Request for Research Proposals:

A FOUNDATION BUILDING STRENGTH FOR

NEMALINE MYOPATHY

MISSION

Our Mission is to find treatments for Nemaline Myopathy (NM).

A Foundation Building Strength (AFBS) is a non-profit organization striving to stimulate and advance the science of treating NM, ultimately laying the groundwork for the development of commercially available treatments for these diseases.

“Building Strength 2017”

AFBS is excited to announce a new initiative to fund up to $1 million in research grants, advancing our mission to find treatments for NM. AFBS has and will continue to fund research advancing our understanding of the pathogenesis of NM. We have started to fund studies looking at some promising treatment approaches. For this round of funding, we are particularly interested in support investigators in these three  areas:

  1.   Finding Treatments for NM: Remember, AFBS will also consider any proposal that gets us closer to our goal! We are interested in drugs that change the way skeletal muscle works, cell therapies that could repair or replace damaged skeletal muscle, and gene therapies that could improve the function of existing cells. We are interested in improving the way that registries work and in making patient tissue available to researchers with great theories to test. We are interested in exploring the efficacy of exercise regimens or devices in improving patient outcomes. We want to fund good science that is aligned with our mission.
  1.   Improving Pulmonary Function: Any project that studies the improvement of pulmonary function in NM patients will be considered. This could include the design and conduct of clinical trials whose aim is to investigate methods to improve pulmonary muscle strength and to optimize clearance of secretions to ultimately improve quality of life for those living with NM.  Endpoints might include impact of exercise, optimization of support for related sleep disorders, or development of methods to more effectively measure pulmonary function. Modalities could include therapeutics, exercise, or devices. Development of validated endpoints of pulmonary function in NM patients would also be considered in this area.  
  1.   Exploring Supplements and Other Alternative Treatments: Periodically we hear reports of supplements, vitamins, or particular foods that seem to help an individual patient. We would like to fund rigorous research to prove or disprove the efficacy and safety of supplements such as these. This could include clinical trials or preclinical studies evaluating the possible mechanism of action of such supplements.

As in the past, AFBS encourages investigators to apply for this research grant as a source of early “seed money” to launch larger studies that will ultimately require further funding.

To reiterate, research proposals need not be limited to these areas. AFBS will seriously consider all proposals supporting our mission.

ELIGIBILITY CRITERIA

To be eligible to apply for the Nemaline Myopathy Research Grant, an applicant must:

  •         Be qualified to conduct a program of original research (generally this will mean the Principal Investigator holds a Doctor of Medicine (M.D.), Doctor of Philosophy (Ph.D.) or equivalent degree (e.g. D.O.) and has their own laboratory)
  •         Assume both administrative and financial responsibility for the grant
  •         Have access to institutional resources necessary to conduct the proposed research properly
  •         There are no geographic restrictions.

REQUIREMENTS FOR GRANT APPLICATION

Lay Abstract

Please provide a statement of no more than 300 words explaining the subject of the research proposal, relationship to NM, and its goals. This should be written using relatively simple lay terminology so that an individual without scientific training will understand the statement.

Scientific Abstract

Please provide a statement of no more than 300 words explaining the subject of the research proposal, its relationship to NM and its goals. This statement will inform the scientific community of the nature of this work.

Research Plan    (No longer than 5 pages of text, plus references)

  1. Hypothesis and / or Specific Aims:  (maximum of one page)

State clearly and concisely the intent of the proposed research and /or the hypothesis to be tested and developed.

  1. Background and Significance:  (maximum of one page)

Briefly describe the background of the present proposal. Explain the potential significance and relevance of the proposed work to advancing knowledge of NM.   Please include a clear description of the scientific background and a demonstration of techniques needed to make the proposed project a success.  

  1. Preliminary Studies:  (maximum of one page)

Cite prior work in your laboratory and published papers directly related to the proposed project.  Include descriptions of established techniques and collaboration with other scientists.

  1. Research Design and Methods:   (maximum of two pages)

Provide a detailed discussion of the experimental design and methods to be used to accomplish the specific aims.   Please include a detailed timeline for experiments included in the project.

  1. Success Criteria: (maximum of two pages)

Provide details of methods used to evaluate the data and prospectively identify criteria to determine success. For quantitative measures this could include description of anticipated statistical analysis.

Team

Provide a detailed description of the team and the roles each team member would play. Include short 1-2 page biographies of each key investigator (using the NIH or similar format).

Budget

Please include a detailed budget and justification that addresses personnel, equipment, supplies, travel, patient care, and any other relevant costs.  As a non-profit, our policy is to not pay indirect cost rates for institutional overhead. We will pay some indirect costs if absolutely required (to be negotiated on a case-by-case basis), but priority will be given to low overhead proposals.

Terms of Award Period

Projects can span more than one year as required by the nature of the research. In general, funding will be tied to milestones.  Funding recommendations will be made by the AFBS Scientific Advisory Board and funding decisions will be made by the AFBS Board of Directors.

Amount of Award

“Building Strength 2017” will total up to $1 million. Multiple grants will be made, and the requested funding should match the proposals. Most proposals are expected to be in the $30,000 – $200,000 range.   

Other Policies and Guidelines

Written progress reports will be required on a quarterly basis.  We would like our investigators to publish their results. Regulatory approvals for proposed clinical investigations or animal studies do not have to be in place at the time an application is submitted.  However, funding will not commence until all necessary regulatory approvals have been obtained.

Deadline

Final applications will be due on February 1, 2017.

Applications should be in PDF format and sent to “rfp@buildingstrength.org”.

All applications should include the full contact information for the principal investigator.