This marks my 20th post, and considering a few days ago there was an article posted about the first endometriosis drug that is currently under clinical trial, I thought it would be more than fitting as my 1st post was about endometriosis (read it here).
For some context, endometriosis (endo) is a disease affecting around 1 in 10 women of reproductive age. It’s known for being very painful, especially during menstruation, and also presents with irregular cycles and infertility. Generally, symptoms are vague and hence why it takes around 8 yrs or so for it to be diagnosed.
What is special about this drug is that it is non-hormonal, which is quite surprising as most women’s issue are dealt by prescribing the contraceptive pill. Reasons include irregular or heavy cycles, acne, dysmenorrhea (painful period), migraines, PMS, PCOS and the list goes on and on. And although it may be deemed effective at mitigating some of these symptoms, (i) it does not address the route cause of them and (ii) it is not side effect free. Basic risks include, increased blood pressure, increased risk of clots, increased risk of breast and cervical cancer; now combine this with a woman that smokes, has a bad lifestyle, high stress, quite overweight it is a recipe for disaster. In the UK it is very easy to even self prescribe yourself the pill and that comes with an even higher risk. Outside of medical side effects, it’s been proved that women that are on the pill are attracted to men with less masculine faces and voices, which it is pretty obvious long term why this may bring more issues once one gets off the pill.1 I’ll stop diverting and resume on the endo drug trial.
Another point of this potential treatment, is that it’s non-surgical. Even now, many women opt for hysterectomies (removal of the uterus) due to the pain being unbearable and the strain it has on their quality of life. If this women is still very young this means she won’t ever be able to carry children. A co-lead of the trial, Prof Horne of Edinburgh University, found in the samples of peritoneal endometriosis (affects ~80% of women with endo) which were taken during surgery significantly higher levels of lactate in their pelvises when compared to those without endo.
Lactate, or lactic acid, is naturally produced in our body, mostly during exercise or when cells are deprived of oxygen. It is a signaling molecule, which basically means it sends info between cells, which affects bodily processes. In this case, it includes blood flow regulation and the production of energy in the brain. Lactate is produced when glucose is broken down. So, it was hypothesised that as it’s in higher amounts it may have something to do with the development of endo lesions, as lactate plays a role in helping cancer cells proliferate (i.e., increasing of number of cells by division and multiplication).
The drug is called dichloroacetate (DCA)2, which has already been tested in cancer patients and is currently used to treat children with rare metabolic disorders that have excess lactic acid building up in their blood.
Current state of trial, is that the small group treated reported less pain and better quality of life. Next step, is a larger trial that also has a placebo arm, to reduce bias. Contingent on the results of the trial, if it is approved for endometriosis treatment we’re still looking at several years ahead until it goes to market for that purpose. Hopefully, if DCA is proved effective and comes with minimal side effects this solves the treatment side, with the diagnostic side still remaining.
Going back to the context bit, in order to have a confirmed diagnosis of endo you need to undergo surgery to confirm it. Which, to not point out the obvious, is not pleasant or risk free, regardless of the diagnosis. Again, it would point to similar treatment plans, as it is a diagnosis of exclusion. A lab in Yale School of Medicine, did find specific serum (i.e. blood and not endo-tissue) microRNAs having significantly higher expression and lower expression, 4 and 2, respectively. 3 What does that mean, similarly to a normal blood test, the levels of those microRNAs may be checked against “normal ranges” and that may pose as a non-surgical and non-invasive diagnostic biomarker. Of course, outside of it being complication and risk free, it is faster and cheaper for both patient and hospital. 4
Hopefully, endo research is on the right path and progress shall continue and not just linearly but exponentially.
As usual, I’ve added some links below. I hope you enjoyed - see you next week! :)