Can modification of either autologous or allogeneic immune cells allow effective treatment of blood diseases and infection with acceptable rates of toxicity?

How can the effectiveness of existing curative therapies be improved for allogeneic hematopoietic stem cell transplantation?

Newer therapies using gene correction, rather than gene addition, are needed for sickle cell disease. Even with this potential advantage, there needs to be a way to safely deliver gene corrected HSC to the sickle cell patient. Chemotherapy is poorly tolerated, and often is the reason patients do not choose the BMT option. What is the status of other less toxic non myeloablative approaches, and how can they best be... more »

Inflammation plays roles in cancer initiation, promotion, and progression. Elevated circulating galectin-3 (Gal-3) protein and other cancer and inflammation promoting factors (CIPFs) such as C-reactive protein and VEGF are associated with tumorigenesis and may play causative roles. Plasma Gal-3 is a biomarker, prognosticator, and pathogenic mediator of diverse cancers and is emerging as a therapeutic target. Preliminary... more »

There is a need to address chronic heart failure (HF) through improved identification of patients at risk for HF and of patients with pathological ventricular remodeling who have minimal evidence of clinical HF, and more focused and individualized pharmacologic and lifestyle treatments and monitoring of patients with HF risk. Approaches would include big data collection, omics, statistical modeling, and focused clinical... more »

There is a need to improve identification and surveillance of persons at risk for heart failure and pathological ventricular remodeling prior to development of clinically overt heart failure.

How can the use of CAR T-cell and checkpoint blockade strategies be optimized in order to cure hematologic diseases?

Major depression (MD) is common in patients with heart failure, and it is an independent risk marker for functional decline, hospitalization, and mortality. Two large trials have shown that it can be difficult to treat. SADHART-CHF, a double-blind, placebo-controlled RCT (n=469), found that sertraline was not efficacious for MD in HF. MOOD-HF (n=372) showed that escitalopram was not efficacious. Smaller trials of cognitive-behavioral... more »

Accelerating the research to find suitable viral vectors and delivery systems to inhale gene therapy deeply into the lungs. Distal therapy is important for several fatal lung diseases. This is urgent and critical research.

Multiple lines of evidence suggest that the beneficial effects of cell therapy are mostly indirect, but what are the key factors responsible for these indirect benefits?

What are the biological properties and key surface markers of human lung progenitor cells and lung epithelial differentiated iPSCs? How can these cell populations be targeted for therapeutic purposes, including regenerative therapy?

Identifying breast cancer survivors at high risk for CV morbidity and mortality to allow targeting of management strategies to reduce CV events and thereby improve overall cancer-related survival.

What options for therapy for severe asthma are available for the non-eosinophilic phenotype?

Which therapy or combination of therapies to reduce injury from acute myocardial infarction (AMI) will be most efficacious for future clinical trials?

There is a need to examine the use of recombinant DNA to the heart for correction of genetic abnormalities or restoration of normal signaling pathways to prevent heart failure. However, gene therapy is a complex process and more studies are needed in which tissue targeting, route of delivery, regulation of target gene expression, therapeutic dose, and identification of robust biomarkers are further investigated.