One neurological challenge that may benefit from the therapy, Gaucher disease, is “a serious and rare genetic condition in which a type of lipid accumulates in cells and certain organs. The disorder can cause bruising, fatigue, anemia, low blood platelet count and enlargement of the liver and spleen, as well as poor coordination, seizures and cognitive problems in some patients; it is caused by a hereditary deficiency of a certain enzyme,” says Qi, who is a corresponding author on the study. “Patients need enzyme replacement therapy to treat this condition, but a major limitation of FDA-approved enzyme replacement therapy is failure to cross the blood-brain barrier in the body. Therefore, the treatments available are only effective for patients who have Gaucher’s affecting their internal organs, like their livers and spleens, but not their brain or nervous system.
“[Certain] nanovesicles have the ability to cross the blood-brain barrier and selectively target brain tissue, providing a biological vehicle for delivering the enzyme replacement therapy.”
In the early 2000s, Qi developed SapC-DOPS, a combination of a cell protein, SapC, and a phospholipid, DOPS, that assembled into tiny cavities can selectively target cells and deliver therapies while sparing all other unaffected cells and tissues. In the past, he has studied that nanovesicle in cancer animal models looking at brain, lung, skin, prostate, blood, breast and pancreatic cancers. Results were promising, and now the human version of this nanovesicle is now being studied in clinical trials for treatment of glioblastoma.