Could this be the solution to endometriosis we have all been waiting for?

Endometriosis is a chronic condition with no known cure and can lead to debilitating pain and infertility for up to 1.5 million women in the UK – but hope for new treatments may finally appear on the horizon.

Thanks to recent breakthroughs in our understanding of the condition, there is promise that trials of two new treatments could provide long-term relief for patients.

One drug, dichloroacetate, will soon be tested in a clinical trial of 100 women with endometriosis in London and Edinburgh. This follows a smaller study that found patients reported fewer painful symptoms and required fewer painkillers when taking the treatment.

The other new therapy involves monthly injections of a man-made antibody that has been shown to partially reverse endometriosis in monkeys — and will now be tested in women.

The condition affects women of all ages and can cause pain, heavy periods, nausea and diarrhea. It is also a cause of infertility: 30 to 50 percent of women who have trouble conceiving have endometriosis.

The condition occurs when tissue similar to the lining of the uterus grows elsewhere in the body. This can block the fallopian tubes (the tubes where eggs travel down to reach the uterus) or form scar tissue, making it more difficult to get pregnant.

While this rogue tissue is often found around the ovaries or just outside the uterus, it can occur throughout the body, including the bladder and bowel. In rare cases, it has even been found on the spine and lungs.

It causes pain as the tissue builds up and then breaks down — just as the lining of the uterus does every month — but this bleeding has no easy way to leave the body, leading to inflammation.

Currently, standard treatment is with hormone suppressing drugs, including the combined contraceptive pill, the progesterone-only pill, and contraceptive implants. These limit or stop the body’s production of estrogen — the hormone that encourages tissue to grow and shed.

But because this treatment stops menstruation, it is not suitable for women trying to conceive; and progesterone-only treatments can also lead to weight gain.

Although surgery to remove the scar tissue can relieve symptoms, the condition often recurs.

One of the new treatments being trialled – an antibody called AMY109 – has been found to reduce inflammation and partially reverse endometriosis in monkeys.

What’s more, it did so without disrupting the menstrual cycle or causing weight gain, according to a study published in the journal Science Translational Medicine.

AMY109 blocks an inflammatory protein (interleukin-8) produced by a gene, IL8, that Japanese researchers found to be more active in endometrial tissue.

Scientists at Chugai Pharmaceutical have designed an antibody to clear interleukin-8, effectively preventing it from causing further inflammation.

In a six-month trial of 17 monkeys with surgically induced endometriosis, scar tissue nearly halved in those who received monthly AMY109 injections; abnormal tissue continued to grow in the sham treatment group.

The trial showed that AMY109 had no effect on menstrual cycle or weight. Chugai Pharmaceutical is now conducting a trial of AMY109 in human volunteers.

“After a long period when endometriosis received little attention from scientists, there is now much more interest in finding treatments for the disease, which can have serious consequences for quality of life,” Emma Cox, CEO of Endometriosis UK, told Good Health. . The charity welcomes the Japanese research, she says, although more is needed “to establish whether it will become a useful therapy in the long run.”

This was echoed by Ertan Saridogan, a professor of gynecologic surgery at University College London. He added that the reduction of scars with AMY109 was particularly interesting. “With the treatments available, we haven’t been able to fight against scars that have already formed and we’ve been limited to surgery to address this,” he says.

Professor Saridogan says endometriosis probably has multiple causes, so a single cure is not immediately likely. “However, science is making huge strides and it wouldn’t be impossible to see game-changers in five to ten years.”

Another possibility being explored is the use of dichloroacetate (DCA) to reduce scarring and pain. DCA is already used to treat some solid tumors and hereditary mitochondrial disorders (where the mitochondria, or ‘powerhouse’ of the cells, do not produce enough energy for the body to function properly).

The use of DCA for endometriosis is now being studied at the University of Edinburgh: it is thought to reduce lactate, a substance produced by muscles and red blood cells, which research shows is higher in women with endometriosis.

“If you target lactate, it can reduce lesions and pain,” explained Andrew Horne, a professor of gynecology who is leading the research in Edinburgh. Previous laboratory experiments have suggested that lactate creates an environment that supports the development and growth of endometriosis.

“When these endometriosis cells were treated with DCA, lactate production dropped to normal levels and the size of the endometriosis lesions was reduced,” says Dr. Lucy Whitaker, Clinical Lecturer in Obstetrics and Researcher.

In a new trial, 100 women will take DCA pills twice a day for 12 weeks. If successful, it could herald larger trials “and DCA could have the potential to be the first non-hormonal treatment for endometriosis,” says Professor Horne.

Another trial in Edinburgh is looking at a new CBD-based drug. “These substances are extracted from cannabis and are promising treatments for pain conditions such as endometriosis,” says Professor Horne.

Studies suggest that CBD may reduce pain by increasing levels of a chemical compound, anandamide, which binds to cannabinoid receptors in the body and may reduce pain perception.

The Edinburgh team plans to recruit 100 women with endometriosis pain later this year for a 12-week study. The participants are randomly assigned to take either a CBD liquid or a placebo and fill out a questionnaire about their symptoms.

There are many theories about what causes endometriosis, including that infections can trigger it in people who are genetically predisposed, says Shaheen Khazali, a gynecologist and endopelvic surgeon at The Lister Hospital in London.

There are three types of endometriosis, depending on the location.

The most common is superficial peritoneal endometriosis, which affects the peritoneum (the membrane that lines the abdominal cavity) and is found in 80 percent of diagnosed women.

Cystic ovarian endometriosis, in which the tissue forms “chocolate cysts” on the ovaries (supposedly because of their color), affects 17 to 44 percent of patients. Less common is deep endometriosis, which has invaded tissues and organs, including the bowel and bladder. It can grow into nerves, usually the sciatic nerves, that run down each leg.

It is possible to have more than one type and it is not clear if they all behave in the same way.

One innovation that could quickly improve our understanding of the disease is gene mapping. The cells involved in endometriosis have now been genetically mapped as part of a small study at Cedars-Sinai Medical Center in Los Angeles, California.

When researchers examined cells from the pelvic tissue of 21 women (17 diagnosed with endometriosis, four without), they found a genetic mutation that apparently affects the lymphatic vessels surrounding the abnormal tissue.

Previous studies suggest that endometriosis tissue can spread through the lymphatic system, the body’s drainage system.

There have been improvements in surgical procedures in the meantime. A new technique used by Mr. Khazali is laparoscopic alcohol sclerotherapy, in which the chocolate cyst is first drained and then a 96 percent alcohol solution is applied to the inside through keyhole surgery, after which it is removed after a short period of time.

This promotes the formation of adhesions, filling the space and preventing fluid from accumulating in the cyst. Because this only targets the cyst, it reduces the potential damage to surrounding ovarian tissue.

Earlier diagnosis is key – but the average waiting time is eight years, says Emma Cox. When it comes to identifying the cause of pelvic pain, “asking patients to keep a pain and symptom diary is a fairly simple and useful tool, but it’s often not done,” she says.

Currently, the only definitive way to diagnose endometriosis is through a laparoscopy (where a small camera is inserted into the abdomen). However, waiting lists for NHS gynecology in the UK have grown by more than 60 per cent since the start of the pandemic.

When there is a high probability that endometriosis is the cause of the symptoms, Professor Horne says it may be better to start treatment as soon as possible – because ‘untreated painful symptoms tend to worsen over time’.