Should I take riluzole?

Over 100 drugs have been tried in the treatment of MND, and to date only one – Riluzole – has been shown to have a significant effect on slowing the disease course. No one would argue that the effect is large, but it is important to explain what is, and what is not fair comment about Riluzole as a treatment for MND.

It is important to note also that this page presents a personal view, and that the drug has been scientifically reviewed and approved by the National Institute for Health and Care Excellence (NICE).

What the clinical trials tell us

In the larger second randomised trial (where half the patients receive a placebo or ‘dummy pill’), after 18 months there was an approximate three-month improval in life expectancy in those MND patients taking Riluzole, which equates loosely to about a 10% life expectancy improvement. This is not the same as saying “Riluzole only gives you an extra three months”.

Major adverse events were extremely rare, and subsequent long-term studies have confirmed that the drug is generally very safe if monitored correctly.

What the clinical trials don’t tell us

As with many trials in MND, there was a large range in the type of patients included, and so the life expectancy improvement is an average – it may be slightly greater for some individuals and a little less for others. This is one of the reasons that more recent MND drug trials limit the range of patient types included.

The trial unfortunately did not include any measure of quality of life improvement (or reduction) which is now a standard aspect of most trials.

Two common, but flawed criticisms

1. “Riluzole is no better than a placebo – it just makes you feel better to take something”

This is what randomised placebo-controlled trials are for, and Riluzole was significantly better than placebo in such a trial that was well-conducted overall.

2. “Riluzole just prolongs the later, disabled stages of MND”

Biologically it seems most probable that Riluzole acts throughout the entire course of the disease.

Riluzole: the facts as we see them

1. Riluzole is not a cure for MND, but in a clinical trial it did show a small effect in prolonging life expectancy.

2. Riluzole will not make you feel better – at best you will feel no different day-to-day.

3. You will never be able to know the exact benefit/difference it made to you.

4. About 10% of patients on Riluzole will experience significant gastrointestinal symptoms or lethargy. This may improve over time, or respond to a dose reduction, but if significant then we would recommend stopping the drug. This is because we believe that for those affected individuals the small benefit does not justify feeling unwell whilst living with the many other challenges of MND.

5. Riluzole rarely causes significantly altered blood tests but these must be monitored at the start, monthly for three months, three-monthly for the rest of the year, then annually thereafter. Life-threatening blood test changes are extremely rare and amount to a handful of case reports. Small alterations in blood tests can respond to a dose reduction.

It’s all about choice

We (like NICE) believe all MND patients should be offered Riluzole and have the chance to take it if they wish. We fully support those who decide to take it, but also the significant number of people who decide it is not for them.

The way Riluzole is provided varies depending on where you live. If you choose to take Riluzole, the care centre staff will inform you how you will receive your medication.

If you have any further questions or concerns regarding Riluzole please contact the Care Centre Coordinator.