Foundation Develops New Strategic Plan to Guide Future Research

The Foundation Fighting Blindness Strategic Planning Report: A Summary

The mission of the Foundation Fighting Blindness (FFB) is to drive the research that will provide preventions, treatments and cures for people affected by retinitis pigmentosa (RP), macular degeneration, Usher syndrome, and the entire spectrum of retinal degenerative diseases.

In keeping with this mission, the FFB has a continuing need to ensure support for a focused and clinically relevant research program. To accomplish this it has evolved a regular, strategic planning process designed to develop recommendations regarding long-term goals, organizational niches and capabilities. This process is embodied in science strategy planning sessions held every four to five years, with the most recent strategic planning meeting occurring in October 2008.

The goals of the 2008 effort were to identify the research priority areas for Foundation support for the next five years. Specific issues addressed as part of this strategic planning effort included:

  • planning how FFB and its clinical trial support organization, the Foundation Fighting Blindness Clinical Research Institute (FFB CRI), will move forward over the next five years to fund the research that is essential to sustain the developmental pipeline for treatment and prevention approaches and products, while maintaining support for basic science discovery;
  • assisting FFB and FFB CRI to align funding priority areas to maximize the potential for Foundation support to make a tangible difference in finding treatments, cures, and prevention strategies for inherited retinal degenerative diseases;
  • determining how FFB and FFB CRI can work most effectively with other interested organizations and individuals, both in the United States and worldwide, to move rapidly and efficiently toward prevention and treatment of inherited retinal degenerative diseases; and,
  • identifying ways in which FFB- and FFB CRI-supported scientists can share data most efficiently and expeditiously to explore promising therapeutic and preventive leads while not wasting valuable resources on scientifically unsound pursuits.

 

A major outcome from the strategic planning session was a realignment of existing FFB Research Priority Areas, both with respect to focus, scope, and actual number. The six new Research Priority Areas are:

 

  • Gene Therapy
  • Regenerative Medicine
  • Novel Medical Therapies
  • Clinical: Structural and Functional Relationships
  • Genetics
  • Cellular and Molecular Mechanisms of Disease

An action plan for identifying and implementing key outcomes of the 2008 strategic planning session was developed. For each research priority area, an overall objective, along with research priorities and recommendations were defined. They are summarized below.

Gene Therapy

 

  • Overall Objective: To make gene therapy available for widespread clinical use in treating retinal degenerative disorders, via research leading to clinical trials, enhanced vector design and optimization of gene delivery.
  • Recommendations and Research Priorities:
    • Support Research Directed Toward Clinical Trials
    • Support Disease-Specific Gene Therapy and Develop Alternative (Non-gene-specific) Approaches
    • Identify the Next Disease Targets for Gene Therapy
    • Identify New Virus Vectors for Gene Delivery
    • Develop Intravitreal Delivery Mechanisms
    • Develop Vectors Capable of Regulating Gene Expression

Regenerative Medicine

 

  • Overall Objective: To rescue and, ultimately, replace dead retinal cells with cells capable of functional replacement (e.g., stem cells or retinal precursor cells), sufficient to restore lost vision.
  • Recommendations and Research Priorities:
    • Engage in Targeted Funding of Research on Mechanisms of Cellular Differentiation
    • Engage in Targeted Funding of Cell Transplantation Research
    • Foster Identification of “Risk” Populations Early in (or Before) Disease
    • Promote Development of Predictive Biomarkers (Disease-specific and Disease Stage-specific) For Risk
    • Promote Standardization of Retina-Targeted Stem Cell Protocols

Novel Medical Therapies

 

  • Overall Objective: To support research directed toward developing drugs to retain retinal function and structure in retinal degenerative diseases, including the creation of improved animal models of human disease, better functional testing of drug effectiveness, and novel drug delivery systems.
  • Recommendations and Research Priorities:
    • Develop Drugs to Preserve Retinal Function and Structure
    • Focus on “Available” Drugs
    • Classify Existing Animal Models and Develop Improved Models
    • Improve Functional Testing of Drug Effectiveness
    • Evaluate Novel Drug Delivery Systems
    • Promote Inter-Disciplinary Technology and Cross-Fertilization

Clinical: Structural and Functional Relationships

 

  • Overall Objective: Develop improved technology and standardized processes to establish relationships between clinical retina function and retina structure in retinal degenerative diseases and to enable early disease detection, utilizing FFB-funded centers to expand patient populations and expedite clinical trials.
  • Recommendations and Research Priorities:
    • Fund Targeted Research on Clinical Characterization of Retinal Function and Structure Across Disease (Phenotyping)
    • Adapt a Standardized Approach for Coordinating the Correlation of Clinical Phenotyping Across FFB/FFB CRI Centers
    • Create a Purpose-Driven Electronic Database of Clinical Phenotype Information
    • Create Partnerships to Fast-Track Retinal Imaging Technology

Genetics

 

  • Overall Objectives: To improve abilities to identify disease-causing mutations in inherited retinal disorders, in part by integrating comprehensive genetic testing into routine clinical care. To identify inherited risk factors for age-related macular degeneration (AMD) and the relative contributions of associated genetic and non-genetic factors (e.g., lifestyle), sufficient to use such knowledge in developing treatments and preventions.
  • Recommendations and Research Priorities:
    • Identify Disease-Causing Genes and Mutations
    • Develop Routine Genetic Testing Services and Methods
    • Identify Modifying Factors for Inherited Retinal Diseases
    • Identify Additional Risk Factors for AMD
    • Investigate Interactions between Genes and the Environment

Cell and Molecular Mechanisms of Disease

 

 

  • Overall Objective: To improve our understanding of the nature and cause of disease in inherited retinal degenerations so that improved therapies for the prevention of vision loss can be developed.
  • Recommendations And Research Priorities:
    • Support Basic Research Directed Toward Fundamental Understanding Of Disease Causes
    • Develop Additional RDD Animal Models as Shared Resources
    • Create Funding Mechanisms to Support/Encourage "High Risk" Projects

 

In addition to the research priority areas, a number of “cross-cutting” research concepts that could contribute to each new FFB-supported research priority area were also indentified during the strategic planning discussions. Several other issues with broad implications for improved management of grants and research funds were also identified and addressed, including:

 

  • Data and Resource Sharing: Develop ideas to enhance the current sharing practices and create new methods to implement them;
  • Industry Relationships: Create initiatives to foster industrial relationships as a means to leverage FFB funding;
  • Inter-Disciplinary Approaches: Identify research technologies that will span several scientific fields and apply to multiple research areas, to leverage funds;
  • Mechanism(s) for Funding Inter-Disciplinary, Goal-Driven Research Initiatives: Retain the existing grant mechanism (an “open” call) and add select Request for Applications (RFAs) for targeted initiatives (e.g., drug delivery, nanotechnology, and imaging).
  • FFB Career Training Programs: Revise the current program mechanism to provide more uniform, enhanced support for clinician-scientists earlier in their careers.