Goal 4: Develop Workforce and Resources

Training in Small Molecule Discovery and Development

The current generation of heart,lung, and blood investigators are not equipped with competitive training needed to approach, design, and test appropriately small molecule therapeutics that may move through the FDA pipeline. Appropriate in silico ligan or structure based design, HTS, design of Pd/Pk models, "go-no-go" branchpoints in drug development, screening approaches, and drug target validation are issues that are ...more »

Submitted by (@mallampallirk)

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 incorporation of workshops, traditional funding mechanisms (T32, F32 etc) earmarked for this type of training will help position and equip NHLBI investigators to more effectively navigate through the landscape of drug discovery and development.

Name of idea submitter and other team members who worked on this idea : Rama Mallampalli, MD

Voting

3 net votes
10 up votes
7 down votes
Active

Goal 3: Advance Translational Research

Cell-specific drug delivery

New paradigms are needed for developing both deliver systems AND drugs to achieve therapeutically effective cell-specific drug delivery.

Submitted by (@klpetrak)

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 proposed development is essentially needed for new effective therapies and personalized medicine.

Feasibility and challenges of addressing this CQ or CC :

The proposed approach is feasible but requires that new paradigms are adopted by organizations and researchers.

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

Voting

19 net votes
22 up votes
3 down votes
Active

Goal 2: Reduce Human Disease

Open up NCATS

The NCATS program for drug repurposing is currently only open to drugs submitted by industry (read, big Pharma). We have had the experience of working to repurpose a generic drug not on their list, and despite great Preliminary data, we could not. This program is a great idea, but needs to be opened to any company and any drug for which solid data backing efficacy and market can be applied. Why do we only want to enhance ...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 :

Opening the program to all FDA approved drugs would benefit development of the most useful repurposed therapeutics. The goal should not be focused upon drugs that large corporations still hold license to, but opened to therapeutic development that helps people, regardless of the size of the corp. Another advantage is that the program could then help small companies, startups, and generic companies develop new uses for drugs proven safe.

Feasibility and challenges of addressing this CQ or CC :

Of course, large corporations can claim that they have the best chances of marketing a repurposed drug, therefore investment should be in areas and drugs they control. The reality is, no matter which entity does the work for repurposing, a successful project will likely be bought and marketed by big Pharma anyway. So the focus should be on repurposing drugs for disease that have few therapeutic options, with exemplary benefit efficacy and low rosk, ect., The focus should not be on a list of drugs submitted by

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

Voting

-5 net votes
5 up votes
10 down votes
Active

Goal 3: Advance Translational Research

Using "omics" technologies to define responders to drug therapies

Metabolomic and proteomic technologies open tremendous avenues to define at the systemic level and, in the case of the lung, the organ level response to drug and non-drug interventions. The concept of responders and non-responders to therapies is poorly defined and hampers development of biomarkers and appropriate animal models. Omics technologies can bridge these important areas. In lung disease, breath analysis could ...more »

Submitted by (@njkenyon)

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

Feasibility and challenges of addressing this CQ or CC :

This work would be slow because it requires analyzing profiles of metabolomes from many drug classes. This long term project is needed to better understand the biological effects of new drugs coming to market.

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

Voting

7 net votes
13 up votes
6 down votes
Active

Goal 2: Reduce Human Disease

What do the newer treatments in R&D tell us about the management of SCD?

What is the molecular mechanism by which new drug therapies propose to reduce the severity and duration of hospitalization and opioid pain medicines? And how does that impact the course of disease progression?

Submitted by (@sicklecellwarrior)

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 : Sickle Cell Warriors, Inc. community members

Voting

11 net votes
20 up votes
9 down votes
Active

Goal 2: Reduce Human Disease

A Program of Research in the Prevention of Chronic Heart Failure

There is a need to improve identification and surveillance of persons at risk for heart failure and pathological ventricular remodeling prior to development of clinically overt heart failure.

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 :

Substantially reduce the age-adjusted incidence and population burden of chronic heart failure.

