Goal 3: Advance Translational Research

Drug Hypersensitivity Databases

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: Drug Hypersensitivity is a growing concern for patients who are unprotected against potentially severe and lethal reactions. It would be important to generate databases to characterized the different drug reactions, their ...more »

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

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-7 net votes
11 up votes
18 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

Voting

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

Early COPD

What does early COPD actually look like. This is defined as severe COPD 30 years prior to its manifestation.

Submitted by (@davidmannino)

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

Details on the impact of addressing this CQ or CC :

Prevention programs in COPD either target smoking or those with established disease. Better understanding the factors that lead to the development of COPD (both in ever and never smokers) is critical to improved disease prevention.

Feasibility and challenges of addressing this CQ or CC :

We need to revisit long term studies in novel ways- and look at new cohorts. Better biomarkers need to be developed.

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

Voting

22 net votes
31 up votes
9 down votes
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Goal 2: Reduce Human Disease

Venous Thromboembolism

There is a great need for the development and evaluation of biomarkers for the study of venous thromboembolism (VTE) pathophysiology and risk assessment.

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

Details on the impact of addressing this CQ or CC :

Recent efforts to evaluate biomarkers for VTE occurrence and recurrence have led to the identification of multiple potential candidates, including P-selectin, E-selectin, D-dimer, various microparticles, and various inflammatory cytokines. However, no specific biomarker has yet emerged for routine clinical use for individual VTE risk stratification and personal targeted therapeutics. The development of improved animal models will advance the study of VTE pathophysiology, allowing for more accurate evaluation of emerging biomarkers and initial assessments of potential advanced therapeutic interventions. Also, the identification and prioritization of novel VTE biomarkers will be needed to help improve our understanding of the molecular mechanisms underlying VTE, so as to shepherd the development of novel mechanisms of therapy beyond anticoagulation.

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

13 net votes
26 up votes
13 down votes
Active

Goal 2: Reduce Human Disease

Sleep quality assessments in health

Develop algorithms/chemistries (i.e. biomarkers) differentiating between sleep deficiency and health in point-of-care diagnostic evaluation of health risks.

Submitted by (@nhlbiforumadministrator1)

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 : NHLBI Staff

Voting

37 net votes
56 up votes
19 down votes
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Goal 2: Reduce Human Disease

Disease Severity Biomarkers for Sickle Cell Disease

Can we identify biomarkers that can predict sickle cell disease severity?

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 :

Identifying reliable biomarkers that can predict severity of sickle cell disease could help doctors and patients make better decisions about a wide range of clinical decisions, including weighing the risks vs. benefits of bone marrow transplantation, chemotherapeutic manipulation of Hb F level, and gene therapy, all of which have potentially life-threatening complications.

Feasibility and challenges of addressing this CQ or CC :

Scientific advances make it feasible to identify biomarkers of disease severity. However, identifying biomarkers with good sensitivity and specificity is likely to be a challenge.

Name of idea submitter and other team members who worked on this idea : The Sickle Cell Association of New Jersey

Voting

7 net votes
7 up votes
0 down votes
Active

Goal 2: Reduce Human Disease

Genetic and Molecular Tools for Drug Allergy - Hypersensitivity

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: Given that more patients are treated with newer and better targeted medications including chemotherapy, monoclonal antibodies, small molecules and others that have increased the number of hypersensitivity reactions, which ...more »

Submitted by (@wheeze)

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

Voting

-4 net votes
10 up votes
14 down votes
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Goal 2: Reduce Human Disease

Biomarkers of Pulmonary Hypertension

What are informative and clinically relevant biomarkers of pulmonary hypertension (PH)?

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 :

This research emphasis would help identify novel pulmonary hypertension biomarkers of disease risk and progression that can be used for early detection or as outcome measures in prevention trials or treatment of PH, which is a disease currently still not curable with high mortality rate.

Feasibility and challenges of addressing this CQ or CC :

NHLBI Division of Lung Diseases just launched the multi-center PVDOMICS research program last September that will enroll ~1,500 patients in the next 5 years for deep phenotyping PH. PVDOMICS will provide a perfect foundation and platform for this proposed featured study about informative and clinically relevant biomarkers of PH, and make answering this proposed question more feasible in the next 5-10 years.

