Showing 10 ideas for tag "therapies"

Goal 2: Reduce Human Disease

Optimization of Existing Therapies for Sickle Cell Disease

How can the safety, dosing and benefits of existing therapies for sickle cell disease such as hydroxyurea, be optimized in order to increase its efficacy and improve patient adherence?

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

Details on the impact of addressing this CQ or CC

Hydroxyurea is a widely available disease-modifying therapy for sickle cell disease (SCD), but its effectiveness is currently limited by inadequate utilization, and less than optimal response. Research is needed to improve adherence to this evidence-based therapy and emphasis needs to be placed on determining whether therapy with hydroxyurea can prevent or even reverse organ dysfunction. In addition, research identifying new adjunct therapies to blood transfusion and hydroxyurea, as well as disease-specific therapies for co-morbidities such as kidney disease, hypertension, obstructive lung disease, and pulmonary hypertension will be valuable in the management and treatment of SCD.

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

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54 net votes
74 up votes
20 down votes
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Goal 2: Reduce Human Disease

Transfusion strategies in pediatric and neonatal populations

What are the optimal strategies for transfusion of blood products in pediatric and neonatal population?

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

Details on the impact of addressing this CQ or CC

Blood is the most prescribed drug in the PICU, and 95% of all neonates are transfused during their stay in the NICU. Currently, there are no evidence-based guidelines for the optimal hemoglobin levels or platelet counts for these populations. There is a balance that must be achieved between hemostasis and thrombosis for this vulnerable population.

Feasibility and challenges of addressing this CQ or CC

Clinical trials have begun to assess the optimal hemoglobin levels in neonates, but there are no trial to asses the optimal platelet count. Neonatologists, pediatric intensivists, and transfusion medicine physicians are beginning to come together to work on solutions to these problems.

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

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39 net votes
52 up votes
13 down votes
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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?

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.

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46 net votes
55 up votes
9 down votes
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Goal 2: Reduce Human Disease

Alternative treatments in sickle cell disease

There is a growing desire for the development of alternative treatments and natural therapies for the treatment of sickle cell disease.

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

Details on the impact of addressing this CQ or CC

Studies have indicated higher levels of fetal hemoglobin, even moderate levels, as being capable of reducing pain episodes. Development of therapies other than hydroxyurea, may be beneficial to individuals with SCD, specifically natural compounds as opposed to chemical based drugs. Additionally, it may be beneficial to the SCD survivors and the medical community to come up with biomedical alternatives to opiates and heavy narcotics used to induce relief and quiet the discomfort of the patient, even at the risk of addiction, resulting from prolonged usage (a life time).

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

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27 net votes
42 up votes
15 down votes
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Goal 3: Advance Translational Research

Identification of autoantigens that elicit pathogenic immune responses in cardiovascular diseases

Pathogenic immune responses participate in the pathogenesis of many cardiovascular diseases. However, the autoantigens and foreign antigens that elicit the pathogenic immune responses have been poorly identified. Currently, the regulatory mechanisms on immune responses associated with diseases got some attentions. But, without detailed characterizations of this wide spectrum of autoantigens and foreign antigens in patients... more »

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

Feasibility and challenges of addressing this CQ or CC
  1. use peptide/protein based microarray to antibody autoimmune responses associated with each major cardiovascular diseases;
  2. use MHC tetra-mers or similar techniques to characterize T cell autoimmune responses associated with each major cardiovascular diseases;
  3. determine autoantigen repertoire changes in patients' responses to current therapies, especially therapies with immune modulating effects;
  4. develop autoantigens-based immune therapies for clinical trials

Name of idea submitter and other team members who worked on this idea Professor Xiaofeng Yang, MD, PhD, Professor Hong Wang, MD, PhD, Professor Klaus Ley, MD

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26 net votes
28 up votes
2 down votes
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Goal 2: Reduce Human Disease

Evidence-based, optimized transfusion practice

What are optimal transfusion practices for acute coronary syndrome?

