Researchers at Washington State University have engineered a targeted nanotherapy that reduces endometriosis symptoms and disease progression after a single dose in a mouse model. Published in Advanced Healthcare Materials, the discovery offers a potential new path for treating a chronic condition affecting roughly 190 million women worldwide.
Endometriosis remains an incurable, chronic disease impacting roughly 1 in 10 women, characterized by the growth of tissue similar to the lining of the uterus outside the organ itself. Patients frequently endure years of suffering before receiving a diagnosis, dealing with debilitating symptoms such as chronic pain, severe cramps, pain during sex, and infertility. While current management relies heavily on hormonal therapies, these treatments come with significant drawbacks, including negative effects on bone density and patient fertility, often inducing menopause-like symptoms like hot flashes in young women. Surgical removal of lesions offers an alternative, but its effectiveness is often short-term. Meanwhile, in a separate neurological breakthrough reported in Alzheimer’s & Dementia, Dr. Satyabrata Kar and colleagues at the University of Alberta found that a family of nanoparticles shows promise in treating Alzheimer’s disease without additional medication. Furthermore, Dr. Cheta Otodile, the ART Coordinator HIV/AIDS resource centre in Rivers State, noted regarding a separate public health initiative that Nigeria adopted the Differentiated Service Delivery (DSD) policy in 2019 to help manage HIV, a virus that attacks the body’s immune system and if not treated can lead to AIDS (acquired immune deficiency syndrome).
Targeting Macrophages With a Specialized Nanocarrier
Recent research points toward immune system dysfunction, specifically involving white blood cells called macrophages that play a key role in preventing disease. In earlier work, scientists identified a unique population of macrophages associated with endometriosis. According to Kanako Hayashi, a professor in Washington State University’s School of Molecular Biosciences in the College of Veterinary Medicine and a corresponding author of the study, researchers struggled to reach these specific disease-associated cells directly.
Hayashi explained that they found the disease-specific immune cell and had a drug, but were unable to target the cell with the drug alone, leading them to use a nanocarrier to deliver the drug specifically to the disease site.
To solve this delivery challenge, the research team utilized a specialized molecule known as a nanocarrier. This structure operates like a key fitting into a lock, transporting medication directly to the disease site without impacting surrounding cells and healthy tissue.
Repurposing Niclosamide for Enhanced Solubility
The team designed their targeting mechanism using niclosamide, a medication currently approved to treat intestinal tapeworms. While niclosamide is undergoing study for various other applications—including cancer treatment—and has shown success in Hayashi’s past research for reducing endometriosis lesions, it suffers from a major chemical limitation: low solubility. Because of this property, patients must take large amounts of the drug for it to work, which restricts how long they can safely use the medication. For a chronic condition requiring years or decades of management, that low solubility presents a severe obstacle.
To overcome this, the Washington State University team engineered a nanosized molecule called a dendrimer to bond with niclosamide. This formulation delivers the drug in a significantly more soluble form.
Single-Dose Efficacy in Mouse Models
In experimental testing using a mouse model, the targeted nanotherapy demonstrated rapid effectiveness after just a single injection. Researchers observed a reduction in both the size and number of lesions, alongside decreased sensitivity to pain across a range of symptoms. Anubhav Dhull served as the lead author on the study alongside corresponding author Hayashi and collaborator Anjali Sharma, a former assistant professor of chemistry at Washington State University who is now located at the University of Michigan.
The findings were detailed in the journal Advanced Healthcare Materials under the title 2‐Deoxyglucose Dendrimer‐Enabled Niclosamide Delivery to FRβ‐Expressing Macrophages Alleviates Endometriosis Progression and Associated Hyperalgesia.
Toward Potential Human Testing and Monthly Care
With early animal testing complete, the research team hopes to eventually test the drug for human use.
Hayashi suggested that if the treatment is stable for a month, patients could potentially visit a doctor once a month for an IV or muscle injection before returning home.