Various chronic diseases like viral infections, cancer, diabetes, etc. are genetic. Drugs have been designed by scientists that can target and disrupt the activity of problematic genes, but these therapeutics are difficult to deliver effectively. Very few have made it to the market.
Researchers at the Northeastern University have devised a delivery system that could ensure that more of these drugs, known as oligonucleotides, reach their intended target in the body. The researchers’ work could make these treatments more effective and less expensive, as well as accelerate the development of this new class of drugs. Oligonucleotide drugs have huge promises and could potentially be considered using these for any disease with a genetic basis.
This drug is easily filtered out of the bloodstream by the kidney and liver however some of these drugs also interact with enzymes in the blood, which results in degrading them. Others can cause toxic effects in patients when the drugs are allowed to float freely. By packaging it in a defensive structure made from a synthetic polymer called polyethylene glycol, the researchers hope to insulate the drug. It looks like a microscopic bottlebrush.
The added material makes the complex too large to be filtered by the kidneys, so now it is able to circulate in the bloodstream for longer. After an hour of the injection, less than one percent of the free drug is left in the blood. This increased circulation gives a drug more time to reach the targeted cells and disrupt the intended genes. Many potential treatments have failed to make it out of the lab because a drug couldn’t stay in the body long enough to be viable. The bottlebrush structure could give these treatments a second chance.