A groundbreaking clinical trial is set to offer women who have endometriosis a potential new treatment that could lead to developing the first new class of drug for the condition in four decades. The trial will involve 100 women in Edinburgh and London. It will examine the efficacy of dichloroacetate, a drug that doctors hope could relieve the often-debilitating pain associated with endometriosis. This would be the first non-hormonal, non-surgical treatment option for the condition, affecting approximately one in 10 women of reproductive age.

Dr. Lucy Whitaker, a clinical lecturer in obstetrics and gynecology at the University of Edinburgh, who is leading the research, expressed hope that the trial will give women a new optimism regarding their treatment options. Current treatment options for endometriosis include conventional pain relief, hormonal contraceptives, and surgery. However, hormone-based treatments, such as pill or contraceptive implants, can have side effects and may not be suitable for everyone, particularly those seeking to conceive. Surgery is also associated with risks and may not be effective in the long term. Studies have shown that about half of those who undergo surgery experience a recurrence of symptoms within five years.

Endometriosis is a condition in which tissue similar to the womb’s lining grows outside the uterus, causing pain and inflammation during menstruation and scar tissue formation. Approximately 1.5 million women in the UK are affected by endometriosis. Still, a lack of awareness of the condition, coupled with the need for a diagnostic laparoscopy, means that women typically wait eight years for a diagnosis after experiencing symptoms.

The trial is being funded by the Scottish government and Wellbeing of Women, a women’s health charity. The chief executive of Wellbeing of Women, Janet Lindsay, called for progress in treating endometriosis, stating that the lack of new treatments for the condition in 40 years was unacceptable. Lindsay said that too many women and girls were suffering from debilitating symptoms, such as chronic pelvic pain, fatigue, and fertility problems, and that current treatment options were not suitable for everyone.

The trial builds on previous research indicating that women with endometriosis have higher amounts of lactate, a potentially harmful waste product, in cells from the pelvic wall. Lab experiments have suggested that lactate creates an environment that fuels the development and growth of endometrial tissue. In previous mice experiments, lactate production decreased to normal levels, and the size of endometriosis lesions decreased when cells were treated with dichloroacetate. The drug is already licensed as a medicine to treat rare childhood metabolic disorders and various cancers, meaning it has an established safety profile. In a pilot study involving 30 women, the primary side effects were a slightly upset stomach on starting the medication and a tingling sensation in the fingers.

The latest trial, which will begin recruiting this autumn, will involve half of the women receiving dichloroacetate and the other half being given a placebo. Participants will take the tablets for 12 weeks. They will complete a series of questionnaires and provide blood samples over two-and-a-half years to determine whether the treatment is effective in relieving pain and other symptoms.

Dr. Ranee Thakar, the president of the Royal College of Obstetricians and Gynaecologists, welcomed the trial, stating that current treatment options for endometriosis did not work well for everyone, leaving many women with symptoms that can have a severe impact on their quality of life, affecting their physical and mental health. Thakar expressed hope that the trial’s results would improve the lives of women with endometriosis.