Helping families find the critical information and support they need
December 11, 2019
Report from Rare Disease Week in D.C.
March 2, 2017
2019 - The Year in Review
December 31, 2019
2019 Wishes for Elliott - AES Grantee
July 10, 2019
General summary of this project:
Epilepsy, a disease defined by recurrent seizures, is a severe neurological disorder that affects millions of patients. For most, the only option is lifelong treatment with anti-seizure medications, but 1/3 of patients do not respond to drugs. Some patients may be controlled with brain surgery, but this is not always an option.
Two reasons that drug efficacy in epilepsy has remained static are likely (1) the lack of novel experimental platforms in which to model epilepsy and (2) the lack of a methodology for rapid and efficient drug screening. Both of these are concerns that I intend to address in my proposed studies. Historically, rodents have been the most commonly used experimental platform for epilepsy research. However, the rodent brain is profoundly different in complexity, cellular composition, and gene activation patterns to human brain.
In this project I propose using a powerful new human stem cell-based 3D cell culture system called brain organoids to study epilepsy and develop patient-specific therapies. Using patient derived samples I have generated proof-of-principle data that brain organoids exhibit complex and reproducible patterns of physiological activity. I now intend to utilize a specialized device called a multi-electrode array (MEA) to simultaneously assess physiological activity in dozens of organoids following drug exposure. I hope to develop an efficient and tractable methodology for individualized, patient-directed, therapeutics in epilepsy.