World-first for East of England endometriosis drug trial

A world-first has taken place in the East of England with the start of a new non-hormonal drug trial for treating endometriosis.

The trial is taking place at Norfolk and Norwich Hospitals NHS Foundation Trust (NNUH).

They are testing if the treatment can reverse the painful symptoms of endometriosis and reduce the need for surgery. The drug works by blocking a protein that promotes inflammation.

Endometriosis affects 1 in 10 women in the UK, and it occurs when the cells that normally line the womb are found elsewhere in the body. This can cause heavy periods, pelvic pain and in extreme cases, scarring and damage to the pelvic organs.

The Gynaecology and Research and Development team at NNUH are playing a key role in leading this Phase II research study. The team is being supported by the National Institute for Health and Care Research (NIHR), which supports the delivery of research in the UK.

The first patient has taken part in the AMY109EU study, which is trialling the safety and effectiveness of an antibody called AMY109. The drug has been developed by scientists from Chugai Pharmaceutical Co Ltd.

Edward Morris, Consultant Gynaecologist and NHS East of England Medical Director, is UK Chief Investigator on the study. He said:

“Patients with endometriosis have inflammation, which is a key component in disease progression and if it goes on long enough it damages tissues.

“Significant new drugs for endometriosis have not come along for decades and the ones we have in daily usage are largely hormone based, which can have unpleasant side effects. The way that AMY109 works in reducing inflammation and potentially the destructive scarring of endometriosis could mean in the long-term that some women may avoid surgery for this debilitating disease.

“Whilst this is the first detailed study of a drug such as this on endometriosis, it is still early days and is likely to take several years from the realisation of a drug to it becoming readily available.”

Other hospitals in the UK are also taking part in this study. Potential participants may be able to take part in the study if they:

• Are female aged 18-49
• Have endometriosis already diagnosed by laparoscopy (incision made in the abdomen and a thin tube with a camera (laparoscope) inserted to look for lesions, adhesions and endometrioma (cyst/s)).
• Willing to have laparoscopic surgery after study treatment.

Paul Simpson, Consultant Gynaecologist, who is the Principal Investigator at NNUH, added:

“Everything so far in the treatment of endometriosis has suppressed the disease symptoms, but this new drug addresses inflammation and potentially reverses the effects of endometriosis without the need for surgery.

“It is different to every other available treatment for endometriosis because it could be disease modifying. Antibody-based treatments are already used in healthcare for treating some cancers and chronic inflammatory conditions, such as rheumatoid arthritis and inflammatory bowel disease.”

Phase I trials of the antibody have involved healthy volunteers and patients in Japan and Taiwan.

Emma Cox, CEO of Endometriosis UK, said:

“The need for more investment in research into endometriosis cannot be overstated. Left untreated, endometriosis may progress. Yet current options are limited to surgery, hormonal management, or pain killers for the chronic and often severe pelvic pain that is a symptom of the disease.

“Whilst this potential new drug is at an early stage in its development, the researchers hope it will not only reduce inflammation caused by endometriosis but may potentially reduce scarring that has already occurred. With the added benefit of being non-hormonal it could be also available to those for who contraceptives are not suitable.

"Whilst it is early days in the research process, it’s great to see much needed investment in novel new ways to manage and treat endometriosis. We look forward to seeing the outcomes of this clinical trial.”

For more information, email gynae.research@nnuh.nhs.uk.

Pictured (L-R) Matilda Matthews, Paul Simpson, Edward Morris and Amy Nichols.