, urgent care visit, or ED visit for COPD exacerbation) or 2) relapse (e.g., re-hospitalization, urgent care visit, or ED visit) following hospital discharge after treatment of COPD exacerbations?

Submitted by (@k.willard)

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

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14 net votes
16 up votes
2 down votes
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Goal 3: Advance Translational Research

Increasing Regenerative Medical Strategies in Pulmonary Arterial Hypertension

Pulmonary arterial hypertension (PAH) is a complex, progressive condition characterized by high blood pressure in the lungs and restriction of flow through the pulmonary arterial system. Current PAH therapies mainly act of the vasoconstrictive component of the disease; however there is a widely accepted view that another contributor to the disease is an abnormal overgrowth of cells that line the pulmonary arteries, which ...more »

Submitted by (@michaelg)

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 the past twenty years, 12 PAH targeted-therapies have been approved by the FDA. This increase in disease state awareness and in the treatment armamentarium have contributed to an increase in average survival from 2.8 years to an estimated 8-10 years. However, current treatments primarily address the vasoconstrictive component of the disease and do not address the now accepted theory of post-apoptotic overgrowth of hyperproliferative cells of the pulmonary vessels. A number of circulating stem and progenitor cells, derived from the bone marrow, have been identified that could have roles in repair of the pulmonary vascular system when interacting with the quickly, abnormally growing cells in the lung vessels. Work in this area has been named as a future research opportunity in the NHLBI-ORDR Strategic Plan for Lung Vascular Research (Erzurum S, et al. 2010).

Feasibility and challenges of addressing this CQ or CC :

Basic and translational research support is needed—including high-throughput approaches such as phage display and large-scale proteomic analysis—to better understand the relationship between circulating bone marrow-derived cells, lung-resident stem and progenitor cells, and endothelial cells of the pulmonary arterial system.

Name of idea submitter and other team members who worked on this idea : Pulmonary Hyeprtension Association, Michael Gray, Katie Kroner

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71 net votes
81 up votes
10 down votes
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