This project is focused on testing the efficacy of new anti-prion drugs that were designed based on the structure of a potential compound previously characterized by our group (Ferreira et al, 2014; Ferreira et al, 2017). All of these new compounds are able to strongly inhibit PrPRes formation in ScN2a cells infected with the rodent RML prion strain. Using RT-QuIC, we have also been able to test human samples as sources of prions. So far, we have found that most of the newly designed compounds inhibit amyloid fibril formation in RT-QuIC reactions seeded with brain homogenates from patients with sCJD, GSS and FFI. Our goal is to test these new drugs in laboratory grown human brain tissue (cerebral organoids) infected with sCJD and, finally, to test the safety and efficacy of the most promising compounds in prion-infected mouse models.
About the Researcher:
Dr. Ferreira received her PhD in Pharmaceutical Sciences from Universidade Federal do Rio de Janeiro, in Brazil. Currently she works as a postdoctoral fellow at National Institute of Allergy and Infectious Disease, NIH. She has devoted her career to investigate new therapies to treat prion diseases and more recently, she is also interested in developing diagnostic tools to detect neurodegenerative diseases, such as Alzheimer and prion diseases at early stages of disease, where therapeutic intervention is more likely to be successful.
Dr. Ferreira is the recipient of:
- The CJD Foundation Grant, contributed by the Families of the CJD Foundation
- The Dora Middendorf Memorial Grant, contributed by Renee Middendorf and Family
- The Jeffrey A. Smith Memorial Grant, contributed by The Jeffrey and Mary Smith Family Foundation
- The Strides for CJD Grant, contributed by the Families of the CJD Foundation