PULLMAN, Wash. — Researchers have engineered a drug treatment that shows rapid effectiveness against endometriosis in early testing, laying the foundation for a potential therapy for an incurable, chronic disease that affects 1 in 10 women.
The targeted nanotherapy, developed by a team of scientists at Washington State University, reduced endometriosis symptoms and disease progression after a single dose, using a mouse model. The results were published in the journal Advanced Healthcare Materials, and the researchers hope to eventually test the drug for human use.
The treatment involves targeting a cell associated with endometriosis with a commonly available drug, delivered by a tiny nanoparticle.
“We found the disease-specific immune cell, and then we had a drug, but we couldn’t target the cell with the drug alone,” said Kanako Hayashi, a professor in WSU’s School of Molecular Biosciences in the College of Veterinary Medicine and a corresponding author of the new publication. “So we used this nanocarrier that delivers the drug specifically to the disease site.”
Endometriosis is a debilitating disease affecting roughly 190 million women worldwide, in which lesions similar to tissue inside the uterus grow outside the uterus. Symptoms can include chronic pain, severe cramps, pain during sex and infertility. Many women experience years of suffering before and after the disease is diagnosed, and it remains incurable.
Hormonal therapies are used to manage symptoms, but they come with a host of drawbacks, including effects on a patient’s fertility and bone density. Young women who take those treatments often experience menopause-like effects, such as hot flashes and infertility. Surgeries can remove lesions, but the effectiveness may be short-term.
Recent endometriosis research has focused on immune system dysfunction, and particularly on problems with white blood cells known as macrophages that are key to disease prevention.
In a previous study, the WSU researchers identified a unique, distinct population of macrophages associated with endometriosis. Those cells stood out as potential targets for drug delivery with a nanocarrier, a specialized molecule that delivers the drug to the disease site without affecting surrounding cells and tissue, like a key fitting into a lock.
Next, they designed that key using niclosamide, a drug currently approved to treat intestinal tapeworms. The drug is being studied for a range of other potential uses, including cancer treatment, and it has been shown in Hayashi’s past research to reduce lesions in endometriosis. But the medication comes with significant drawbacks, including its low solubility—which means patients have to take large amounts for it to work, limiting how long they can safely take the drug.
For a disease requiring years or even decades of treatment, that’s a major limitation, Hayashi said.
The WSU team engineered a nano-sized molecule known as a dendrimer to bond with niclosamide and deliver it in a more soluble form. In an experiment using a mouse model, they found that a single injection delivered results across a range of symptoms, including a reduction in size and number of lesions and less sensitivity to pain.
Hayashi’s collaborator and corresponding author was Anjali Sharma, a former assistant professor of chemistry at WSU who is now at the University of Michigan. Other co-authors included Rishi Sharma, a former director of business development at WSU’s College of Arts & Sciences who is now at Michigan; Anubhav Dhull, a WSU graduate student in chemistry; Mingxin Shi, a postdoctoral fellow; Madeleine Harvey and Taylor Page, student research associates in the School of Molecular Biosciences; and researchers from the University of Florida.
The researchers are working with the WSU Office of Entrepreneurship and Innovation to pursue patenting and commercialization opportunities as they seek support to test the technology in human trials.
“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,” Hayashi said. “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.”
Media Contacts
- Kanako Hayashi, WSU School of Molecular Biosciences in the College of Veterinary Medicine, 509-335-7022, k.hayashi@wsu.edu
- Shawn Vestal, WSU News & Media Relations, shawn.vestal@wsu.edu