Feasibility and challenges of addressing this CQ or CC :

The big data and omics revolutions have made it feasible to collect and analyze a variety of data in large numbers of persons within a relatively short time. A very large sample size provides excellent statistical power. Also, the public health and economic magnitude of the problem create the urgency needed to address the critical challenge expeditiously.

Chronic heart failure (HF) is easily the most common and growing cardiovascular cause of hospitalization and impaired functional status and quality of life in the U.S. and much of the world. This is the case despite improved pharmacologic and lifestyle treatment of HF, as well as improved control of blood pressure in the general population. While some HF in the very elderly may reflect the aging process, the epidemiology suggests that most incident cases could be prevented or postponed for years. Also, there are major ethnic and socioeconomic disparities in the incidence of HF.

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

Voting

14 net votes
28 up votes
14 down votes
Active

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

Voting

-7 net votes
11 up votes
18 down votes
Active

Goal 3: Advance Translational Research

Dissemination & Implementation of new treatments and therapies in sickle cell disease

Are current advances in gene editing, new drug therapies and less restrictive BMT criteria being explained and rolled out to the sickle cell community in an effective and timely manner? When can people living with sickle cell disease experience a better quality of life on more permanent based on the treatments we already have?

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 :

Bone marrow transplant criteria has become less restrictive yet there has not been a steep increase in procedures. My understanding is that a sibling or child with the trait can be a donor. At some point this treatment needs to become more widely accessible and discussed with all patients by their doctors.

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

Voting

46 net votes
55 up votes
9 down votes
Active

Goal 2: Reduce Human Disease

Consequences of drug interactions leading to QTc prolongation

Better understand the consequences of drug interactions leading to QTc prolongation. About 1/3 of cardiac ICU patients develop QT prolongation and about 45% receive drugs that are possibly contributing to this problem. The full spectrum of contributors and causes, as well as the patient-centered and health-system-centered clinical outcomes, are not known.

Submitted by (@greg.martin)

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

Details on the impact of addressing this CQ or CC :

Reduction in adverse drug events and preventable deaths

Feasibility and challenges of addressing this CQ or CC :

Combining the power of predictive analytics of high dimensional data streaming continuously from critically ill patients, combined with precision medicine genomics and metabolomics, makes this imminently feasible.

Name of idea submitter and other team members who worked on this idea : Society of Critical Care Medicine Executive Council

Voting

-1 net votes
1 up votes
2 down votes
Active

Goal 2: Reduce Human Disease

Contraindications of daily dose of aspirin

More information on contraindications of daily dose of aspirin, e.g. Can ibuprofen, acetaminophen, or other non-steroidals interfere, and what about larger doses than 81 mgm/day?

Submitted by (@nhlbiforumadministrator1)

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 : Jerry Uhler

Voting

-1 net votes
13 up votes
14 down votes
Active

Goal 2: Reduce Human Disease

A Program of Research in the Prevention of Chronic Heart Failure

There is a need to address chronic heart failure (HF) through improved identification of patients at risk for HF and of patients with pathological ventricular remodeling who have minimal evidence of clinical HF, and more focused and individualized pharmacologic and lifestyle treatments and monitoring of patients with HF risk. Approaches would include big data collection, omics, statistical modeling, and focused clinical ...more »

Submitted by (@nhlbiforumadministrator1)

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

Details on the impact of addressing this CQ or CC :

Substantially reduce the age-adjusted incidence and population burden of chronic heart failure.

Feasibility and challenges of addressing this CQ or CC :

The big data and omics revolutions have made it feasible to collect and analyze a variety of data in large numbers of persons within a relatively short time. A very large sample size provides excellent statistical power. Also, the public health and economic magnitude of the problem create the urgency needed to address the critical challenge expeditiously.

Chronic heart failure (HF) is easily the most common and growing cardiovascular cause of hospitalization and impaired functional status and quality of life in the U.S. and much of the world. This is the case despite improved pharmacologic and lifestyle treatment of HF, as well as improved control of blood pressure in the general population. While some HF in the very elderly may reflect the aging process, the epidemiology suggests that most incident cases could be prevented or postponed for years. Also, there are major ethnic and socioeconomic disparities in the incidence of HF.

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

Voting

17 net votes
28 up votes
11 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