Abstract
Infantile spasms (IS) is a devastating epilepsy syndrome that typically begins in the first year of life. Symptoms consist of stereotypical spasms, developmental delay, and electroencephalogram (EEG) that may demonstrate Hypsarhythmia. Current therapeutic approaches are not always effective, and there is no reliable way to predict which patient will respond to therapy. Given this disorder's complexity and the potential impact of a disease-modifying approach, Citizens United for Research in Epilepsy (CURE) employed a “team science” approach to advance the understanding of IS pathology and explore therapeutic modalities that might lead to the development of new ways to potentially prevent spasms and Hypsarhythmia. This approach was a first-of-its-kind collaborative initiative in epilepsy. The IS initiative funded 8 investigative teams over the course of 1-3 years. Projects included the following: discovery on the basic biology of IS, discovery of novel therapeutic targets, cross-validation of targets, discovery of biomarkers, and prognosis and treatment of IS. The combined efforts of a strong investigative team led to numerous advances in understanding the neural pathways underlying IS, testing of small molecules in preclinical models of IS and generated preliminary data on potential biomarkers. Thus far, the initiative has resulted in over 19 publications and subsequent funding for several investigators. Investigators reported that the IS initiative generally affected their research positively due to its collaborative and iterative nature. It also provided a unique opportunity to mentor junior investigators with an interest in translational research. Learnings included the need for a dedicated project manager and more transparent and real-time communication with investigators. The CURE IS initiative represents a unique approach to fund scientific discoveries on epilepsy. It brought together an interdisciplinary group of investigators—who otherwise would not have collaborated—to find transformative therapies for IS. Learnings from this initiative are being utilized for subsequent initiatives at CURE.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 49-61 |
| Number of pages | 13 |
| Journal | Epilepsia Open |
| Volume | 6 |
| Issue number | 1 |
| DOIs | |
| State | Published - Mar 2021 |
Bibliographical note
Funding Information:Team science initiatives have gained traction in recent years in an effort to address complex healthcare questions. CURE has broad expertise in funding investigator‐initiated research projects and collaborative, multi‐disciplinary grant programs. CURE took on the challenge to advance the understanding of and discover potential new treatments for IS through this team science approach. The IS initiative brought together a diverse team of experts to rapidly advance IS research and was the first such initiative in the field of epilepsy. Advantages of the IS initiative included an accelerated timeline for discoveries, increased collaboration between research teams, and facilitation of cross‐validation of targets. Learnings from the IS initiative are being used for CURE’s PTE initiative. 50
Funding Information:
The projects described in the paper and this publication are funded by Citizens United for Research in Epilepsy (CURE). We thank Dr Lauren Harte‐Hargrove for her thoughtful review of the manuscript. AT Berg acknowledges funding from NINDS, Pediatric Epilepsy Research Foundation, FamileSCN2A. AS Galanopoulou acknowledges grant support by NINDS RO1 NS091170, U54 NS100064, the US Department of Defense (W81XWH‐18‐1‐0612 and W81XWH‐13‐1‐0180 grants), NICHD U54HD090260, the American Epilepsy society (seed grant), CURE Infantile Spasms initiative, and research funding from the Heffer Family and the Segal Family Foundations and the Abbe Goldstein/Joshua Lurie and Laurie Marsh/Dan Levitz families. Moshé is the Charles Frost Chair in Neurosurgery and Neurology and acknowledges grant support by NIH U54 NS100064 and NS43209, US Department of Defense (W81XWH‐18‐1‐0612 and W81XWH‐13‐1‐0180 grants), CURE Infantile Spasms initiative, the Heffer Family and the Segal Family Foundations, and the Abbe Goldstein/Joshua Lurie and Laurie Marsh/Dan Levitz families. W Mowrey received grant support by NINDS U54 NS100064, the US Department of Defense (W81XWH‐13‐1‐0180), and the CURE Infantile Spasms initiative.
Publisher Copyright:
© 2020 The Authors. Epilepsia Open published by Wiley Periodicals LLC on behalf of International League Against Epilepsy
Keywords
- Citizens United for Research in Epilepsy (CURE)
- Hypsarhythmia
- West syndrome
- infantile spasms
- team science