Established by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) in 1999, the Interdisciplinary Centers for PKD Research (ICPKD) are a partnership of scientific investigators from various disciplines who use complementary and integrated approaches in PKD research. The NIDDK now funds two types of PKD Centers: Interdisciplinary Centers (P50) and PKD Research and Translation Core Centers (P30).
Research Goals and Scope for Centers for PKD Research (P50)
Proposed studies should foster and extend the development of new approaches into the causes, early diagnoses, and improved treatments for PKD. Therefore the following research goals are provided as potential areas of investigation that may be considered in developing innovative studies in an ICPKD. These are provided as examples and should not be viewed as restrictive or all inclusive:
o Genetic Mechanisms:
Define primary and secondary mutagenic mechanisms of PKD1 & PKD2;
Localize and clone genes for other forms;
Identify genetic loci or genes that modify the PKD disease process;
Develop genomic reagents useful to these goals.
o Biology of Polycystin function
Define cellular and biochemical functions of PKD1-PKD2 and their downstream mediators.
Identify genes responsible for renal cystic development and the progression of renal dysfunction in animal models.
o Physiological Studies
Develop physiological measurements in murine models of PKD;
o Pathogenesis and Progression
Define the basis for increased epithelial proliferation;
Define the role of inflammation in disease progression;
Define the role of the renal microvasculature in disease progression;
Develop novel markers or end points to monitor the progression of renal dysfunction in PKD;
Develop imaging methods and other means to monitor renal dysfunction before the decline in GFR;
Further elucidate control of fluid secretion and accumulation in cysts.
o Innovative therapeutic strategies for ameliorating the initiation of cyst formation and course of progression of renal dysfunction in humans and in animal models
Explore treatments based on new pathogenic insights;
Explore innovative therapies to ameliorate the course of disease using pharmacologic and gene therapeutic approaches in animal models;
Explore innovative therapies to ameliorate the course of disease using pharmacologic therapeutic approaches in patients;
Identify and initiate innovative treatments in specific cohorts of patients with unusual clinical characteristics (including extra-renal manifestations of disease) or rates of progression of renal disease.
Description of a Center
An ICPKD must be an identifiable organizational unit within a single university medical center or within a consortium of cooperating institutions with a university affiliation. The overall goal of an ICPKD is to bring together, in a cooperative, multidisciplinary and integrative manner, basic science and clinical investigators to enrich the effectiveness of research on PKD.
The ICPKD grant consists of a cluster of individual, but interrelated, basic and / or clinical research projects, each with high scientific merit and clear research objectives. In the aggregate, the projects should be directed to the development of fundamental knowledge leading to understanding of the disease processes and the design of curative or preventive strategies.
Core facilities in an ICPKD are shared resources that enhance productivity or in other ways benefit a group of investigators working to accomplish the stated goals of the ICPKD. Cores should be designed to furnish a group of investigators with some technique, service, determination, or instrumentation in a manner that will enhance the research in progress, consolidate manpower effort, and contribute to cost-effectiveness by providing a service at lower cost and possibly higher quality than if each investigator were to attempt the same activity individually.
Research Goals and Scope of Rsearch and Translation Cores (P30)
The goal of these PKD Research and Translation Core Centers is to provide resources for communication and collaboration between basic and clinical researchers in the field of polycystic kidney disease. Core Centers will provide shared resources to enhance the efficiency of research and foster collaborations within and among institutions with strong existing bases of research on PKD. Centers may be located in a single institution or in multiple institutions with complementary research bases.
A biomedical research core is defined as a shared resource that provides essential services, techniques, or instrumentation to Center participants enabling them to conduct their funded individual research projects more efficiently and/or more effectively. Cores provide specialized technologies and expertise needed to accomplish the stated goals of the Center. Each core should provide services to multiple funded research projects. Centers may propose either Institutional Cores or Regional/National/International Cores. Whereas Institutional Cores support research at a single institution or a set of cooperating institutions, Regional/National/International Shared Resources serve specific scientific communities on a regional, national, or international level. A new category of research base for cores that are used as a regional, national, or international resource should be considered the "extended research base". The extended research base for a regional, national or international core could include all investigators who might expect to use the core in some way. This might include investigators who would be expected to fully compensate the core service through a charge-back, and thus would not be obtaining direct financial assistance from the Center. The list could include investigators who use the core services but otherwise have no collaborative interactions with other Center investigators. The extended research base should be defined as an entity separate from the institutional research base. For review purposes, it should be evaluated as part of the core, in order to distinguish it from the local institutional research base. Examples of types of biomedical core resources that would be considered responsive to this Request for Applications include:
o Collection, analysis, storage and distribution of data and samples;
o Provision of specialized tools and technologies or access to specialized
o Development, standardization and distribution of reagents and/or protocols;
o Provision of technical assistance, training, and enrichment programs;
o Recruitment of patients and coordination of patient studies;
o Development, beta-testing and dissemination of specialty assays, methods,
and services on an institutional level;
o Increase interdisciplinary interactions at the institution through cross-
o Sharing of specialized tools, technologies and expertise between
In addition to biomedical cores, an administrative core must be described which will be responsible for allocation of resources within the Center and distribution of resources to Center participants. The Administrative core will also be responsible for planning the Educational Enrichment Program consisting of a seminar series, guest lectures, and workshops, and convening a Committee to oversee the solicitation, review and selection of the pilot projects. Although funds are not provided directly for training purposes, the core laboratories and program enrichment activities should provide training opportunities for Center members.
Each Core Center must develop a cohesive Pilot and Feasibility Program to develop new research directions or provide an opportunity for new investigators or established investigators to enter the field of PKD research. A pilot and feasibility project is intended to provide modest support that will allow an investigator the opportunity to develop sufficient preliminary data as a basis for an application for independent research support. Pilot and feasibility projects are not intended to support or supplement ongoing research of an established investigator. This Program should be integrated into the overall research goals of the Center and make use of the resources provided by the cores. Pilot and Feasibility projects could include clinical projects to investigate basic research findings in a clinical setting.
NIDDK/KUH Project Officer: Marva Moxey-Mims, M.D., 301-574-7717.