Lessons learnt in the past year…update on clinical trials

Looking for a treatment for MND is the ultimate goal of the whole MND community. Unfortunately, as MND is a very complicated disease, it is not as easy as it may sound. Setting aside the sheer cost of running trials, researchers have to look at all the possible causes of MND (the genes, lifestyle and environment) and then target these with specific compounds and hoping that this strategy won’t be halted by a different biological process. This is made even harder by the large number of possible combinations of these causative factors and the many different ways these can interact.

Thankfully, lots of research groups across the world are doing their best to tackle the adverse disease mechanisms, which is why we heard lots of results of early as well as late stage clinical trials, new strategies to design better treatments in the future, and lessons learnt from previous studies.

While there was much more to hear and read at the Symposium, here we summarise the Clinical trials session (4B), where five presenters reported results and analyses of the treatments they have been investigating.Read More »

Clinical trials update from Symposium

Clinical trials determine if potential treatments are safe and aim to prove beyond reasonable doubt whether a drug is beneficial. They are therefore the gold standard of treatment research.

More information on the different types of clinical trial can be found on our website and in our information sheet on the topic.

This year the Symposium session on clinical trials looked at three drugs and one therapy. Dr Brian Dickie has posted a separate blog on one of these drug treatments – Edaravone.

A summary of the results from the drugs and treatments discussed is below. More information on each of them in detail is later on in this blog.

Ibudilast: This drug was safe and well tolerated in those who were not using non-invasive ventilation. However, these are results from an early stage trial so more research is needed to establish possible long-term benefit.

Methylcobalamin (Vitamin B12 injections): If this treatment is given early (within 12 months of diagnosis) then it showed an effect at increasing survival in a small sub-group of those taking part in the trial. This effect was not seen when the treatment was given further on from diagnosis.

Stem cell therapy: This small, early Phase 1/2 trial was testing the safety of bone-marrow derived stem cell injections into the spinal cord. The researchers found this treatment had no major side effects. Further studies are needed to evaluate the effectiveness and safety of this treatment over the long-term.Clinical trial flow chart

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