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Nanotech-based drug system may help treat rare genetic disorder

Xinhua, September 4, 2016 Adjust font size:

Researchers have reported that a nanotech-based drug delivery system may offer new hope for patients with a rare, ultimately fatal genetic disorder, known as Niemann Pick Type C1 disease, or NPC1.

No treatment currently exists for NPC1, which affects about one in every 120,000 children globally and results in abnormal cholesterol accumulation, progressive neurodegeneration and eventual death. While a compound that shows promise is now undergoing clinical trials, its high doses necessary cause significant hearing loss, requires direct brain injection and causes lung damage.

In a study published this week in Scientific Reports, researchers at Oregon State University (OSU) and other institutions outline the potential for the new delivery system to carry the new drug into cells far more effectively, improve its efficacy by about five times, and allow use of much lower doses that may still help treat the condition without causing severe hearing loss.

"Right now there's nothing that can be done for patients with this disease, and the median survival time is 20 years," Gaurav Sahay, an assistant professor in the Oregon State University/Oregon Health & Science University College of Pharmacy, and corresponding author on the new study, was quoted as saying in a OSU news release.

With the new approach, the HP Beta CD drug is attached to an extraordinarily small, nanotech-sized lipid particle that can carry it into cells, where it helps to flush out cholesterol.

Surprised to discover that the carrier itself also helped address the problem, while working in synergy with the drug it carries to greatly increase its effectiveness, the researchers said the system should allow use of much lower dosages, and possibly an easier delivery through intravenous injection, instead of brain injection.

In the form currently used, only 0.2 percent of the drug is able to cross the blood brain barrier.

The researchers noted that this type of drug delivery system has several advantages, including prolonged circulation times, the ability to incorporate multiple drugs with different mechanisms of action, and a variety of "targeting ligands" that can help cross the blood brain barrier. And the same system may ultimately show similar benefits for 50 or more other genetic disorders. Endit