A prominent neuroscientist at the University of Southern California (USC), Berislav V. Zlokovic, is on leave following allegations of research misconduct that have cast doubt on his published work and led to the derailment of clinical trials for an experimental stroke treatment. The allegations, submitted by whistleblowers to the National Institutes of Health (NIH), involve potentially doctored images and data in 35 research papers where Zlokovic was the sole common author. These concerns extend to the Phase II clinical trials of 3K3A-APC, a drug developed by ZZ Biotech intended to reduce post-stroke brain bleeds.
Investigation and Institutional Response
Carolyn Meltzer, Dean of USC's Keck School of Medicine, informed faculty that Zlokovic was on leave for an "indefinite period." Steve Kay has been appointed as acting director of both the Zilkha Neurogenetic Institute and the department of physiology and neuroscience, positions previously held by Zlokovic. USC has not provided further details, citing confidentiality.
The NIH has paused the clinical trial and initiated an investigation into Zlokovic's research, instructing USC to return $1.9 million in funding already provided for the study, according to a report in Science. USC has not yet responded to questions regarding the return of federal funds.
Impact on Clinical Trials
ZZ Biotech, the company co-founded by Zlokovic, officially canceled the clinical trial of 3K3A-APC on September 26, according to a notice of withdrawal filed on ClinicalTrials.gov. Kent Pryor, Chief Executive of ZZ Biotech, stated that decisions regarding the future development of 3K3A-APC will be deferred until the investigations into Zlokovic's work are completed. While Zlokovic no longer holds managerial or scientific roles within the company, he remains a minority equity holder.
Scope of Allegations
The whistleblower report questioned the integrity of Zlokovic's research and the safety of 3K3A-APC. Matthew Schrag, an assistant professor of neurology at Vanderbilt and co-author of the whistleblower report, noted that while they did not find evidence of manipulated data in the drug trial itself, the ethical concerns surrounding Zlokovic's earlier work warranted further scrutiny, especially given the life-threatening nature of stroke.
Zlokovic's Research and Retractions
Zlokovic is a prominent researcher known for his work on the blood-brain barrier and its role in stroke and dementia. Since the allegations surfaced, three of his published research papers have been retracted, and eight others have been issued corrections or expressions of concern by journals. These expressions of concern are appended to articles when journals identify potential problems but lack definitive proof of misconduct.
