Novel brain injury treatment tested in 1st human subject
Posted on: Wednesday, June 15th, 2022
An experimental brain injury medication conceived in laboratories at The University of Texas Health Science Center at San Antonio (UT Health San Antonio) was tested Tuesday, June 14, in the first human subject, a clinical trial participant in Hungary. If the drug, called AST-004, performs well in human studies, it will be an urgently needed and novel treatment for stroke and traumatic brain injury (TBI) victims, including those with concussions. Plans are to study it as a chronic therapy for neurodegenerative diseases such as Alzheimer’s, as well.
The first trial is evaluating the safety, tolerability and pharmacokinetic profile of AST-004 in healthy volunteers. Pharmacokinetics is the study of how drugs move within the body. AST-004 currently is an intravenously delivered medication, although an oral form is also being developed.
James D. Lechleiter, PhD, professor of cell systems and anatomy at UT Health San Antonio, made the discoveries that enabled AST-004 development. After a decade of research, he was the inventor of record on the first U.S. patent awarded in 2013 for a class of small molecules that showed neuroprotective effects. Astrocyte Pharmaceuticals, a privately held pharmaceutical company in Groton, Conn., entered an exclusive worldwide license agreement with UT Health San Antonio on the invention in 2015, and the health science center maintains an equity interest in its commercialization. The Office of Technology Commercialization at UT Health San Antonio played a strong assistive role, particularly during the years Dr. Lechleiter was seeking a patent and continuing through the licensing agreement process.
“We started with preclinical experiments and next moved through a series of animal studies, beginning with mice and continuing in larger, more complex models,” Dr. Lechleiter said. “In each species, AST-004 protected the brain effectively and safely. Now, after these successes and approval by regulatory authorities, we have reached trials in humans. The science we delineated, and the results every step of the way, give us a measured confidence that this treatment will in the future be used in everyday life, including with mild concussions.”
Translating laboratory discoveries into practical applications used by people is central to UT Health San Antonio’s role as the largest research university in South Texas.
“We are proud of Dr. Lechleiter’s research contributions that have led to this first-in-man clinical trial,” said Andrea Giuffrida, PhD, vice president for strategic industry ventures at UT Health San Antonio. “A novel treatment for TBI, stroke and neurodegenerative diseases will significantly impact the field of neurology and improve the lives of countless patients.”
The first-in-human study will include up to 52 healthy participants ages 18 to 55, according to a press release by Astrocyte Pharmaceuticals. The study is funded in part by the Medical Technology Enterprise Consortium in cooperation with the U.S. Army Medical Research and Development Command of the U.S. Department of Defense.
“The collaboration with UT Health San Antonio has been sustained for almost eight years, and it has been a fantastic partnership,” said Ted Liston, PhD, vice president of research at Astrocyte Pharmaceuticals. “Our future plans are to continue to advance AST-004 and conduct clinical trials with patients in the U.S. and other countries, and then to seek approval of AST-004 from the U.S. Food and Drug Administration and other world regulatory agencies.”