Two New Drugs Show Promise for Endometriosis

Early research suggests that two new drug compounds could better treat endometriosis, according to a study published in Science Translational Medicine.

Scientists relying on mouse model experiments and lab cultures of human tissue found that the new compounds were effective in reducing inflammation and suppressing the development of new neurons and blood vessels that support the misplaced tissue found in endometriosis.

Key Points:

- Scientists report successful early results of treating endometriosis with two new compounds.

- The tested drugs lowered the amount of endometriotic tissue and reduced inflammation.

- This work could help inform treatments for other disorders driven by estrogen and inflammation, such as multiple sclerosis, inflammatory breast cancer, liver fibrosis, and obesity-related cardiovascular and metabolic problems.

The new compounds are OBHS (oxabicycloheptene sulfonate) and CLI (chloroindazole). OBHS interacts with the estrogen receptor ER-alpha, and CLI targets the ER-beta estrogen receptor.

While the promising research described in the journal article focused on endometriosis and human endometriotic cells, the researchers said the work could help inform treatments for a number of disorders that are driven by estrogen and inflammation. Multiple sclerosis, inflammatory breast cancer, liver fibrosis, and obesity-related cardiovascular and metabolic problems are other potential areas to benefit from these findings, they said.

"The usual treatments for endometriosis are aimed at suppressing estrogen production because it's an estrogen-driven disease," said University of Illinois molecular and integrative physiology professor Benita Katzenellenbogen, PhD, who led the new study with chemistry professor John Katzenellenbogen, PhD. "We thought that a better approach might be to interfere with both of the main aspects of endometriosis: the growth-promoting actions and also the inflammatory aspects-both of which involve the estrogen receptor.”

The lab results showed that each drug successfully reduced the amount of endometriotic tissue in mice. Also, the drugs reduced inflammation. Finally, the early indications are that the treatment did not reduce the fertility or health of the young born to mouse mothers who had received the drugs.

With years of testing still ahead, it is too soon to know the potential clinical implications, the authors said. However, the aim would be to better ease the painful adverse effects of the disease by changing how treatment targets the estrogen receptors.