Goal 2: Reduce Human Disease

Signaling in AVM Developmoent

BMP9 circulates in the blood and signals through the endothelial cell. IN the absence of Alk 1, such as in HHT, the vessels become over-active. The overactivity can be partially balanced by activation of a second signaling pathway: notch. Would targeting notch be a useful drug target to reverse AVM formation

Submitted by (@mariannes.clancy)

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 : Marianne Clancy MPA, Chris Hughes PhD

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

Screening for SDB and Sleep Disorders in School-Aged Chidren by School Nurses

Can school nurses effectively screen for SDB and Sleep Disorders in school aged children? Who else in the school setting could provide such screening? Should such screening be limited to "at risk" children who display identified markers, or be open to all children? What is the role of teachers to "identify" children in need of such screening? What role will such screening serve to mitigate learning, behavioral, developmental ...more »

Submitted by (@nancyh.rothstein)

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 impact of screening at risk children for sleep disorders and sleep disordered breathing, including subsequent referrals and professional treatment, may serve to mitigate future health, learning, developmental and behavioral risks/issues for children by addressing these issues in early childhood. Research based protocols will be accessed and used for screening.

Feasibility and challenges of addressing this CQ or CC :

Additional considerations:

Does the nurse refer an at risk child to the Pediatrician? Dentist? ENT?

What questionnaires or other identifiers would be used for screening? Is there a bio test to assess risk for a SD or SDB?

What should the target age level be for children undergoing proposed screening?

 

How can sleep education and training be integrated with this screening process to promote good sleeping habits/hygiene at home, for all children, parents and caretakers, as well as teachers. Who creates and provides the educational material? Who does the teaching?

 

Parental involvement- KEY!

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

Voting

5 net votes
6 up votes
1 down votes
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Goal 4: Develop Workforce and Resources

Professional Development Pathway

Transformation of the professional development pathway – moving promising trainees and junior faculty to independent and productive investigators– is critical to ensuring a vibrant clinical and basic research workforce.

Submitted by (@skrenrich)

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 : Cystic Fibrosis Foundation

Voting

4 net votes
7 up votes
3 down votes
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Goal 4: Develop Workforce and Resources

Using virtual learning technologies for workforce development

How can we harness virtual learning technologies to address the competency development needs of the modern and future biomedical workforce? Virtual learning tools, e.g. immersive learning simulations and serious games, offer tremendous possibilities for creating engaging and compelling learning experiences for biomedical scientists and providing them with opportunities to practice research skills within the context of ...more »

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 :

Virtual learning technologies have been successfully used to promote workforce competency development in a variety of fields including defense, business, and surgery, particularly in areas that require higher-order cognitive skills, such as critical thinking, problem solving, decision making by simulating the real-life situations and conditions under which these skills are developed. We believe that significant improvements can be achieved in the overall preparedness of the biomedical workforce through the use of flexible virtual training and education tools that can help address the critical competency areas that have the most impact on research success. For instance, considering the team-based nature of most biomedical research efforts requiring effective communication and collaboration of experts from different fields, new workforce development approaches must be grounded in team science. Virtual learning environments provide an excellent opportunity to practice effective teamwork skills within the context of realistic scenarios, virtual and live character interactions, and structured reflection/debriefing exercises. Similarly, harnessing the power of virtual learning tools to train up-and-coming biomedical scientists to think through scientific and organizational challenges in a disciplined yet creative way, leveraging evidence from decades of research on decision making, would help accelerate the expertise development process of a new generation of biomedical researchers

Feasibility and challenges of addressing this CQ or CC :

Virtual learning tools represent a powerful mechanism for activating the principles of problem-based learning and experiential learning through anchored instruction, meaningful contextualization, active participation, intrinsic motivation, and continuous assessment. While there is a growing interest in bringing virtual learning tools to the biomedical science domain, this area remains relatively untapped and calls to advance an understanding of how particular types of instructional strategies and virtual learning tools/resources compare in terms of promoting target competencies, behaviors, and research outcomes in real life, i.e. the learning transfer. This remains a challenge considering the lack of funding opportunities available to explore not only the feasibility of bringing these tools to the biomedical sciences domain but also examining the sustaining effects of the novel training and education interventions going forward. We feel that NHLBI is better positioned than ma ny other NIH entities to address this challenge and spearhead the learning innovation efforts for the biomedical research workforce, as it reaches across a very diverse community of biomedical scientists who need to work together effectively and efficiently to solve the most pressing biomedical and healthcare challenges we face today.

