A clinical trial for a drug typically used to treat metabolic diseases in children is moving into its second phase
Dichloroacetate (DCA), a drug typically used to treat metabolic diseases in children, has been seen to be effective in breaking down the cells that cause endometriosis – a painful gynaecological disease where cells similar to those that line the uterus grow elsewhere in the body.
The condition affects one in ten women globally and causes debilitating symptoms – including severe pain, fatigue, gastrointestinal issues, painful periods, pain during sex, and infertility.
There’s currently no cure for endometriosis, which is estimated to cost the UK economy £8.2bn a year in treatment, loss of work, and healthcare costs.
Management options available – including hormonal contraception, laser surgery, and even hysterectomies – can cause further complications for sufferers, and often fail to get the progressive condition under control.
This spring, Andrew Horne, Professor of Gynaecology and Reproductive Sciences at the University of Edinburgh, is moving a clinical trial of DCA in endometriosis cases into its second phase – with hopes the drug could be prescribed as a treatment on the NHS within the next five years.
Women with endometriosis produce more lactate – a harmful human waste product of glucose metabolism – than those without the disease. Professor Horne and his team found that increased lactate created an environment that supported the growth and spread of endometriosis cells. Targeting the cells with DCA was subsequently found to reduce lactate production – which could halt the progression of the disease in its tracks.
At the end of the clinical trial’s first phase, women who were given DCA reported significant improvements in symptoms as well as a reduction in the amount of pain medication they needed during their menstrual cycle.
“We’re really excited about the next phase of the trial. There have been very few trials of non-hormonal treatments, and so we are largely the leaders in this field,” Prof Horne told The Telegraph.
“We hope the drugs will reduce the disease volume, and our studies so far have given us an indication that they don’t only affect the symptoms but the disease and prevent it from progressing,” he continued.
The trial is still in its preliminary stages, and Horne and his team of researchers hope the launch of the next phase, EpIC2, will give a more definitive answer on DCA’s efficacy.