OSU team treats rare genetic disease with nanotech-delivered drugs

To treat a rare genetic disorder that is ultimately fatal, researchers at Oregon State University have turned to a nanotech drug delivery system.

Niemann Pick Type C1, or NPC1, affects lysosomal storage with severe physical symptoms that, half the time, occur before a patient turns 10. The compound currently used to treat the disease is an HP Beta CD drug that needs to be injected directly into the brain and can cause serious hearing loss and lung damage at its necessarily high doses. Only 0.2% of the drug is able to cross the blood-brain barrier.

To overcome this, the scientists attached the Beta drug to a nanosized lipid, which helps to carry it into brain cells. Surprising the researchers, the lipid also worked to increase the effectiveness of the drug in flushing out cholesterol. They found that, using the nanovehicle, the delivery into cells was more effective, the drug’s performance improved about 5-fold and they could use a much lower dose that wouldn’t cause such severe side effects.

What’s more, it could be delivered via an intravenous injection rather than directly into the brain, the team published in Scientific Reports.

"Right now there's nothing that can be done for patients with this disease, and the median survival time is 20 years," lead researcher Gaurav Sahay said. "The new cholesterol-scavenging drug proposed to treat this disorder, called cyclodextrin or HPβCD, may for the first time offer a real treatment. But it can cause significant hearing loss and requires multiple injections directly into the brain, which can be very traumatic. I'm very excited about the potential of our new drug delivery system to address these problems."

Ultimately, the scientists believe the system could benefit more than 50 other genetic disorders.

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