Although significant advances in the treatment of pulmonary hypertension have been made in the past two decades, currently pulmonary hypertension remains a devastating disease without many clinically relevant and specific biomarkers available. Novel new informative and clinically relevant pulmonary hypertension biomarkers would greatly help advance the subtype-specific early diagnosis and precision treatment of this disease that could potentially decrease the mortality of PH.

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

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75 net votes
87 up votes
12 down votes
Active

Goal 3: Advance Translational Research

Molecular determinants of vascular wall development and aneurysm formation that can be used as markers for early diagnosis

To increase the potential of translating basic research discoveries into the clinic, there is a need to discover molecular biomarkers that confer risk for aneurysms and vascular dissections. The creation of a nation-wide biorepository of well-defined tissue and plasma samples along with research utilizing these tissue samples employing state-of-the art proteomics, genomics and development of appropriate mouse models will ...more »

Submitted by (@dstrickland)

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 challenge is the coordination of all components of the Project (i.e. development of a national biorepository along with coordination of proteomics and genomics analysis as well as proof of concept in animal models).

Feasibility and challenges of addressing this CQ or CC :

This process is not feasible via the R01 funding mechanisms. The feasibility of addressing this critical challenge is excellent provided adequate resources are provided.

Name of idea submitter and other team members who worked on this idea : Dudley Strickland and Selen Catania Muratoglu

Voting

5 net votes
7 up votes
2 down votes
Active

Goal 2: Reduce Human Disease

Heterogeneity in Asthma Phenotypes

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: Asthma appears to be due to heterogenous etiologies.  To better characterize the various phenotypes and potential etiologies, it would be important to create more epidemiologic and biomarker focused databases, which could ...more »

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

-12 net votes
6 up votes
18 down votes
Active

Goal 3: Advance Translational Research

Tools to facilitate availability and safe use of innovative blood products and their analogs

Novel blood products are being developed based on innovative science (e.g., ex vivo manufactured RBC and platelets, and platelet and plasma derived hemostatic products). However, there is a significant lag in the development of appropriate tools and model systems, which poses a challenge when evaluating such products for regulatory approval.

Submitted by (@chintamani.atreya)

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 rapid advancement in science and technology has identified pathways for ex vivo manufacturing of RBC and platelets; platelet and plasma derived hemostatic products and recombinant coagulation factors, etc. However, these novel products have to be evaluated for their safety, efficacy, purity and potency as part of regulatory approval process. Because the new products are manufactured using innovative technologies some of the existing tools for their evaluation are inadequate. Therefore, future investments in the development of necessary predictive tools (I.e. regulatory science tools) in areas such as the following will positively impact the availability and safe use of innovative blood products and their analogs: 1) the development of analytical and biochemical assays, 2) animal models for product safety and efficacy, 3) product quality-associated biomarkers to characterize storage lesions of RBC and platelets and 4) functional assays and molecular and bioinformatics models that can predict the success of novel blood products both during manufacturing and with respect to clinical patient outcomes.

Feasibility and challenges of addressing this CQ or CC :

This initiative is feasible as investigators in blood organizations, academia and government are already working independently towards this goal in their defined areas of study. However, a coordinated effort among these independent entities could help in the faster development of necessary regulatory science tools to optimize care of individual patients and patient groups.

Name of idea submitter and other team members who worked on this idea : Office of Blood Research and Review, CBER, FDA

Voting

12 net votes
17 up votes
5 down votes
Active

Goal 3: Advance Translational Research

Establish COPD Biomarkers

Specific biomarkers to monitor COPD disease activity are needed.

Submitted by (@jsullivan)

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

Details on the impact of addressing this CQ or CC :

Much is understood about the pathogenesis of COPD at the cellular and biochemical level. There is no established way these insights can be used to test or implement new medicines. An explicit and economical set of procedures need to be established that will facilitate the development of new medicines and guide their clinical use.

Feasibility and challenges of addressing this CQ or CC :

Many very small studies have identified biomarkers that relate to COPD activity in large groups of patients. There is no clear regulatory path for these insights to lead to tests that can be used with confidence in the development and implementation of novel treatments. Realistic regulatory pathways and criteria should be adopted to facilitate the development biomarkers of disease activity in COPD.

Name of idea submitter and other team members who worked on this idea : COPD Foundation, COPD Biomarkers Qualification Consortium

Voting

15 net votes
17 up votes
2 down votes
Active