New DoD Grant to Develop Novel ALS Therapy

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.

鈥淭his process is much like making FedEx boxes for the drugs,鈥� said Dr. Devika S. Manickam, associate professor of pharmaceutical sciences in Duquesne鈥檚 School of Pharmacy. 鈥淲e want to create a delivery system that safely and effectively delivers healthy mitochondria to repair damaged cells.鈥�

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

鈥淎LS and similar diseases damage motor neurons, which break the signals between the brain and other parts of the body,鈥� Manickam said. 鈥淥ur 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鈥檚 therapy.鈥�

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.

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

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.

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