Name of idea submitter and other team members who worked on this idea : Anya Andrews, Ph.D., PMP, Director of Research Initiatives, Associate Professor of Medicine, University of Central Florida College of Medicine

Voting

2 net votes
5 up votes
3 down votes
Active

Goal 2: Reduce Human Disease

Fetal basis for Adult Disease

Maternal exposures during pregnancy have the potential to alter development and lead to lifelong susceptibility to disease. There is epidemiological evidence of this in the asthma field, where maternal smoking leads to increased asthma rates. However, the molecular mechanisms by which maternal exposures cause lung disease later in life are not known and the influence of in utero exposures on susceptibility to lung cancer, ...more »

Submitted by (@dc0000)

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

Details on the impact of addressing this CQ or CC :

Addressing the impact of maternal exposures on fetal lung disease would make a major impact on human health by giving regulatory agencies the data they need to make educated decisions with respect to issues such as nicotine regulation, air pollution, water pollution, and other sources of maternal exposures.

Feasibility and challenges of addressing this CQ or CC :

With the established animal and stem cell models of development, this research is immediately feasible.

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

Voting

10 net votes
17 up votes
7 down votes
Active

Goal 2: Reduce Human Disease

Neurocognitive development and delays in sickle cell disease

Are neurocognitive developmental delays significantly present in children and adolescents living with sickle cell disease? What effect do these delays have on the overall morbidity associated with sickle cell disease?

Submitted by (@sicklecellwarrior)

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

Details on the impact of addressing this CQ or CC :

Conversations among the sickle cell population are increasingly focusing on mental health, neuropsycology as it relates to mental health, and the need to develop community life skills and personal development.

Name of idea submitter and other team members who worked on this idea : Sickle Cell Warriors, Inc. community members

Voting

30 net votes
41 up votes
11 down votes
Active

Goal 3: Advance Translational Research

Substituting scientific-medical insight before profit in drug development

Since the Pure Food and Drug Act of 1906 and the rise of the FDA, the US federal government has directly inserted itself into medical research, primarily from a business perspective. The Orphan Drug Act of 1983 monetarily incentivized the process for rare diseases, but for ultra-rare diseases of < 1,000 patients, it does not work. Successful drugs for rare diseases have enormous price tags to compensate their development ...more »

Submitted by (@mtothbsf)

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

Details on the impact of addressing this CQ or CC :

By harnessing the vast resources of US biomedical research and biomedical researchers, many of whom were trained with NIH funding, the US can more effectively translate scientific discoveries into clinical benefit--many treatments for many diseases using many PIs. While the monetary profit incentive has been a vital aspect of pharmaceutical development over the last 50 years, in this era of personalized medicine it may not be as relevant. The individual PI with a good idea and sound scientific background may hold the key to a cure for a rare or personal disease, but is prevented from translating this because of ignorance, lack of institutional support, or the need for substantial capital investment to perform a proper clinical study. The FDA could recalibrate its requirements for clinical studies to allow even a single PI to test their idea economically, and perhaps offer the funding to do it properly. It should not go unnoticed that by understanding and treating a specific rare disease, the medical world may use this knowledge to better understand and treat diseases that affect larger numbers of people. For example, understanding or treating heart failure in a specific rare disease, may offer benefits to the larger group of heart failure patients.

Feasibility and challenges of addressing this CQ or CC :

Meeting this challenge will require a major rethinking of the traditional FDA approach of drug development and testing in humans at least for the non-profit or single PI scenario. For ultra-rare diseases of < 1,000 individuals there seems little hope that the for-profit sector would choose to be involved. For that reason and to take advantage of the enormous biomedical research resources the the US federal government has sponsored though the NIH, the US could advance translational research and usher in an era of true "personalized medicine'.

Name of idea submitter and other team members who worked on this idea : Matthew J. Toth, PhD, Science Director, Barth Syndrome Foundation Inc.

Voting

6 net votes
11 up votes
5 down votes
Active

Goal 1: Promote Human Health

Intersecting Developmental Biology with Vascular Physiology and Biology

Although many think of the vasculature as a lump sum of vessels that all react in a similar fashion to a certain stimulus, e.g., alpha-adrenergic activation, this is not the situation. For example, coronary resistance vessels show little to no direct response to alpha-adrenergic activation while resistance vessels in most organs show marked constriction. Another example is the response of different vessels to angioplasty ...more »

Submitted by (@wchilian)

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

Details on the impact of addressing this CQ or CC :

A challenge facing many specialists in vascular medicine, vascular surgery, and cardiology is understanding the ramifications, and the basis, of the vascular pathology in the context of the organ system. Another way of re-stating this as a question is: Are the unique attributes of the vascular biology, pathology and physiology of a particular organ system connected to specific aspects of development. This question would help both the basic on clinical scientists understand the basis of why a blood vessel in the kidney may be different than one in the heart, or in the brain with the goal of devising more selective therapies to approach vascular disease in specific organs. Scientists in the area of vascular development have long appreciated that vascular cells in different organs arise from different embryological origins; yet how this information translates into the intricacies of vascular control, or responses to pathology is not resolved. Understanding the basic biological mechanisms of how the embryological source of the vasculature affects pathology and physiology could engender treatment of vascular disorders.

