Targeted nanotherapy shows endometriosis promise

A targeted nanotherapy reduced endometriosis lesions and pain sensitivity after a single dose in mice, early research has found.

The experimental treatment uses a nanoparticle to deliver an existing drug to immune cells associated with endometriosis.

Researchers hope the approach could eventually lead to a treatment for people with the chronic condition, but it has not yet been tested in humans.

 

Kanako Hayashi, professor in Washington State University’s School of Molecular Biosciences in the College of Veterinary Medicine and a corresponding author of the research, said: “We found the disease-specific immune cell, and then we had a drug, but we couldn’t target the cell with the drug alone.

“So we used this nanocarrier that delivers the drug specifically to the disease site.”

The Washington State University team developed the therapy after previously identifying a distinct population of macrophages associated with endometriosis. Macrophages are white blood cells involved in immune defence.

They designed a nanocarrier, a specialised molecule used to transport drugs to the disease site, to target these cells without affecting surrounding cells and tissue.

The treatment uses niclosamide, a drug approved to treat intestinal tapeworms that is also being studied for other potential uses, including cancer treatment.

Previous research by the team found that niclosamide could reduce endometriosis lesions. However, the drug has low solubility, meaning large amounts are needed for it to work and limiting how long it can safely be taken.

The researchers engineered a nano-sized molecule called a dendrimer to bond with niclosamide and deliver it in a more soluble form.

In a mouse model, a single injection reduced the size and number of endometriosis lesions as well as sensitivity to pain.

Endometriosis affects roughly 190 million women worldwide and occurs when lesions similar to tissue inside the uterus grow outside it.

Symptoms can include chronic pain, severe cramps, pain during sex and infertility. Hormonal therapies can help manage symptoms but may affect fertility and bone density, while surgery can remove lesions but its effectiveness may be short-term.

Recent research has focused on immune system dysfunction in endometriosis, particularly problems involving macrophages.

The researchers are working with the WSU Office of Entrepreneurship and Innovation as they pursue patenting and commercialisation opportunities and seek support to test the technology in human trials.

Hayashi said: “Although we still need to clear multiple phases, this study is telling us the efficacy is strong, and this nanocarrier is stable, so far, for two weeks—and we think we can go longer.

“The idea is that if it’s stable for a month, you could go to the doctor once a month, get the shot, either IV or muscle injection, and then go home.”