Goal 3: Advance Translational Research

To facilitate innovation and accelerate research translation, knowledge dissemination, and implementation science that enhances public health.
Showing 17 ideas for tag "therapy"
(@nhlbiforumadministrator)

Goal 3: Advance Translational Research

Expediting Gene and Cell Therapies to the Clinics

What methodologies will best enhance the translation of technologies for gene and cell therapies into potential products for clinical application and commercial development?

In considering a strategy for NHLBI investment in gene therapy, it is important to note that we are only at the beginning of a revolution that will eventually impact biomedical research across a broad range of specialties. NHLBI/NIH needs to create... more »

Voting

66 net votes
97 up votes
31 down votes
Active
(@nhlbiforumadministrator1)

Goal 3: Advance Translational Research

Arrhythmia Therapies Based on Understanding Mechanisms

There is a need to translate these new insights of genetic, molecular, cellular, and tissue arrhythmia mechanisms into the development of novel, safe, and new therapeutic interventions for the treatment and prevention of cardiac arrhythmias.

Voting

51 net votes
86 up votes
35 down votes
Active
(@akuaban)

Goal 3: Advance Translational Research

Genome Editing and Gene Therapy

There is a critical need for the establishment of strategies that will determine the efficacy, safety, and toxicity of genome editing techniques specifically in hematologic diseases.

Voting

69 net votes
87 up votes
18 down votes
Active
(@judith.l.bettencourt)

Goal 3: Advance Translational Research

Translational research supporting stem cell therapy for cardiovascular disease

Translational research supporting stem cell therapy for cardiovascular disease, including: core laboratories for preclinical IND-enabling studies (e.g., PACT), and clinical trials networks for evaluating promising new treatments (e.g., CCTRN).

Voting

115 net votes
149 up votes
34 down votes
Active
(@sophpacz)

Goal 3: Advance Translational Research

Maximizing anti-tumor immunity following allogeneic HCT with biomarkers

Allogeneic hematopoietic cell transplantation (allo-HCT) is one of the most effective forms of tumor immunotherapy available to date. Allo-HCT can be life-saving for patients with aggressive malignancies that cannot be cured through other strategies. The immunotherapeutic efficacy of allo-HCT depends on donor T cell recognition of alloantigens on leukemic cells, which is known as the graft-versus-tumor effect (GVT). No... more »

Voting

32 net votes
52 up votes
20 down votes
Active
(@nhlbiforumadministrator)

Goal 3: Advance Translational Research

Next generation of cellular blood products for clinical use

What advancements in our understanding of hematopoietic stem cell differentiation can be combined with practical developments in novel processing and storage technologies to develop the next generation of transfused blood therapeutics?

There is a dearth of tools to evaluate the efficacy of transfused blood products, although this is the most commonly employed therapeutic procedure in the US. There is also no financial... more »

Voting

-3 net votes
24 up votes
27 down votes
Active
(@chanduvem)

Goal 3: Advance Translational Research

Develop Targeted Therapeutics to Treat Venous Thrombosis and Inflammation in Venous Thromboembolism

Venous Thromboembolism (VTE) afflicts nearly a million Americans yearly, has a mortality of 6-12% and has costs of more than $15 billion. Current treatment regimens, systemic anticoagulation and compression stockings, fail patients in multiple ways: risk of major bleeding episodes; failure of clot resolution in up to 50% of patients; failure to prevent the development of post-thrombotic syndrome (PTS) in up to 40% of... more »

Voting

4 net votes
6 up votes
2 down votes
Active
(@nhlbiforumadministrator)

Goal 3: Advance Translational Research

Translational Research for HIV/AIDS and HLB Health and Diseases

What are the best inroads for the NHLBI to support innovative approaches in the next 5-10 years, especially blood cell therapies based on hematopoietic stem cell and novel gene therapy approaches to control or even cure HIV infection?

Voting

-16 net votes
15 up votes
31 down votes
Active
(@hubel001)

Goal 3: Advance Translational Research

Advancing the preservation of cellular therapies

Cell therapies are produced in specialized facilities and the viability/function of the cells must be retained in order to permit transportation to the site of use, coordination with patient care, etc. Current options for preserving cells are limited. Conventional methods of cryopreservation may result in poor post thaw function and are difficult to use at the point of care. Liquid storage of cells is typically limited... more »

Voting

-12 net votes
11 up votes
23 down votes
Active
(@xuejunparsons)

Goal 3: Advance Translational Research

Exploring Future Cardiovascular Medicine: Heart Precursors Directed from Human Embryonic Stem Cells for Myocardium Regeneration

Cardiovascular disease (CVD) is a major health problem and the leading cause of death in the Western world. Currently, there is no treatment option or compound drug of molecular entity that can change the prognosis of CVD.

Voting

-19 net votes
9 up votes
28 down votes
Active
(@xuejunparsons)

Goal 3: Advance Translational Research

Deriving Cardiac Elements from Pluripotent Human embryonic Stem Cells for Heart Reconstitution

to date, the existing markets lack a clinically-suitable human cardiomyocyte source with adequate myocardium regenerative potential, which has been the major setback in developing safe and effective cell-based therapies for regenerating the damaged human heart in cardiovascular disease.

Voting

-33 net votes
10 up votes
43 down votes
Active
(@xuejunparsons)

Goal 3: Advance Translational Research

Current State of Regenerative Medicine: Moving Stem Cell Research from Animals into Humans for Clinical Trials

Realizing the developmental and therapeutic potential of pluripotent human embryonic stem cell (hESC) derivatives has been hindered by the inefficiency and instability of generating clinically-relevant functional cells from pluripotent cells through conventional uncontrollable and incomplete multi-lineage differentiation.

Voting

-21 net votes
13 up votes
34 down votes
Active
(@xuejunparsons)

Goal 3: Advance Translational Research

Embedding the future of regenerative medicine into the open epigenomic landscape of pluripotent human embryonic stem cells

Large-scale profiling of developmental regulators and histone modifications by genome-wide approaches have provided powerful genome-wide, high-throughput, and high resolution techniques that lead to great advances in our understanding of the global phenomena of human developmental processes. However, without a practical strategy to convert pluripotent cells direct into a specific lineage, previous studies are limited... more »

Voting

-24 net votes
9 up votes
33 down votes
Active
(@xuejunparsons)

Goal 3: Advance Translational Research

The Designation of Human Cardiac Stem Cell therapy Products for Human Trials or First-in-Human Studies

For successful pharmaceutical development of cardiac stem cell therapy, the human cardiac stem cell therapy product must meet certain commercial criteria in plasticity, specificity, and stability before entry into clinical trials.

Voting

-14 net votes
12 up votes
26 down votes
Active