Key Research Priorities for Applicants

The Cystic Fibrosis Foundation regularly assesses its key research priorities to ensure we are on track to accomplish our mission to cure CF and to provide people with CF the opportunity to lead long lives. Applicants are encouraged to align submissions to these priorities to maximize their potential for being funded.

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In late 2019, the Cystic Fibrosis Foundation released its five-year strategic plan. This plan is broken down into three key parts: Cure, Care, and Community. Most of the Foundation's research priorities center on tackling the first two components of this plan -- developing a cure for CF and optimizing care of the whole person with CF. The research priority areas below elaborate on the Key Areas of Focus outlined in our strategic plan. Each program and funding opportunity may further develop or define these broad areas, so please consult specific requests for applications (RFAs) for more detailed information.

Cure

Key Area of Focus: Treat the underlying cause of CF for all people with the disease and drive progress toward a cure

Research Priority Area: Establish the necessary tools and knowledge to enable researchers to evaluate genetic-based therapies that may have clinical impact for people with CF

  • Identify key targets for therapy that can prevent, halt or reverse CF disease
  • Optimize delivery of genetic therapy
  • Develop tools and models to predict efficacy of treatments

Research Priority Area: Advance therapies that address the underlying cause of CF for individuals with nonsense and rare mutations

  • Identify and characterize mutations other than F508del, including rare mutations
  • Optimize means of overcoming nonsense mutations, including mRNA stability optimization and translational regulation

Research Priority Area: Support the availability of novel cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies and combinations to ensure that people with CF have robust treatment options

  • Improve upon existing approaches to repair and/or replace the mutant CFTR gene
  • Confirm mechanism of modulator action via structure-function studies

Related Links: Path to a Cure, PTAC Stem Cell Consortium

Research Priority Area: Address the fundamental and translatable knowledge gaps in lung epithelial stem cell biology to enable the development of genetic- and cell-based therapies for CF

  • Identify and characterize the target stem/progenitor cell populations in the airways
  • Refine differentiation protocols and characterize and validate iPSCs for cellular engraftment therapy
  • Develop cellular engraftment models and strategies

Grant Programs Addressing These Priorities:

Key Area of Focus: Advance new and improved treatments to address the many complications of CF, including challenges associated with advanced disease

Research Priority Area: Improve outcomes of infections for people with CF

  • Improve current understanding of acquisition, detection, pathogenesis, host-pathogen interactions, diagnosis, treatment approaches, and outcomes for difficult to treat CF infections

Related Topics: Infection Research Initiative, Lung Transplant Initiative/Advanced Lung Disease Program

Research Priority Area: Advance approaches to prevent and treat serious complications of CF outside the lungs

  • Understand and treat CF-related GI complications (including in the liver, the pancreas, and the impact of nutritional deficiencies)
  • Assess the effects of endocrine system dysfunction in CF, including cystic fibrosis-related diabetes (CFRD) and CF bone disease
  • Investigations into other CF complications, including mental health, sinus disease, sexual and reproductive health, etc.

Related Topics: Mental Health, CFRD Survey, Therapeutics Development Network Women's Health Research Working Group

Research Priority Area: Improve understanding of chronic rejection following lung transplant with the goal of developing a prevention or treatment

  • Study the biological mechanisms, predictors, and biomarkers of lung allograft dysfunction/rejection and transplant immunology with the intention of leading to therapeutic targets for prevention and treatment
  • Advance new therapies for lung transplantation through translational studies and clinical trials

Related Topics: Lung Transplant Initiative (Cystic Fibrosis Lung Transplant Consortium, Lung Transplant Consortium, Lung Transplant Patient Registry)

Research Priority Area: Improve understanding of inflammation, impaired airway hydration, and mucus clearance to advance new therapies for CF

  • Mechanisms of defective mucus production, mucociliary clearance (MCC), and airway surface liquid (ASL) that are relevant for therapeutic applications
  • Mechanisms of inflammation and potential targets relevant to CF
  • Assess the influences of CFTR modulation on the airway milieu and other disease manifestations [including resident pathogens, inflammation and inflammatory cell function, mucin structure (tethered and secreted), ASL, and MCC]

Grant Programs Addressing These Priorities:

Care

Key Area of Focus: Support the best possible CF treatment and care delivery by generating high-quality data and evidence

Related Topics: Community-Generated Areas of Encouragement

Research Priority Area: Assess the feasibility and potential of linking novel data sources to the CF Foundation Patient Registry, e.g., administrative databases, patient-reported outcomes, wearables, and home monitoring.

Related Links: Patient Registry (Advanced Lung Disease Registry)

Research Priority Area: Characterize the best CF care and treatment regimens to provide optimal, individualized care as the CF treatment landscape evolves

  • Assess and validate biomarkers, clinical trial tools, outcome measures, and efficacy endpoints
  • Translate laboratory findings of disease pathogenesis/treatment to clinical evaluation
  • Strategies to improve care and quality of life of people with CF

Research Priority Area: Improve understanding of system-level and societal barriers to optimal CF care and explore opportunities to minimize their effects

  • Test interventions that are effective, practical, measurable, scalable, and sustainable
  • Identify innovative ways to support people with CF in managing their disease
  • Identify and characterize racial, ethnic, and/or societal contributors to CF disease expression and management

Related Links: Success with Therapies Research Consortium

Grant Programs Addressing These Priorities:

  • Clinical Research Award (multiple types)
  • Clinical Fellowship Award
  • LeRoy Matthews Physician-Scientist Award
  • CFF/NIH K-Boost Award
  • Harry Shwachman Clinical Investigator Award
  • EnVision CF: Emerging Leaders in CF Endocrinology II Program
  • DIGEST
  • PACE

Key Area of Focus: Evolve and support the CF care model and network

Research Priority Area: Ensure that the CF care model adapts to meet the future needs of people with CF across their lifespan

Research Priority Area: Sustain a culture of continuous learning and improvement in care across the CF community

Related Topics: Quality Improvement Efforts (Cystic Fibrosis Learning Network, Learning and Leadership Collaboratives)

Research Priority Area: Ensure that leading clinicians and researchers across a variety of disciplines continue to focus on CF and are equipped to address the changing health care needs of people with CF

Grant Programs Addressing These Priorities:

  • Clinical Fellowship Award
  • LeRoy Matthews Physician-Scientist Award
  • CFF/NIH K-Boost Award
  • Harry Shwachman Clinical Investigator Award
  • EnVision CF: Emerging Leaders in CF Endocrinology II Program
  • DIGEST
  • PACE
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Infection Research White Paper Addendum Download (PDF)
Research Priorities in the Health of Women With CF for 2021 RFA Download (PDF)
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