Clinical trial for new endometriosis treatment offers hope to millions of women

UK clinical trial for potential new endometriosis treatment, funded by Wellbeing of Women and the Scottish Government, offers hope to millions of women  

• Researchers have been awarded nearly £250,000 by Wellbeing of Women and the Scottish Government to investigate if a drug called dichloroacetate is an effective treatment for endometriosis 
• 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 is a debilitating condition that affects around 1.5 million women in the UK, yet it is chronically under-funded and treatment options are limited 
• Wellbeing of Women and the Scottish Government are working in partnership to improve endometriosis treatment and care – a key aim of Scotland’s Women’s Health Plan.  

A clinical trial to study a potential new treatment for endometriosis is set to go ahead thanks to funding made possible by a partnership between leading women’s health charity Wellbeing of Women and the Scottish Government. 

Researchers 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. They will investigate whether a drug called dichloroacetate is an effective pain management treatment for those with the condition.  

Endometriosis affects 1.5 million women and those assigned female at birth in the UK. It occurs when tissue similar to the lining of the womb grows elsewhere in the body, most commonly in the pelvic area. This tissue (known as endometriosis lesions) bleeds during a period but has nowhere to go – and causes inflammation, pain and the formation of scar tissue.  

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. 

 

Dr Lucy Whitaker, Wellbeing of Women researcher and Clinical Lecturer in Obstetrics and Gynaecology at The MRC Centre for Reproductive Health, University of Edinburgh, is leading the research.

She said: “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.  

“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. 

Janet Lindsay, Chief Executive of Wellbeing of Women, said: “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. 

“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 Ranee Thakar, President of the Royal College of Obstetricians and Gynaecologists, said: “It is really positive news that funding has been secured to research this potential new drug treatment.

“We know current endometriosis treatment options don’t work well for everyone, leaving many women with symptoms that can have a serious impact on their quality of life, affecting their physical and mental health.   

“We look forward to the results of this trial and its potential to improve the day-to-day lives of women and people living with endometriosis.”