The IFOPA Preclinical Drug Testing Program

The goal of the IFOPA Preclinical Drug Testing Program is to accelerate the discovery of novel therapies for FOP. This program is being offered to the FOP research community as a means to lower the costs and barriers of evaluating promising therapeutic agents in in-vivo models of the disease.

By offering this free testing service through a competitive review process, the FOP community will be able to rapidly evaluate therapeutic ideas in in-vivo FOP models without the time or expense of acquiring, expanding and maintaining mice colonies. Centralizing preclinical testing also allows for results to be standardized and compared across compounds, thereby enabling the identification of those agents that have the greatest beneficial in-vivo effect.  

How It Works

The Drug Testing Program is operated through collaboration with investigators at the Mayo Clinic (Drs. Robert Pignolo and Haitao Wang), where all preclinical testing will take place.

The IFOPA will accept preclinical drug testing proposals from researchers in academia and industry, as well as from FOP community members. Applicants will submit a Preclinical Drug Testing proposal, which outlines the scientific rationale for the proposed compound(s). Proposals will be confidentially evaluated. The IFOPA has committed funding to screen four compounds per year.

Proposal Timing

Therapeutic target nominations may be submitted at any time during the year for evaluation by the Preclinical Drug Testing Review Committee.

Eligible Compounds

Nominated therapeutic compounds should be supported by compelling scientific rationale for preclinical testing, and may include:

  • Drugs, compounds or dietary supplement formulations that are currently marketed for other indications (i.e. repurposed drugs)
  • Investigational drugs/new chemical entities
  • Combinations of drugs that have different mechanisms of action and/or different disease targets
  • Analogs of existing drugs which may have an improved efficacy or safety profile
  • Over-the-counter (OTC) medicines that have strong scientific rationale for testing in FOP

All proposed compounds need to be readily available (through purchase or directly supplied) to the Mayo Clinic research team.   


The IFOPA Preclinical Drug Testing Program is open to submissions from:

  • Researchers at academic medical centers or universities worldwide
  • Biotech or pharmaceutical companies with commercial interest in FOP
  • Members of the FOP community (e.g. people with FOP, their family members and friends)

Preclinical Testing

Compounds that have been accepted for testing in the Preclinical Drug Testing Program will be tested in two FOP Mouse Models, a physiological model based on the classic FOP mutation (R206H) and a non-physiological model that produces robust heterotopic ossification (HO). The experimental scheme will consist of a preventative approach to injury-induced HO.


For non-commercial parties, the IFOPA supports all associated costs for testing the drugs, compounds or dietary supplement formulations that are accepted into the Preclinical Drug Testing Program. The program also includes a limited budget to cover the purchase of the nominated drug to be tested. If the drug/compound makes use of materials that are not yet available and/or whose production depends on proprietary or unpublished methods, or is prohibitively expensive, these materials will need to be provided to Mayo Clinic investigators, at the submitter’s expense.   

For commercial entities looking to test their compounds in the IFOPA’s Preclinical Drug Testing Program, it is asked that the submitting company pay for the testing and provide the investigational drug.

Publication and Availability of Results

The submission of proposals and the identity of applicants is sensitive information that is treated by the IFOPA, the Mayo Clinic and members of the Review Committee as confidential. Winning applications will be announced publicly, but other applications will remain confidential.

All results from the IFOPA Preclinical Drug Testing Program will be made available to the public and scientific community as soon as possible, preferably in an open-access, peer-reviewed journal or publicized scientific conference. The IFOPA will catalogue compounds tested and the corresponding results will be posted on (timing of results may be negotiated and your institution/company may be blinded, upon request). Confidentiality and intellectual property concerns with data publishing should be discussed with the IFOPA prior to the application submission. 

Investigators at the Mayo Clinic will serve as co-authors on any publication, and the contributions of all authors will be specified in all publications and presentations. The responsibility for assignment of authorship and the timely publication of findings will fall to both the submitter and to Mayo Clinic investigators. The submission of results for publication should be no more than twelve (12) months subsequent from final data collection. The IFOPA should be recognized for its funding support in any publications, abstracts or presentations resulting from the research. The IFOPA and Mayo Clinic investigators must receive a copy of the published material or paper, including presentations, at the time of publication.

Any published peer-reviewed manuscripts that arise, in whole or in part, from IFOPA funding must be submitted to the National Library of Medicine’s PubMed Central no later than twelve (12) months after the official date of publication.


Proposals for the IFOPA Preclinical Drug Testing Program are accepted throughout the year. Individuals or groups who wish to nominate more than one compound for consideration may do so. However, separate application forms should be submitted for each proposed intervention.

Completed applications should be sent to [email protected].

Questions about the program or the application process should be sent to the IFOPA and Dr. Robert Pignolo at [email protected].

Statement of Understanding

All applicants will be required to accept the following Statement of Understanding at the time of submitting the Preclinical Drug Testing application.

In submitting this proposal, I agree to the following:

  • I understand all information presented in the proposal will be freely shared with members of the IFOPA, Mayo Clinic investigators and the Preclinical Drug Testing Program Review Committee during their evaluation of proposals, but will otherwise be considered confidential.
  • If my proposal, or a modification of it (such as altered dosage or frequency of administration), is accepted for inclusion in a research protocol, I may be asked to help evaluate the data and to prepare the data for written and/or oral publications. I also agree that the Mayo Clinic investigators will serve as co-authors on any publications, and share responsibility for assignment of authorship and timely publication.
  • I understand the IFOPA intends to post the results of all supported studies on its website, irrespective of whether the results are positive or negative (timing may be negotiated and your institution/company may be blinded upon request).
  • I understand data generated by IFOPA-supported experiments using the nominated drug compound may be used in applications for further research support by anyone (unless compounds are proprietary to a company or lab).
  • I understand that I will be free to use IFOPA-generated data in the context of applications for research support or for any other purpose.
  • For applicants that make use of materials that are not yet freely available and whose production depends on proprietary or unpublished methods: If my application is approved for incorporation in the IFOPA Preclinical Drug Testing Program, a mutually acceptable Materials Transfer Agreement will be developed with the parties, which would permit me to provide the Mayo Clinic with the compound(s) needed for the experimentation.


We are grateful to Drs. Robert Pignolo and Haitao Wang, Mayo Clinic, for administering the program on behalf of the FOP community.




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