Million Dollar Bike Ride Pilot Grant

2024 Grant Cycle 

The KCNT1 Epilepsy Foundation, along with the Orphan Disease Center (ODC) at the University of Pennsylvania are pleased to announce the 2024 Million Dollar Bike Ride Pilot Grant Program. This year, the program provides a one‐year award of $68,667 to support KCNT1 research.

The 2024 grant cycle is now open for submission. One-page LOIs are due no later than Friday, September 20, 2024 by 8pm (EST).

KCNT1 iPSC cell lines, blood, tissue, and mice are available. Please contact ali@kcnt1epilepsy.org for an updated list.

Eligibility


This RFA is open globally. International applicants are invited to apply. 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. Prior MDBR award recipients must have current and updated project reporting to be eligible for selection.

Letter of Interest Instructions:

Please visit the ODC website to submit your Letter of Interest (LOI). This one-page LOI is due no later than Friday, September 20, 2024 by 8pm (EST). Please see below for our areas of interest.

Full Application is by invitation only after review of Pre-Application, and will be due Monday, October 21, 2024 no later than 8pm (EST), uploaded on the ODC's website.

Award winners will be selected through a peer-review process designed to support research towards understanding KCNT1-related epilepsy and finding potential treatments.

Our 2023 co-funded MDBR grant was awarded to Dr. Rajvinder Karda at University College London to study novel Adeno-associated viral vector (AAV) mediated RNA editing treatment for KCNT1 epilepsy. Dr. Karda’s proposal aims to develop a novel RNA editing therapy treatment for EIMFS, altering the mutated KCNT1 protein code so less mutant protein is made. They will deliver the RNA treatment within a virus called adeno-associated virus (AAV), seeking a reduction in the amount of mutant KCNT1 protein in neurons and normalization of channel activity. They will test the treatment in cells donated by patients with KCNT1 epilepsy.

Areas of Interest

The KCNT1 Epilepsy Foundation is seeking proposals for a $68,667 award for a translational research project focused on the development of therapies for KCNT1 disorders. The goal of this grant is to support research that can lead to the development of effective treatments for KCNT1 disorders. Examples of research priorities include and are not limited to:

  •  Research into understanding KCNT1 function that helps inform the development of targeted, novel therapies. This can include cellular mechanisms, splice variants, and gene modifiers that potentially influence KCNT1 and could serve as a potential therapeutic target. Proposals should emphasize therapeutic potential.

  •  Validation of assessment tools in KCNT1 patients for use in clinical trial outcome measures, especially non-seizure outcomes. The proposal should include details of the assessment tools, the validation process, and the expected outcomes.

  •  Investigation of symptoms/pathophysiology outside of the brain, especially the role of KCNT1 in the heart. Proposals may include clinical studies or translational lab research.

  •  Stratification of KCNT1 variants by genotype, phenotype, and severity, including the analysis of variants of uncertain significance. This study should elucidate correlations that can inform diagnosis and treatment strategies.

  •  Development of gene therapies: the proposal should include details of the gene therapy approach, preclinical data, and the proposed plan for clinical translation.

  •  Investigation into the relationship between fever-range hyperthermia and KCNT1-related epilepsy:KCNT1 patient caregivers report that KCNT1 patients experience fewer seizures during illnesses that include fever, and following recovery, seizures are often worse. Proposals should include a plan to identify thermometric contributors to seizure frequency and intensity as a means towards development of a therapeutic.

  • Research that explores the causal relationship between KCNT1, seizures and traumatic brain injury (TBI).

    Applicants are encouraged to collaborate with existing KCNT1 researchers and to leverage existing disease models and data (e.g., animal models, Citizen Health databank, registry data, biobank, cell lines, etc.) and should include a statement on resource sharing in their proposal. This grant is made possible by the KCNT1 Epilepsy Foundation, its generous supporters, and the Penn Orphan Disease Center.