The technology leverages bio-manufactured exosomes.
There has been a lot of attention paid in recent years to traumatic brain injury (TBI), particularly in light of the findings of chronic traumatic encephalopathy (CTE), a degenerative brain disorder linked to repetitive head trauma, in NFL football players.
New research coming out of the University of Georgia may lead to improved treatments. It was published in the Journal of Neurotrauma.
“Mechanistically, TBI is a physician’s nightmare,” said Lohitash Karumbaiah, associate professor of regenerative medicine in UGA’s College of Agricultural and Environmental Sciences and one of the study’s lead authors. “Because there are so many things going on in the brain, you can’t really exactly pinpoint what is going wrong, and without therapies to immediately improve recovery, the situation becomes extremely complex.”
The technology leverages bio-manufactured exosomes. Exosomes are extracellular vesicles (EVs) bound to cell membranes and implicated in cell-to-cell communication and the transmission of disease states—they are sometimes referred to as “cargo” molecules because of the way that neural exosomes carry messages to the brain.
They have natural abilities to cross the blood-brain barrier, and to enhance the body’s anti-inflammatory, self-repair and protective mechanisms.
The researchers’ bio-manufactured exosomes can be stored and dosed as an injection. Once dosed, the exosomes act as message mediators that can reset, regenerate and coordinate communication with close-by and distant cells. In rats with TBI, it has showed improved functional recovery.
“The technology takes full benefit of the desirable properties of a neural stem cell therapy without introducing cells into patients,” said Steven Stice, Georgia Research Alliance Eminent Scholar and DW Brooks Distinguished Professor in the College of Agricultural and Environmental Sciences. “We are working toward a therapeutic that has a multifunctional promise to repair brain injury and be reproducible in a cost-effective, off-the-shelf drug format.”
The treatment could theoretically be administered at the time of injury via intravenous fluids. It is designed to accelerate neuronal regeneration and support cells after injury. There is also some speculation the technology could be used as a preventive management program for all levels of TBI, including mild and moderate concussions.
“Administrating exosomes into a patient’s IV drip would always be preferable to invasive brain surgery,” Karambaiah said. “What we can do is give physicians a fighting chance to regulate the inflammatory response of TBI, rather than trying to treat it after it occurs.”
The technology has been licensed to Aruna Bio, one of UGA’s first Innovation Gateway startups. The technology gained the interest and financial support of the Georgia Research Alliance’s Venture Fund, which invested $13 million in common stock in July. The company is also looking to develop exosome therapies for neurodegenerative diseases like ALS, Huntington’s Disease, and stroke.
The company opened offices in Research Triangle Park, North Carolina. The company plans to use the funds raised to continue validating the neural exosomes as well as to deliver therapeutic payloads including siRNAs and antibodies across the blood brain barrier. It hopes to use the research data to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) in the near future.
“Drug development for acute TBI has suffered so many clinical failures and will need to take an imminent paradigm shift toward a more targeted and personalized treatment,” Stice said. “We still have a lot to learn, but our success could create the tipping point.”
