Showing 4 ideas for tag "therapeutics"

Goal 3: Advance Translational Research

Microvascular Dementia

The catchword Alzheimer's Disease (AD) is used to describe almost all types of dementia these days, and a lot of work has gone into developing preventive and/or therapeutic interventions for AD, but studies show that 40% of "AD" patients may in fact have something else. Microvascular Dementia (MD) presents with a similar phenotype. Delineating these two entities and then developing treatment strategies for MD is my... more »

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

Details on the impact of addressing this CQ or CC

Developing therapeutics for AD that target amyloid and/or tau are not going to work on 40% of the population that have incorrectly been diagnosed with AD when they may instead have MD. The compelling question is how to easily delineate the two followed by the challenge of how to treat the population that has MD.

Feasibility and challenges of addressing this CQ or CC

Accurate diagnostic imaging for true AD (with plaques) is now possible. Therefore, in the presence of cognitive decline, delineating AD versus other entities is now a reasonable, albeit expensive and time consuming, goal. Step one would be to bring costs and time to diagnosis down by developing a less expensive means of testing for true AD versus MD while verifying with the more expensive technologies. The next step would be to determine what contributes to the pathology of MD. For example, is it another type of plaque that is forming (little clots)? Or is the AD plaque injuring the vasculature? Is there genetic predisposition? As far as I know this area is not being explored in depth (other than possibly, the relationship to stroke). Finally, the one could seek to target therapeutics for this particular form of dementia.

Name of idea submitter and other team members who worked on this idea Wendy Mars

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Goal 3: Advance Translational Research

Develop alternatives for patients for whom routine red cell transfusion is unavailable or impractical

There is a compelling need to advance research to understand the physiology governing the safety and efficacy of hemoglobin-based oxygen therapeutics functioning outside the red cell.

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

Details on the impact of addressing this CQ or CC

Adequate numbers of red blood cells are required to sustain human life. Neurocognitive deficits and mortality in acutely anemic humans increase significantly at a hemoglobin level of below 5 g/dL even in the absence of significant cardiovascular disease. At extremely low hemoglobin levels, alternative treatments (supplemental or hyperbaric oxygen, sedation, muscle paralysis and mechanical ventilation) are of only limited benefit and are not without risk. Several classes of patients cannot be routinely transfused with red blood cells. These classes of patients for whom blood is not an option would include patients who will not accept transfusion for religious or personal reasons, patients who due to multiple prior transfusions have developed red cell antibodies without the option for compatible red cells, and massive trauma patients needing treatment in a remote location. The development of cell-free hemoglobin-based oxygen carriers, stable at room temperature and not requiring cross-matching prior to transfusion as a red cell substitute, has been a sought after goal for several decades, yet to date all attempts have met with failure during clinical trials. There is a compelling need to advance research to understand the physiology governing the safety and efficacy of hemoglobin-based oxygen therapeutics functioning outside the red cell.

Feasibility and challenges of addressing this CQ or CC

Multiple physiologic insults and adverse events seen with earlier modified hemoglobins, compared to banked red blood cells, have been described and are now better, but not completely, understood. Advances in hemoglobin modification could allow for successful use in a variety of clinical scenarios with life-saving results. Additional clinical indications could be investigated and established, such as identification of clinical situations where additional oxygen delivery could modulate the effects of chronic ischemic conditions. In addition, the hemoglobin molecule could be modified to deliver additional therapeutic benefit.

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

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5 down votes
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Goal 3: Advance Translational Research

Novel Technologies & Clinical Therapeutics

How can NHLBI harness the power of novel technologies involving nucleic acid delivery and gene editing for clinical therapeutics?

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 Cystic Fibrosis Foundation

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