Researchers at the Centre for Reproductive Health have carried out a clinical trial to test dichloroacetate as a potential non-hormonal treatment for endometriosis. Work in the laboratory at the EXPPECT Centre, Institute for Regeneration and Repair, has previously discovered that in women with endometriosis, the cells lining the pelvic cavity undergo metabolic changes that create an environment supporting the growth of endometriosis lesions. In laboratory models these changes could be corrected with a treatment called dichloroacetate (DCA). The paper, published today, reports the results of a small preliminary study in which 30 women with endometriosis were given DCA for twelve weeks. As DCA has already been used as a treatment for metabolic disorders in children and has been shown to be safe for use in humans, this was a feasibility study to see if this potential treatment plan is practical for endometriosis patients. The study showed that endometriosis patients were willing to be recruited to a study using DCA, though the COVID-19 pandemic impacted the study delivery and not all follow-up study visits were completed. Many participants reported experiencing less pain and using less pain medication while taking DCA. Some participants experienced mild side effects, like tingling in their fingers. Genetic analysis revealed that the version of an enzyme responsible for clearing DCA from the body appeared to influence the risk of side effects. DCA now stands out as a promising potential non-hormonal treatment for endometriosis and a larger placebo-controlled study is planned.A placebo-controlled study is the necessary next step to assess the drug’s actual effectiveness on symptoms, so researchers can tell what effects are due to the drug itself and what effects might be due to the placebo effect. This proof-of-concept study has given us promising early insights into the potential for DCA as a non-hormonal treatment for endometriosis. However, we still need to carry out a large, randomised placebo-controlled trial before we can be confident if it is truly effective. We also need to the determine how someone’s genes affect the dose of DCA, in order to minimise side-effects. We are very grateful for the funding from the Scottish Government and the charity, Wellbeing of Women, that we have been given to deliver this next trial. Dr Lucy Whitaker Senior Clinical Research Fellow and Honorary Consultant Gynaecologist Dr Lucy Whitaker Lucy's research focuses on precision treatment to reduce endometriosis-associated pain, including clinical trials of non-hormonal treatments and surgical interventions, and novel imaging techniques to identify and profile endometriosis. Her clinical work focuses on management of endometriosis, chronic pelvic pain and menopause in the South-East Scotland BSGE Centre for the management of endometriosis in the Royal Infirmary of Edinburgh Read the paper in The Lancet EXPPECT Edinburgh Wellbeing of Women Dr Lucy Whitaker's Research Tags CRH Publication date 06 Nov, 2025
