UK to run clinical trial for potential new endometriosis treatment

A clinical trial to study a potential new treatment for endometriosis is set to go ahead after researchers in England and Scotland have been awarded nearly £250,000 to investigate a novel drug.

Scientists from the Universities of Edinburgh, Aberdeen and Birmingham will set up and run the clinical trial, called EPIC2, which will involve 100 women with endometriosis in Edinburgh and London.

The study, supported by funding from the women’s health charity Wellbeing of Women and the Scottish Government, will investigate whether a drug called dichloroacetate is an effective pain management treatment for those with the condition.

If successful, the drug could be the first ever non-hormonal and non-surgical treatment for endometriosis and the first new treatment in 40 years.

Endometriosis, a condition that occurs when tissue similar to the lining of the womb grows outside the uterus, affects one and a half million women and those assigned female at birth in the UK.

Earlier research, funded by Wellbeing of Women, discovered that cells from the pelvic wall of women with endometriosis behave differently compared to those without the condition.

Researchers from the University of Edinburgh found that these cells produce higher amounts of lactate, a chemical generated by the body to give us energy when there is a lack of oxygen. This creates an environment that supports the development and growth of endometriosis.

When these endometriosis cells were treated with dichloroacetate, a drug previously used to treat rare metabolic disorders in children, lactate production decreased to normal levels and the size of the endometriosis lesions were reduced.

The EPIC2 research team will build on this knowledge with their clinical trial to determine the optimum dose of dichloroacetate that will provide the most benefit, both in terms of tackling painful endometriosis symptoms and limiting side-effects.

“We’re grateful to Wellbeing of Women and the Scottish Government for giving us the opportunity to progress our research and hopefully move another step closer to the reality of a new, non-hormonal and non-invasive endometriosis treatment,” said Dr Lucy Whitaker, Wellbeing of Women researcher and clinical lecturer in obstetrics and gynaecology at The MRC Centre for Reproductive Health, University of Edinburgh, who is leading the research.

“We know women with endometriosis desperately want more treatment options and better ways to manage the often-debilitating pain that it causes.

“Our research so far shows promising results that dichloroacetate can make a huge difference. I hope our new trial will confirm this and give women hope that new treatments and a better quality of life are on the horizon.”

In the EPIC2 clinical trial, which will start recruiting this autumn, half of the women will receive dichloroacetate while the other half will be given a placebo.

These will be allocated at random and taken for 12 weeks. Every woman will complete a series of questionnaires and give blood samples over the course of two-and-a-half-years.

In a move towards personalised medicine, the dose of dichloroacetate for each woman will be determined by which version of a gene called GSTZ1 they carry.

This gene is responsible for the speed at which dichloroacetate is metabolised by the body.

Some variants do this more slowly than others, which could lead to a build-up of the drug in the bloodstream and increase the risk of side effects unless the dosage is tailored appropriately.

“It is completely unacceptable that there have been no new treatments for endometriosis in 40 years. Too many women and girls are suffering from debilitating symptoms, such as chronic pelvic pain, fatigue and even fertility problems, and current hormonal and surgical treatments aren’t suitable for everyone,” said Janet Lindsay, chief executive of Wellbeing of Women.

“Endometriosis is an extremely under-funded area of women’s health, so we are very pleased to partner with the Scottish Government and invest in medical research that could transform how the condition is treated for millions of women.

“Dichloroacetate has the potential to be the very first non-hormonal and non-invasive treatment for endometriosis, which will be truly ground-breaking. With limited options currently available and no cure, advances like this are long overdue.”

Maree Todd, Women’s Health Minister for Scotland, said:“Scotland is the first country in the UK to introduce a women’s health plan, with endometriosis being one of its early priorities.

“The plan includes several actions to help improve care and support for those with endometriosis, including a vital action to invest in further research to develop much needed improvements into treatment and management options for the condition.

“I am pleased that we are jointly funding research with Wellbeing of Women into what could be the first non-hormonal treatment for endometriosis.

“It is a stepping stone to ensuring that those with endometriosis are given treatment choices that suit their needs.”

Dr Lucky Saraswat, honorary senior lecturer at the University of Aberdeen, added: “Endometriosis can have a significant impact on women’s health and quality of life so I am delighted to be involved in this project which could improve the lives of millions of women.”

“This has a potential to be a real breakthrough in the management of endometriosis. We are very excited to collaborate with Edinburgh University in the EPIC2 trial.”