NIH P30 CFRTC Pilot and Feasibility Program

The Pilot and Feasibility (P&F) program, directed by Dr. Pradeep Singh, will manage pilot studies funded by the Cystic Fibrosis Research and Translation Center (CFRTC) at the University of Washington and its affiliated institutions.  These include the University of Washington Medical Center, the Fred Hutchinson Cancer Center, Seattle Children’s Hospital/Research Institute, the Benaroya Research Institute, Seattle Biomedical Research Institute, and other affiliate institutions that operate under a sub-contractual relationship with Seattle Children’s and the University of Washington.

The objective of the P&F program is to serve the CFRTC mission of advancing CF research to improve the health of patients with this devastating disease.  The P&F program will contribute to this mission by recruiting young investigators to CF-related research, recruiting established investigators who have not previously worked in CF to CF research, and to enable accomplished CF investigators means to pursue novel and high risk ideas that represent departures from their established work.

Specific Aims:

1. To recruit early stage and established investigators to CF-related research in areas of National Institute of Diabetes, Digestive and Kidney (NIDDK) Diseases emphasis. 

We will engage promising new and established investigators with novel CF research ideas in areas relevant to NIDDK.  This will occur via personal outreach by Core Directors and PIs, interactions with academic and research leaders of the relevant departments and divisions, the monthly CFRTC and CF Research Development Program (RDP) seminar series, monthly CF research meetings at the University of Washington and Seattle Children’s Hospital, the annual CFRTC/RDP retreat, and the CFRTC web-site.  We will strengthen existing interactions the CFRTC, and the UW Diabetes Education and Research Center and the UW Kidney Institute.  Finally we will seek advice from the Internal and External Advisory Boards on strengthening the pilot program, and on identifying emerging areas of CF research and new pilot study investigators.

2. To provide oversight in the management of the P&F studies.

We will strengthen the pilot study review committee to include both prior and new members with a broad scope of expertise related to NIDDK emphasis areas.  We will engage multiple outside expert reviews for each pilot application, and use the NIH scoring system.  Funding decisions will be based on the results of reviews, the project’s relationship to the CFRTC mission, linkage to and collaboration with the Cores, and scientific merit.  Finally we will provide oversight of P&F budgets in partnership with the Administrative Core, review annual progress for each pilot study, and track publications and funding received by pilot investigators.

CF RDP Pilot and Feasibility Program

Funds from the RDP may be used for young investigators or established investigators from diverse basic or clinical science disciplines who may apply their expertise to the problems of CF. Funding of these studies is intended to provide modest support enabling investigators to obtain sufficient preliminary data to successfully compete for longer-term, more substantial funding from agencies such as NIH.

Translational pilot and feasibility projects are projects that seek to: apply basic scientific knowledge to a clinical problem, acquire basic knowledge from clinical specimens, or examine the feasibility of a therapeutic approach in an appropriate animal model or with appropriate clinical specimens.

General examples of pilot and feasibility studies include:

• • Studies proposed by young investigators interested in pursuing a career in CF research. Such investigators may not yet have obtained individual grant support.
  • Studies proposed by established investigators who have experience in an area related to CF and wish to test their abilities to contribute new knowledge on CF. In some cases, this may represent applying techniques, methods, approaches, or theories from other fields to the problems of CF.
  • Studies proposed by established CF investigators who wish to develop and/or test a new approach that is not an extension of their ongoing research.

Pilot and Feasibility projects addressing key questions related to understanding F508del CFTR processing defects or CF airway microbiome, physiology and inflammation remain priorities for CFF. Applicants are also encouraged to consider how pharmacological restoration of CFTR activity impacts the airway milieu including resident pathogens. Other newly emerging research opportunities to be considered include: molecular characterization of CFTR mutations other than F508del, examination of potential approaches to restore CFTR function regardless of mutation, overcoming barriers to gene and oligonucleotide transfer, non-embryonic stem cell therapy, and identification/optimization of tools for personalized medicine.