The 2023 Million Dollar Bike Ride Pilot

Grant Program is now open!

The MDBR Pilot Grant Program provides a one-year grant to support research related to a rare disease represented in the 2023 Million Dollar Bike Ride.

Team NBIA Disorders represented the NBIA Disorders Association at the Tenth Annual Million Dollar Bike Ride for rare disorders. We are proud to announce that with a $30,000 match from the University of Pennsylvania's Orphan Disease Center, we have a total of $120,000 available for two $60,000 research grant awards for Beta-propeller Protein-Associated Neurodegeneration (BPAN).

This Request for Applications (RFA) is open to the international community. All individuals holding a faculty-level appointment at an academic institution or a senior scientific position at a non-profit institution or foundation are eligible to respond to this RFA. 

MDBR 2023 - Team NBIA Disorders

BPAN RFA INFORMATION

Beta-propeller protein-associated neurodegeneration (BPAN)/Neurodegeneration with Brain Iron Accumulation Disorder (NBIA) disorders: Two pilot grants for $60,000 each are available for clinical and translational research studies related to the detection, diagnosis, or treatment of this rare, X-linked disorder caused by mutations in WDR45. BPAN typically is recognized in early childhood with delayed development and seizures. In adulthood, people with BPAN develop rapidly progressive parkinsonism. At the present time, symptoms may be treated but there is no cure.

Grants are expected to generate essential information for the scientific community to advance knowledge about BPAN disease processes and to produce preliminary data to enable national and international funding to carry the work forward.

Examples of priority topic areas include: developing and exploiting disease models including computer models, identifying biomarkers, delineating the molecular cascade that leads to early cellular changes, developing rational therapeutics, establishing outcome measures to be used in clinical trials, and developing other essential resources to substantially prepare the BPAN community for clinical trials. Natural history studies proposals should reflect knowledge of existing, ongoing studies and include a statement indicating how the proposed study would complement or integrate with existing studies. Moreover, proposals for natural history studies must have a component that includes participation in the TIRCON International NBIA Patient Registry & Biobank. This grant is made possible by Team NBIA Disorders and BPAN families with the NBIA Disorders Association.

Letters of Interest Instructions:

Please visit the Orphan Disease Center website to submit your Letter of Interest (LOI), which can also be found here. This one-page LOI is due no later than Friday, September 15, 2023, by 8pm (EDT).

Full Application Instructions and Review Procedure:

NOTE: Full Application is by invitation only after review of Pre-Application

Proposal Due Date: Monday, October 16, 2023, no later than 8pm (EDT)

Grant Review Process:

1) Grants will be reviewed for scientific content and relevance to the goals of the RFA.

2) Full applications proceed through a two-step review process. The first step includes external review and rating with an assessment of the strengths and weaknesses of each application based on the defined review criteria described below. During the second step, funding recommendations are determined based on an assessment of the reviewer scores and written comments. Final decision of funding will be made by Center Leadership.

3) Proposal Content and Review Criteria: The following criteria will be utilized in proposal review.

• Project Proposal - Is the proposed project of high scientific quality? Is the budget fully justified and reasonable in relation to the proposed project?

• Background - Is the fundamental objective of the study and hypothesis to be addressed clearly defined?

• Scientific Approach - Will the proposed specific aims answer the study hypothesis? Will the scientific approach effectively test and answer each specific aim? Are the study goals supported by existing data?

• Clinical Impact - Is the answer to the study hypothesis important to our ability to treat or reduce rare disorders/disease incidence and/or mortality? Will the proposed research lead to substantial advances and/or contribute to large leaps of understanding or knowledge that will contribute to reductions in disease incidence and/or mortality within the decade?

• Research Significance - Does the study address an important question that is not likely to be addressed without this funding? Does the proposed study offer a unique opportunity to explore an important issue and/or employ a novel approach to this disease research? Will the study outcomes advance our knowledge of this disease and/or contribute to changes in the focus of future research questions or the way we conduct research on this issue?

• Investigator Qualifications – Does the investigator hold a track record of outstanding accomplishment, as evidenced by peer-reviewed publications and funding awards? Does the investigator have access to the resources and environment necessary to complete the study as outlined?

Anonymous reviewer feedback is shared upon the request of the applicant at the discretion of the Orphan Disease Center where appropriate.

Confidentiality:

The MDBR Grant Program is a confidential process and all content of the LOIs and Full Applications will be kept confidential by the ODC; our expert reviewers sign a CDA in advance of the review process. In order to encourage sharing of new techniques and findings to advance science, after funding decisions are made, the ODC will share a non-confidential lay summary of the research proposals received (required with your letter of intent), including those that were not funded, with each participating funding organization upon request.

Fund Disbursement:

Funds will be issued through a cost reimbursement mechanism executed by purchase order from the University of Pennsylvania. Details of invoicing schedules and reporting requirements will be made available upon award.

For additional information, please contact Samantha Charleston at scharle@upenn.edu.