In endometriosis, tissue similar to the lining of the uterus grows outside the organ, and these patches of tissue, called "lesions," trigger painful inflammation and heavy menstrual bleeding. In the new study, scientists fitted an anti-worm drug with nanotech armor, and it directly targeted these painful lesions in laboratory mice.
Endometriosis is a chronic, hormone-driven condition that affects roughly 10% of reproductive-age females — an estimated 190 million individuals worldwide. "The main symptoms are chronic pelvic pain and infertility," said Ioannis Simitsidellis, a postdoctoral researcher at the University of Edinburgh's Centres for Reproductive Health and Inflammation Research, who wasn't involved in the study.
In previous research with mice, study co-author Kanako Hayashi, a professor in the Washington State University School of Molecular Biosciences, identified a group of rogue immune cells specific to endometrial lesion tissue. These cells turbocharged the painful inflammatory reactions and drove the growth of more of this problematic tissue.
Enter niclosamide, a drug approved by the Food and Drug Administration to treat tapeworm infections. In recent years, clinicians have been exploring whether this medicine could also prove effective for other conditions, ranging from cancer to Parkinson's disease.
Anjali Sharma, nanomedicine specialist at the University of Michigan
So the researchers teamed up with Anjali Sharma, a nanomedicine specialist at the University of Michigan, to tweak the drug's structure. Using click chemistry — a simple reaction that bolts together two chemical building blocks — Sharma attached the niclosamide unit to a large, tree-like molecule called a dendrimer. This helped the drug dissolve better in blood.
"The drug is attached in such a way that when it is in systemic circulation, nothing happens until it goes inside the diseased cells," Sharma said. Once there, it is slowly released inside the cell over a period of two weeks. "Basically, the dendrimer is like the Uber, the folate is like the GPS, and the drug is like the passenger," she explained.
The next steps
Treating mice with a single injection dramatically reduced their pain symptoms, compared with untreated mice. It also slowed — or, in some cases, entirely halted — the growth of new lesions.
Related storiesBecause mice don't naturally menstruate or develop endometriosis, there is still significant work to do before these findings can be replicated in humans. But for now, the study is a promising stepping stone for future research, Simitsidellis said.
Even at this early stage, though, patients are clamoring to get involved in the upcoming research. Hayashi and Sharma have received hundreds of emails asking to join a future trial.
This article is for informational purposes only and is not meant to offer medical advice.
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