What is dichloroacetate, which could become the first non-hormonal treatment for endometriosis?

While theendometriosis affects one in ten women of childbearing age in the world, how is it possible that no treatment still exists today? A new British trial could aim to remedy this and thus open the way to a new treatment. Scheduled for the end of 2023, it will involve around 100 women in Edinburgh and London and assess whether the dichloroacetate can help relieve pain caused by endometriosis.

What are the current treatment options? No treatment exists today to treat and eliminate this chronic inflammatory pathology. To relieve pain, two options are nevertheless offered to women with endometriosis:

  • A hormonal contraceptive treatment : continuous pill or contraceptive implant. This technique allows to block the rules to try to stop the progression of the lesions;
  • Surgery, when hormonal treatment is not enough: the surgeon will remove (almost) all visible lesions.

Nevertheless, hormonal contraceptives can have serious side effects (low or loss of libido, weight gain, depressionvaginal dryness) and may not be suitable for all patients, and surgery does not prevent the risk of recurrence: in the context of an operation for deep endometriosis, 30% of patients usually need another surgery within seven years.

It is totally unacceptable that there has not been a new treatment for endometriosis in 40 years.”indignant Janet Lindsay, director of Wellbeing of Woman, which funds the trial, with the Guardian. “Too many women and girls suffer from debilitating symptoms, such as chronic pelvic pain, fatigue and even fertility issuesand current hormonal and surgical treatments are not suitable for everyone.”

How does dichloroacetate work?

In previous experiments conducted in mice, the results of which were published in the journal PNAS in december 2019, researchers from the university of edinburgh showed that the administration of dichloroacetate reduced the production of lactates, molecules normally produced by the muscles when they lack oxygen but produced in an abnormally high way by the cells pelvic walls of women with endometriosis.

This drug is far from being unknown: it is used today in the treatment of childhood metabolic disorders or certain cancers. After an initial pilot study, the main side effects reported were mild stomach problems and tingling sensation in the fingers.

The recruitment of this last trial will begin in the fall, half of the women recruited will receive dichlorocetate and the other a placebo for 12 weeks. “We know that current endometriosis treatment options don’t work well for everyone, leaving many women with symptoms that can seriously impact their quality of life, affecting their physical and mental health.”, said Dr Ranee Thakar, president of the Royal College of Obstetricians and Gynecologists. An immense hope for many women with endometriosis, sometimes feeling misunderstood or abandoned.

Source :

  • Researchers optimistic about potential new treatment for endometriosis, The GuardianMarch 8, 2023

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