The Foundation for Prader-Willi Research (FPWR), a nonprofit organization dedicated to supporting research to advance the understanding and treatment of Prader-Willi syndrome (PWS), announces the availability of funds to support innovative PWS research.  FPWR is particularly interested in supporting projects that will lead to new treatments to alleviate the symptoms associated with PWS.  Funding priorities include:

  • Genotype to phenotype: understanding PWS genetics / how loss of PWS-critical region genes leads to the phenotype
  • Neurobiology of hunger / feeding behavior in PWS
  • Neurobiology of cognitive deficits, maladaptive behavior and mental illness in PWS
  • Clinical care research: evaluation of existing drugs and interventions to improve health and quality of life in PWS
  • Therapeutics development for PWS- Genetic therapies; novel pharmaceuticals; drug repurposing for PWS

A full description of FPWR's funding priorities can be found here.  In addition to these funding priorities, FPWR is pleased to announce two special priority areas for the Fall 2018 grant cycle Brain Imaging Studies in Individuals with PWS and Genetic Therapy for PWS.  

Brain Imaging Studies in Individuals with PWS 

Despite a growing number of brain imaging studies suggesting altered functional connectivity in individuals with PWS, the precise patterns of the network abnormalities in PWS are still unknown. Identification of specific connectivity patterns of brain network disturbances characteristic of PWS across development or when PWS symptoms first emerge and manifest are needed to further our understanding of the neurodevelopmental abnormalities in PWS.

Thus, FPWR is interested in proposals that apply cutting-edge functional brain imaging to examine connectivity in individuals with PWS at specific developmental stages or across development. The scope of this RFA is broad, encompassing descriptive studies or focusing on the neurobiological correlates underlying phenotypic abnormalities characteristics of PWS such as hyperphagia and mental health.

Genetic Therapy for PWS

Proposals submitted to this special priority area should focus on establishing early proof-of-concept, and should address one or more of the following questions aimed at determining the feasibility of gene activation or gene replacement therapies for PWS.

  1. Does activation/replacement of PWS genes reverse the PWS phenotype in cellular models and/or animal models of PWS?
  2. Which gene(s) need to be targeted?
  3. What organs and cell types need to be targeted?
  4. What level of expression needs to be achieved to have a therapeutic effect?
  5. Is there a critical window for genetic therapy? At which point during development does gene activation/replacement need to occur to achieve a therapeutic effect? Do different aspects of the phenotype have different critical periods?
  6. What are on target side effects? How is expression of UBE3a affected?
  7. What are off target side effects?
  8. Can delivery of the therapeutic molecule be achieved / what is the best delivery system?

Funds Available:  Applicants may request up to $100,000 USD in direct costs for the initial year of support, with the possibility of a second year of funding upon competitive renewal.  For the Special Priority Areas (Brain Imaging, Genetic Therapy), applicants may request up to $150,000 per year in direct costs for the initial year of support, with the possibility of a second year of funding upon competitive renewal.  Indirect costs of up to 8% are allowed.

Eligibility:  Principal Investigators must have a primary faculty appointment at the level of Instructor or higher.  This grant program will be useful for junior faculty in the early stages of their careers, established investigators in other areas of research who wish to enter the field of PWS research, or investigators in the PWS field who are seeking funding to support pilot studies in a new area of PWS research. FPWR seeks to support innovative, high-risk/high reward research.

Application Review and Award: Letters of intent (LOI) will be reviewed for innovation and potential impact, merit and relevance to the mission of FPWR.  Selected LOIs will be invited to submit a full grant proposal.

Full grant proposals meeting the technical requirements will be reviewed on the basis of merit by scientific reviewers with expertise in the research area.  An advocate reviewer will evaluate the relevance of the proposed research to the PWS community.  Final funding decisions will be based on scientific merit, relevance and the suitability of the research with respect to the programmatic goals of FPWR.

Application Guidelines: All LOIs and applications are to be submitted on-line through ProposalCentral (https://proposalcentral.altum.com/). The LOI is limited to a 2-page description. Full grant proposals are limited to a 5-page research plan. Guidelines, review considerations, a list of currently funded projects, can all be found through the links below.

FPWR-LOI-Instructions

Scientific and advocate review considerations

Previously funded projects

Letter of Intent Due Date:              September 17, 2018, 5pm EST

Full Proposal Due Date:                November 16, 2018, 5pm EST

Please direct questions to: jessica.bohonowych@fpwr.org

FPWR also welcomes highly integrated, multi-disciplinary research teams to address ambitious and challenging research questions that are important for the mission of FPWR and are beyond the scope of one or two investigators. If your project fits into this category, please contact Theresa Strong (theresa.strong@fpwr.org) or Nathalie Kayadjanian (nathalie.kayadjanian@fpwr.org).