Endometriosis treatment in the pipeline
About 190 million reproductive-age women and girls are affected by endometriosis globally, according to the World Health Organization (WHO). It is a chronic disease with symptoms that can render women and girls disempowered as they go about their daily routine at work, school, or home. It can disrupt them from being productive because they are having severe, life-impacting pain during periods, bowel movements and/or urination, chronic pelvic pain, abdominal bloating, nausea, and fatigue.
The US Centers for Disease Control and Prevention (CDC) said that the pain is usually in the abdomen, lower back, or pelvic areas. While some women have no symptoms at all, the first sign they have for endometriosis is having difficulty getting pregnant. It is also sometimes associated with depression, anxiety, and infertility.
The WHO explained that endometriosis is a disease in which tissue which is similar to the lining of the uterus grows outside the uterus. It can develop on the ovaries, behind the uterus, on the bowels, on the bladder, or even in other parts of the body in rare cases. The CDC said that this “misplaced” tissue can cause pain, infertility, and very heavy periods.
It can start at the first menstrual period and last until menopause. Symptoms often improve after menopause, but not always, the WHO said.
Unfortunately, there is still no known cure for endometriosis and treatment is usually aimed at controlling symptoms like those for alleviating pain. In some cases, symptoms can be treated by surgery.
The cause of endometriosis is unknown, and there is no known way to prevent it. Since endometriosis symptoms are variable and broad, diagnosing this disease can often be challenging and is sometimes dismissed. With this, women and girls with symptoms may not be aware of the condition.
Understanding the disease is the first step in biopharmaceutical research and development process. Knowing why and how a disease develops would be crucial in finding a potential treatment or cure.
The Pharmaceutical Research and Manufacturers of America (PhRMA) explained that scientists often search for biological markers (biomarkers) to help in diagnosing diseases, understanding how a disease progresses, and measuring how a body is responding to a treatment. An example of a biomarker is the use of blood sugar levels to help identify and monitor patients with diabetes and indicate how their body may react to a change in diet or treatment.
In relation to endometriosis, researchers from the University of Edinburgh earlier discovered that cells from the pelvic wall of women with endometriosis behave differently compared to those without the condition. They found that these cells produce higher amounts of lactate, a chemical generated by the body to generate energy when there is a lack of oxygen.
The high lactate level creates an environment that supports the development and growth of endometriosis.
Armed with this knowledge, researchers in England and Scotland are investigating 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.
Researchers from the Universities of Edinburgh, Aberdeen, and Birmingham are conducting a clinical trial, called EPIC2, involving 100 women with endometriosis in Edinburgh and London. Their aim is to determine whether dichloroacetate is an effective pain management treatment for endometriosis. Dichloroacetate was previously used to treat rare metabolic disorders in children.
When these endometriosis cells were treated with dichloroacetate, lactate production decreased to normal levels and the size of the endometriosis lesions were reduced. The clinical trial is building on this knowledge 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. EPIC2 is jointly funded by a partnership between leading women’s health charity Wellbeing of Women and the Scottish Government.
Dr. Lucy Whitaker, lead investigator for EPIC2, Wellbeing of Women researcher and Clinical Lecturer in Obstetrics and Gynecology at The MRC Centre for Reproductive Health, University of Edinburgh, underscored the urgent need for more treatment options for endometriosis and better ways to manage the often-debilitating pain that it causes. She expressed hope that their research will confirm dichloroacetate is a safe and effective treatment that will give hope and a better quality of life for women living with endometriosis.