Objective 5: Develop and optimize novel diagnostic and therapeutic strategies to prevent, treat, and cure HLBS diseases

Recent scientific and technological developments offer especially promising opportunities to prevent disease, manage and treat illness, and promote resilience. These wide-ranging developments include, but are not limited to, new gene editing techniques that could safely treat and prevent  heart, lung, blood, and sleep (HLBS) diseases, “smart” tools that could monitor and adjust biological processes, techniques that modulate stem cell or immune system signaling to reduce disease risk, and advances in materials science that could yield vastly improved implant devices. Other technologies and bioengineering developments may further facilitate diagnostic capabilities, and new lifestyle interventions may improve the maintenance of continued health and wellness and facilitate behavioral modification to prevent disease. Many of these advances, which are the results of past investments in basic research, are critical to developing and optimizing novel diagnostics and therapeutics strategies.

Envision a future in which we are able to...
  • Develop surgical grafts (using stem cells, 3-D printing, and nanotechnologies) for children born with heart defects, such that the grafts grow as the child grows into adulthood, thereby avoiding multiple operations.
  • Transform the safety and availability of the nation’s blood supply by using genetically engineered stem cells to eliminate histo-incompatibility antigens and generate universal donor red blood cells that are available off the shelf by a high-throughput process.

Related Priorities

Compelling Question
Would reduction of known cardiac and vascular risk factors during childhood and adolescence translate into the prevention or delayed development of atherosclerosis and other heart diseases? (5.CQ.01)
Compelling Question
Would interventions in pregnancy or early childhood designed to modulate immune development result in primary prevention of asthma? (5.CQ.02)
Compelling Question
How should the management of diseases that typically develop in childhood (including childhood interstitial lung disease, hemoglobinopathies, congenital heart disease, cystic fibrosis, and asthma) be modified as affected individuals mature into adulthood? (5.CQ.03)
Compelling Question
Would using multidisciplinary teams (e.g., nutritionists, exercise physiologists, social workers, psychologists, nurses) be an effective approach to developing, testing, and ultimately applying lifestyle interventions as part of routine patient care in a variety of contexts from community to patient care settings? (5.CQ.04)
Compelling Question
Would circadian-based strategies (e.g., sleep, timing of medication, meals) improve the efficacy of treatments for HLBS diseases (e.g., hypertension, asthma, thrombosis, obesity/diabetes)? (5.CQ.05)

Overarching Objectives

  1. Understand normal biological function and resilience
  2. Investigate newly discovered pathobiological mechanisms important to the onset and progression of HLBS diseases
  3. Investigate factors that account for differences in health among populations
  4. Identify factors that account for individual differences in pathobiology and in responses to treatments
  5. Develop and optimize novel diagnostic and therapeutic strategies to prevent, treat, and cure HLBS diseases
  6. Optimize clinical and implementation research to improve health and reduce disease
  7. Leverage emerging opportunities in data science to open new frontiers in HLBS research
  8. Further develop, diversify, and sustain a scientific workforce capable of accomplishing the NHLBI’s mission