Treatment Resistance and Therapeutic Vulnerabilities in Metastatic Fusion-Positive Rhabdomyosarcoma

The National Pediatric Cancer Foundation (NPCF) aims to fund novel and innovative science and strives to make radical progress against pediatric cancers. Metastatic fusion positive rhabdomyosarcoma (MFPRMS) is an extremely rare sarcoma and presents with widely metastatic disease and an aggressive course. MFPRMS is often highly sensitive to therapy initially, with dramatic and even complete radiographic responses with treatment. However, disease almost inevitably recurs, with 3-year event-free survival rates of around 10%. There has been no recent improvement in survival outcomes for MFPRMS, and no promising new agents ready for testing in up-front clinical trials. The disease is highly challenging to study given its rarity, infrequent tissue acquisition, and predominance in children, a vulnerable population for research initiatives. 

Such dramatic responses followed consistently by recurrence begs the question: How does a cancer so sensitive to early treatment recur so readily; why are a population of cells intrinsically resistant or develop resistance to initial therapy which will help us identify and exploit vulnerabilities of resistant cells to reduce the relapse rate and improve outcome? 

  • Maximum award of $1,000,000 subject to paid over a 5 year period.
  • Proposals should focus on mechanisms and biological insights of therapy resistance in MFPRMS in the time frame between the initial treatment and progressive disease or relapse.
  • The proposal may encompass basic fundamental and developmental biology. Translational science and the use of human or mouse models and human samples of MFPRMS are encouraged. Clinical trials will not be supported.
  • This grant is open only to US licensed/registered/incorporated institutions with lawful permanent residence within the US. Applicants need not be United States citizens.
  • Applicants must have an MD, PhD, or MD/PhD (or equivalent) and be appointed as faculty (or equivalent) by an academic or non-profit research institution or laboratory.
  • The PI must demonstrate the expertise and track record to conduct and complete the proposed study successfully.
  • The Institution must have the infrastructure and resources necessary to support the proposed research.
  • NPCF is seeking innovative solutions that achieve significant advances addressing pediatric cancer treatment & therapies.
  • Multi-disciplinary / joint teams from US based public/private academic, science, healthcare, corporate, and community health/clinical trial research institutions are highly encouraged to apply.
  • The NPCF has overall responsibility for governance of this grant. The recipient institution/organization is a shared responsibility of the awardee’s program director/principal investigator (PD/PI), who leads the scientific aspects of this grant, and the awardee’s authorized organization representative (AOR) and research/project administrator, who leds the administrative aspects of this grant.
  • Teams must designate one individual member as the “Team Leader” for contact and Contest administrative purposes. The Team Leader will be responsible for submitting the Entry on behalf of the Team. In the event an individual on a Team is disqualified, the Team the individual belongs to, and the Entry submitted by the Team will be disqualified.
  • Participants may only submit one Entry as the lead investigator/team leader. Participants are eligible to participate on other/multiple projects as team members.
  • Participants are responsible for any costs or expenses associated with preparing and submitting an Entry.
  • All Entries suspected of violating intellectual property rights, or any local or country law(s) will be ineligible. Participants assume all risk of damaged, lost, late, incomplete, invalid, incorrect, or misdirected Entries.
  • All Entries (and related information) shall be deemed collected and judged in the United States of America. All Entries must be submitted in English.
  • Each Participant agrees that NPCF will have the right, but not the obligation, in its sole discretion to contact any Participant regarding a possible transaction or other business relationship with NPCF to commercially pursue a submitted idea.
  • Each Initial letter of intent along with the other information requested of Participant during the registration process, may be reviewed by NPCF and/or one or more judges selected by NPCF (collectively, the “Review Panel”), to confirm if the submission meets the eligibility criteria for this grant and is deemed a desired research interest for NPCF to support. In NPCF’s discretion, NPCF may engage third-party subject matter experts and judges to serve on the review panel and/or assist with the review of Entries and selection of winner(s).
  • If NPCF leadership (with input from the Review Panel, as appropriate) determines that any Submission does not or may not meet the eligibility criteria for this grant, NPCF may (a) disqualify the Entry, or (b) request that the Participant submit a revised, clarified description of the Entry, for further consideration by NPCF.
  • To enter the final selection process each nominee selected from Letter of Intent review will be notified by email and be required to submit the “Full Grant Application”.
  • Grant winners agree that the information, ideas, and materials provided to NPCF in connection with the selection process and any other information, ideas or other materials otherwise provided to NPCF in connection with this grant, may be publicly disclosed by NPCF or its agents, and used for NPCF’s marketing and public relations activities, in NPCF’s sole discretion.
  • The Selection Panel may select one or more finalists (*). The Selection Panel will make its selections based on the merits/content of the Entries using their best judgement and/or as outlined in these Terms and Conditions.
    • * NPCF reserves the right to modify.
  • Innovation and impact in MFPRMS
  • Scientific and clinical significance, quality, and rigor
  • Investigator qualifications
  • Institutional infrastructure and support
  • Proposals involving collaborators at multiple institutions will be viewed favorably
  • Salary support with fringe is allowed, up to the Executive Level II NIH Salary Cap for PI’s and Co-Investigators
  • Indirect Costs are limited to no more than 15%
  • Lab supplies and small equipment are allowed
  • Funds may not be used for tuition, office/lab furniture, recruiting/relocation expenses, membership dues, trainee support, travel, or research that involves the use of human embryonic or fetal tissue.
  • Length: maximum 1 page for rationale, specific aims, research design/approach, and relevance to RFA and innovation statement. No figures or tables. Minimum ½ margin, font 11 or larger Arial.  A cover page and lay person abstract should also be included with the LOI, but do not count against the 1 page maximum.
  • LOI should include the following sections:
    • Cover Page (PI, affiliation, project title)
    • Lay Person Abstract (brief description of the project, objectives, and significance)
    • Rationale
    • Specific Aims
    • Research Design/Approach
    • Relevance to the RFA and Innovation Statement
  • Additional materials to submit:
    • References (max 1 page)
    • NIH Biosketch of PI

