A new U.S. Department of Defense grant awarded to Duquesne University will develop a novel therapy for ALS and other neurodegenerative diseases using mitochondria to help restore damaged cells.

The study looks at a promising class of natural drug delivery processes called extracellular vesicles (EVs), which work as natural, cell-derived carriers for drug delivery. The EVs incorporate mitochondria during their development, and can deliver healthy mitochondria to treat neurodegenerative, cardiovascular and metabolic diseases.

“This process is much like making FedEx boxes for the drugs,” said Dr. Devika S. Manickam, associate professor of pharmaceutical sciences in Duquesne’s School of Pharmacy. “We want to create a delivery system that safely and effectively delivers healthy mitochondria to repair damaged cells.”

Drug delivery to certain parts of the body, such as the brain and cells such as neurons, is difficult because of the body’s barriers to such treatments. This is especially true of neurogenerative diseases, such as ALS, which has few effective treatments.

“ALS and similar diseases damage motor neurons, which break the signals between the brain and other parts of the body,” Manickam said. “Our goal is to see if the damaged motor neurons respond to healthy EVs once they are delivered into damaged cells. If so, the treatment could serve as part of a patient’s therapy.”

In previous studies, Manickam and her research team have explored how EVs might treat brain cell damage caused by strokes. One study showed that a subset of EVs demonstrated a greater selectivity to transfer mitochondria and increased the survival of brain cells post-stroke, which may help to prevent long-term neurological damage.

“The development of EVs has been pursued recently, so this is new territory for our field,” said Manickam, who is the lead researcher for the DoD study. “This grant will allow us to develop and deliver EVs to damaged motor neurons, which may lead to improved treatments for ALS in the future.”

Manickam recently received the Young Investigator Award in Bioengineering from the Controlled Release Society (CRS), the major international society for drug delivery research. She also presented her findings on EV mitochondria delivery to treat strokes at the CRS Annual Meeting in July.

Duquesne University

Founded in 1878, Duquesne is consistently ranked among the nation's top Catholic universities for its award-winning faculty and horizon-expanding education. A campus of nearly 8,000 graduate and undergraduate students, Duquesne prepares students by having them work alongside faculty to discover and reach their goals. The University's academic programs, community service and commitment to equity and opportunity in the Pittsburgh region have earned national acclaim.

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September 15, 2023