Joaquín Castilla, PhD

Grant Title: Early vs late intervention: PrP-lowering therapy stage-dependent outcomes in sCJD and GSS mouse models

Location: Center for Cooperative Research in Biosciences (CIC bioGUNE), Basque Research and Technology Alliance (BRTA), Derio, Spain

Grant Year: 2026

New therapies that lower prion protein levels are showing great promise for treating prion diseases, but we face a critical question that affects every patient and family: what happens when treatment starts at different stages of the disease? In reality, many patients won’t be diagnosed at the earliest possible moment—diagnosis can take time, and disease progression varies greatly between conditions like sporadic CJD and GSS. Our study will test a promising prion-lowering therapy at three different time points in two complementary mouse models: before symptoms appear, when symptoms first emerge, and after the disease has progressed. By measuring survival, clinical progression, and blood biomarkers, we aim to understand whether starting treatment later can still extend life, stabilize the disease, or allow for recovery—or whether the timing of treatment fundamentally changes what patients and families can expect. This information is essential for designing future clinical trials, helping doctors counsel patients about realistic outcomes, and guiding families as they make difficult decisions about treatment options. Understanding these treatment windows will ensure that as new therapies become available, we can offer families honest, evidence-based guidance about what to expect based on when the diagnosis is made.

About the Researcher:

Dr. Joaquín Castilla is a senior investigator and director of the Prion Research Laboratory at CIC bioGUNE, where he has served as an IKERBasque Research Professor since 2009. His expertise in prion research spans over two decades, beginning in 1998 at the National Center for Animal Health (CISA-INIA) in Madrid. In 2003, he moved to Switzerland as a Research Scientist at the Serono Research Institute, and subsequently served as Assistant Professor at the University of Texas Medical Branch (2003-2006) and Scripps-Florida (2006-2009), where he led an independent research group.

Dr. Castilla’s research focuses on the in vitro and in vivo propagation of prions, investigating strain and species barrier phenomena to dissect the molecular mechanisms by which prions form and propagate. Notable achievements of his group include the development of highly sensitive methods for prion detection, successful generation of prion infectivity in vitro, and pioneering detection of prions in blood from pre-symptomatic animals. He maintains over 20 active international collaborations with leading research groups in the prion field and has extensive experience with European Community, NIH, and national research projects.

Beyond his scientific work, Dr. Castilla is deeply committed to supporting families affected by prion diseases and currently serves as President of the Spanish Foundation for Prion Diseases (https://fundacionprionicas.org/). He has supervised 20 doctoral theses and published more than 125 peer-reviewed articles in prestigious journals, contributing significantly to our understanding of prion propagation mechanisms and therapeutic approaches.