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

Details on the impact of addressing this CQ or CC

Blood transfusion is the most commonly employed therapeutic procedure in the US affecting about 5 million patients per year. Having evidence-based transfusion practice guidelines would result in optimized clinical management of patients who need blood transfusions (red blood cells, platelets, plasma) whether for specific conditions (e.g., acute coronary syndromes, sickle cell disease, prenatally) or in specific age groups. Factors that need further evaluation include transfusion trigger in various patient populations, component type and volume transfused, storage age of blood products, patient blood management tools, donor factors that impact on product characteristics, and component processing and storage.

Feasibility and challenges of addressing this CQ or CC

It is increasingly being recognized that much of the transfusion practice has been empirical, without vigorous scientific evaluation, and thus should be evaluated and optimized. For example, it is not clear if patients with acute coronary syndrome would benefit more from a liberal than a more restrictive red blood cell transfusion strategy; and it is not clear whether plasma transfusion benefits patients with slightly elevated INR (international normalized ratios). Additionally, research capabilities both at the molecular level and at the population level have now made such scientific evaluations possible.

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

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19 net votes
32 up votes
13 down votes
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Goal 3: Advance Translational Research

Translating cardiac development/genetics knowledge into therapy

What is needed to translate our knowledge of cardiac development and congenital heart disease genetics into novel diagnostic and/or therapeutic strategies for congenital or acquired heart disease?

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

Details on the impact of addressing this CQ or CC

Develop new therapies for congenital or acquired heart disease.

Feasibility and challenges of addressing this CQ or CC

We are poised to take advantage of the incredible advances in our understanding of cardiac development and genetics which have resulted from the development of high throughput technologies.

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

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10 net votes
21 up votes
11 down votes
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Goal 2: Reduce Human Disease

Engineered ECM for heart repair

Utilizing advances in nano, bio, tissue and related engineering technologies to construct cardiac ECM for heart repair.

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

Details on the impact of addressing this CQ or CC

Will advance cell and gene based therapeutics for cardiac repair. Despite promise, efficacy of cell based therapies remains largely unproven and this may in part be due to poor understanding of cell-ECM interactions. Research efforts in engineering cardiac ECM have the potential to greatly advance such therapeutic approaches.

Feasibility and challenges of addressing this CQ or CC

This research field is ripe for experimentation and testing.
A major thrust of recent efforts in repairing cardiac injury has focused on cell therapies. However, since the ECM provides the necessary scaffold for the cells it is important to consider the cell-ECM interactions when utilizing these approaches.

Will require multi-disciplinary expertise.

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

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3 net votes
19 up votes
16 down votes
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Goal 1: Promote Human Health

Epigenetics and Genomics

There is a need to target epigenetic mechanisms as new treatment options for hematologic disorders.

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

Details on the impact of addressing this CQ or CC

Advances in the field of epigenetics and the understanding of various epigenetic mechanisms has provided a completely new ensemble of therapeutic targets for treating hematologic disorders – both non-malignant and malignant. These mechanisms have enormous implications for understanding the molecular underpinnings of the normal orderly development of hematologic disorders. Although one of the greatest challenges in effectively treating hematologic disorders is the diversity of molecular abnormalities that underlie a disease, there are a number of common threads emerging, including alterations in proteins that function through epigenetic mechanisms. Additional research focusing on epigenetic alterations and emerging targets is needed to identify the role of such proteins in the development of hematologic disorders in order to design potential targeted treatments to counter their effects. This research will further lay the groundwork for precision medicine, and will help to provide more insight on potentially critical determinants of responsiveness to therapeutic regimens.

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

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-2 net votes
13 up votes
15 down votes
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Goal 3: Advance Translational Research

Developing Curative Approaches to Cardiovascular Disease rather than Chronic Magement

Most Pharmacological interventions in cardiovascular diseases tend to address to be used in palliative manner and chronically. There is urgent need for treatments that are curative. This goes to identifying molecular drivers of the diseases and targeting them to correct the underlying pathophysiology rather than symptom suppression.

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

Details on the impact of addressing this CQ or CC

True conquer of cardiovascular diseases, and reversing the chronic nature of these diseases.

Feasibility and challenges of addressing this CQ or CC

Possible but challenging requiring a highly multidisciplinary approach and collaboration.

Name of idea submitter and other team members who worked on this idea John. K. Buolamwini

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1 net vote
2 up votes
1 down votes
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