Feasibility and challenges of addressing this CQ or CC :

This idea could be implemented by encouraging multi-PI efforts from vascular developmental biologists, and investigators engaged in studies of microvascular control mechanisms and/or vascular biologists interested in vascular pathologies such as restenosis and vascular lesions. Advances in fate mapping techniques have enabled developmental biologists to track embryological origins of cells into specific organ systems into adulthood. With such a multi-faceted approach a better understanding of vascular physiology and pathophysiology will be obtained that hopefully will be translated into more effective treatments.

Name of idea submitter and other team members who worked on this idea : William M. Chilian

Voting

15 net votes
26 up votes
11 down votes
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Goal 3: Advance Translational Research

Rx for HFpEF

HLBI should make it a priority to develop therapeutic options for the treatment of heart failure with preserved ejection fraction.

Submitted by (@johnrobinson)

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

Details on the impact of addressing this CQ or CC :

Heart failure with reduced ejection fraction (HFrEF), formerly called systolic heart failure, is the classical form of heart failure that is characterized by defective ventricular contraction. The most common variant of heart failure is heart failure with preserved ejection fraction (HFpEF), formerly called diastolic heart failure, characterized by resistance to ventricular filling. The prevalence of HFpEF has been rising steadily over the past two decades at a rate of increase of 1% per year, while the prevalence of HFrEF which has remained stationary. The most common causes of HFpEF are ischemia, obesity, hypertension, diabetes and ageing. Since the population is increasingly obese, hypertensive, diabetic and ageing, the incidence of HFpEF will be the dominant heart failure phenotype over the next decade. The clinical management of HFpEF is complicated by lack of therapeutic options that provide survival benefit. Therapies of proven benefit in HFrEF have repeatedly been shown to add little if any benefit in HFpEF. The prognosis of HFpEF is about the same as HFrEF, with 5-year mortality ranging from 54% to 65%.

Feasibility and challenges of addressing this CQ or CC :

Recent developments in our understanding of the molecular mechanisms of myofilament regulation and assays can be used to develop lead compounds for treating HFpEF.

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

Voting

1 net vote
13 up votes
12 down votes
Active

Goal 3: Advance Translational Research

Genome Profiling

What structural changes need to be implemented in the health-care community in order to support the use of genomic information in clinical trials and drug development for 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 :

In various blood disorders, including hematologic malignancies, there are both inherited and somatic genetic alterations that contribute to predisposition, transformation, disease progression, responsiveness to therapy, and treatment complications. The presence of such genetic alterations underscore the need for the identification of rare but traceable mutations as well as the integration of such genomic information into clinical trials. By implementing a few structural changes in the healthcare sector, a clinical trial infrastructure can be established that accounts for proper application of sequencing technology. Some examples include the creation of genome diagnostic networks that address accrual of sufficient patients, procurement of suitable tumor/non-tumor material for sequencing, as well as pharmacodynamic and correlative biology studies in hematologic diseases.

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

Voting

11 net votes
22 up votes
11 down votes
Active

Goal 4: Develop Workforce and Resources

Career Development in "Group Based" Science

As the current chair of the Research and Training Division, I would like to convey that the AAAAI membership would like the NHLBI to consider the following in the development of its strategic plan:

 

NHLBI should be challenged on how best to provide career development grants to junior faculty involved in “group based” clinical and bench science.

Submitted by (@wheeze)

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 : Mitchell Grayson on behalf of the American Academy of Allergy, Asthma, and Immunology

Voting

7 net votes
26 up votes
19 down votes
Active

Goal 4: Develop Workforce and Resources

Modernizing Research Training

As the current chair of the Research and Training Division, I would like to convey that the AAAAI membership would like the NHLBI to consider the following in the development of its strategic plan:

 

Since the focus of research has changed over the past decade, training programs need to be encouraged to use newer models of research in their training and mentoring of potential research faculty.

Submitted by (@wheeze)

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 : Mitchell Grayson on behalf of the American Academy of Allergy, Asthma, and Immunology

Voting

10 net votes
23 up votes
13 down votes
Active