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)

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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)

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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)

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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)

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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)

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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)

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