Trials and Tribulations

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Posted By Scott

The quest for a COVID-19 vaccine has made medical trials a hot topic.  Since my diagnosis in 2018, I’ve developed a new interest in such trials and the search for effective MS treatments. 

The Ultimate Goal

As I gradually came to terms with diagnosis, I started exploring the MS Society’s clinical trial pages.  From these, I learned that the Shangri-La of MS research is a drug to promote remyelination.  This would repair the damage that MS does to the nerves which could potentially reverse the effects of the disease.  If this were to be found, it would offer not just hope but an effective treatment for all MS sufferers.

One trial of such a drug caught my eye.  In the summer of 2017, a trial of bexarotene started.  This is an existing drug already licensed to treat some cancers.  I find it fascinating that drugs developed for one condition may eventually be applicable to others.  The knowledge and patience to pursue these different avenues is incredible.  Given my limited medical understanding, I can’t even guess how that even gets started. 

How do they identify that the treatment for a particular condition might also lend itself to an entirely different one?  Is there a link in symptoms or the disease between say MS and cancer?  Just in my own MS treatment, Lemtrada was originally used to treat leukaemia and the amitriptyline that I use to ease the neuropathic pain started life as an antidepressant.

Bexarotene On Trial

Anyway, the bexarotene trial involved 50 people with relapsing remitting MS, with results originally scheduled for autumn 2019.  I frequently visited the MS Society website, fingers tightly crossed that the trial would be a success.  The results date was then put back to March, and then to September 2020.  Was this a good or bad sign?  Was it a sign at all?

The MS Society announced in a tweet on 25th September that the results were available.  I eagerly clicked on the link.  It started promisingly as the trial showed that bexarotene could indeed promote remyelination, as identified on vision tests and MRI scans.  Crack open the champagne!

Not so fast.  Unfortunately, participants in the trial did experience some serious side effects, including an underactive thyroid gland and high levels of fats in the blood. This means bexarotene won’t be taken forward into a Phase 3 study.  Disappointing news.

What Next?

So, we don’t have a remyelination treatment just yet, but the report strikes an optimistic note.  The lessons learned from this trial will be taken forward into a new trial.  This will feature two existing treatments, one for diabetes and one an antihistamine, which may also promote remyelination without the side effects.  This phase 2 trial will start with 50 participants with relapsing remitting MS.

I will admit to being far too afraid of taking part in a trial, especially in the early stages.  Maybe that’s because my MS symptoms seem relatively mild at present.  If they were more severe, I might be more inclined to try an experimental treatment in the hope it would help.

This trial is not an overnight proves.  The MS Society hopes that by 2025, a range of treatments for all MS patients will be in the final stages of testing.   This horizon brings the realities of medical trials into sharp focus, particularly as the world strives for a COVID-19 vaccine and rapid timescales for it are bandied about.  I don’t think it can be a quick process. 

Safe Not Sorry

Identifying both the efficacy and safety of a new treatment must be both a rigorous and difficult process.  Take Lemtrada.  It went through trials and was approved but some serious side effects were not identified until much later. 

Read the information leaflet of an every-day drug such as paracetamol.  It’s been around for ages and you might think it would be harmless, but you’ll see a long, occasionally terrifying, list of potential side effects.  How are these found?  Is there a threshold of when a drug can be released before the long term effects are known?

I think we’d all rather be safe than sorry.  Whilst it is disappointing bexarotene isn’t the treatment all us MSers were hoping for, it does feel like a big step has been made.  I have all my fingers, and toes, crossed that within the next five years we’ll be celebrating reaching Shangri-La

You can read more about the trial and results at the MS Society’s website here.

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