After the LOI deadline, researchers will be invited to submit a full grant proposal. This will include:

  • Cover Page (Title, PI, and institution)
  • Scientific Abstract (max ½ page)
  • Lay Abstract (max ½ page)
  • Background, Significance, Hypothesis, Preliminary Data (if applicable), Specific Aims (½ page)
  • Research Plan (max four pages), including statement on the Innovation and Impact
  • References (2-page maximum)
  • Facilities and Resources
  • Budget and Justification, template will be provided upon invitation of full application
  • Timeline and Milestones (½ page)
  • Human Subjects. Animal Study Approval, Ethical Considerations (if applicable)
  • Letters of Support, Biosketches of Co-investigators/Collaborators, and other supporting documents.

Further details for grant submission will be provided upon invitation of full application. 

Timeline & Submission

  • May 1, 2025

     

    Applications Open for Letter of Intent

  • July 1, 2025

     

    Applications Close / Deadline for Letter of Intent Letters

  • August 1, 2025

     

    Invitation to Submit Full Application

  • November 1, 2025

     

    Full Proposal Due

  • December 15, 2025

     

    Announcement of Winner(s)

Submission Process

  • Letter of Intent Form Submission
    Deadline to Submit: July 1, 2025
    This LOI / ABSTRACT will be used to ensure eligibility and to determine which projects will be selected to submit a full application by the NPCF Initial Review Committee.
  • Projects Reviewed/Selected to Submit Full Application 
    Notified of Selection: August 1, 2025
    Those selected by the Review Committee to submit a full application will be notified and provided more information to prepare a full application. 
  • Full Application Submission
    Deadline to Submit: November 1, 2025
    The projects selected to submit a full application will be reviewed and interviewed by the NPCF Selection Committee. 
  • ANNOUNCE & AWARD WINNERS
    Notified of Selection: December 15, 2025
    The NPCF Selection Committee will select one or more award winners. 

RFA - TP Rhabdomyosarcoma Letter of Intent Form

Please fill out the form. Failure to complete this form in its entirety can result in a delay or incompletion of your submission.

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