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Early research points to possible new endometriosis treatment

Tuesday 3 December 2019

"Cancer drug offers hope of new endometriosis treatment for millions," reports The Sun.

Endometriosis is a condition that can cause a great deal of distress and pain in women. It can also cause infertility. It happens when cells similar to those that make up the tissue lining the womb grow outside the womb, elsewhere in the pelvic region.

At the moment, treatment for endometriosis is not always effective.

Medicines include painkillers and hormonal treatments, but they're usually unsuitable for women wanting to get pregnant.

Surgery works for some women, but in half of cases the problem returns within 5 years.

Researchers investigated the effect of a drug called dichloracetate, that has previously been tested against cancer (although it is not licensed as a cancer treatment).

In the laboratory, they tested the drug on cells from women with endometriosis, and on mice that had been given an endometriosis-like condition.

The results were positive. In both cases, the dichloracetate reduced the growth of new endometriosis cells.

However, these type of studies cannot tell us whether the treatment will work in women. The researchers say they are now recruiting women for a clinical study.

Find out more about endometriosis

Where did the story come from?

The researchers who carried out the study were from the University of Edinburgh. The study was funded by the Medical Research Council and the charity Wellbeing of Women.

It was published in the peer-reviewed medical journal Proceedings of the National Academy of Sciences on an open access basis, so it is free to read online.

The study was covered in the Mail Online and The Sun. Both media outlets gave a fairly balanced and accurate account of the study. However, the headlines suggest that a treatment is imminent, whereas these are very early studies and it is likely to be years before clinical trial results tell us whether dichloracetate could be a safe and effective new treatment for endometriosis.

What kind of research was this?

This was a laboratory study involving a combination of experimental studies conducted on cells obtained from women with endometriosis, and on laboratory mice.

While this type of early-stage research is important in the development of new treatments, it cannot tell us whether it will work as a treatment for women with endometriosis.

Many drugs that show potential in early animal and cellular research do not make it through all of the subsequent testing and trial stages, as they are found to be ineffective or associated with harms in humans.

What did the research involve?

The human cell samples were obtained from the lining of the abdominal cavity of women who had attended a pain clinic and had keyhole surgery to investigate the cause. Some women were diagnosed with endometriosis (the doctors could see visible signs of endometriosis during surgery), while others did not show signs of the condition.

Researchers studied the cell samples and found that those from women with endometriosis were breaking down glucose abnormally – they produced more of a waste product called lactate. The researchers think the excess lactate may encourage growth of endometriosis tissue.

They then treated the cells of women with endometriosis with either dichloracetate, water or dichloracetate plus a protein called transforming growth factor (which is thought to promote this abnormal glucose breakdown and the development of endometriosis).

They measured the cells' lactate production and the growth of further endometrial tissue.

Finally, the researchers induced a condition similar to endometriosis in female mice. The mice were given either water or dichloracetate solution for 7 days. Researchers then measured the lactate levels and amounts of endometriosis tissue in the mice's pelvic cavities.

What were the basic results?

Results from human endometrial cells:

  • treating the cells from women with endometriosis with dichloracetate reduced their production of lactate, even when they were also exposed to transforming growth factor
  • treating with dichloracetate also seemed to reduce the growth of further endometrial cells, possibly because of the reduction in lactate production

Results from mice:

  • mice treated with dichloracetate had lower concentrations of lactate in the fluid of their abdominal cavity
  • mice treated with dichloracetate had smaller endometriosis growths

How did the researchers interpret the results?

The researchers said: "These findings provide the rationale for targeting metabolic processes [cell activity] as a non-contraceptive treatment for women with endometriosis either as a primary non-hormonal treatment or to prevent recurrence after surgery."


Endometriosis is difficult to treat. An effective option that does not involve hormone treatment or surgery would be very welcome.

While these results suggest that dichloracetate is worth researching further, it is far too early to know whether this could in the future become a safe and effective new treatment for endometriosis.

If you have symptoms of endometriosis, see a doctor. It can sometimes take many months to get diagnosed as other possible causes of pain have to be ruled out.

Although there is no cure for endometriosis, there are treatments available. These include:

  • painkillers such as paracetamol and ibuprofen
  • hormonal treatments such as the contraceptive pill
  • surgery

You may also find it helpful to contact a support group, such as Endometriosis UK, for information and advice.

Analysis by Bazian
Edited by NHS Website

Links to the headlines

Cancer drug offers hope of new endometriosis treatment for millions, docs say

The Sun, 3 December 2019

Controversial cancer drug may offer hope to millions of women battling endometriosis as scientists find it can reduce size of painful lesions

Mail Online, 3 December 2019

Endometriosis could be treated with cancer drug, study suggests

Medical Press, 3 December 2019

Links to the science

Horne AW, Ahmad SF, Carter R, et al

Repurposing dichloroacetate for the treatment of women with endometriosis

Proceedings of the National Academy of Sciences of the United States of America published online 2 December 2019