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. Continue reading

Tirasemtiv not found effective for treatment of MND

In their official press release published on 21 November 2017, Cytokinetics Inc. announced that they will not be continuing work on tirasemtiv after disappointing results in the latest Phase 3 clinical trial. The trial, known under the acronym ‘VITALITY-ALS’, tested whether the drug has a beneficial effect on the breathing function and muscle strength of people with MND. This is very unfortunate news for everyone affected by the disease, however, Cytokinetics are already testing another compound with the hope that this will be more effective and better tolerated than tirasemtiv.

Tirasemtiv is a drug that aims to improve quality of life of people living with MND by increasing strength of their skeletal muscles (controlling body motion and posture) and therefore postponing muscle fatigue. It compensates for the missing nerve signal from a motor neurone to a muscle that instructs it to contract. Tirasemtiv activates a protein called troponin by increasing its sensitivity to calcium, which is crucial for muscle contraction. Continue reading

Tirasemtiv Phase II clinical trial – results

The top-line results from the Cytokinetics Inc Phase IIb clinical trial of Tirasemtiv were announced last week. Following this announcement, detailed results were then presented on 29 April 2014 at the Annual Academy of Neurology (AAN) meeting.

Unfortunately, although the drug was found to be safe, the results concluded that Tirasemtiv did not meet its primary objective, showing no difference in disease progression, as measured by the ALS Functional Rating Scale (ALSFRS) compared to placebo matched controls. Cytokinetics Inc have stated that further study of Tirasemtiv is needed.

Continue reading

Tirasemtiv – detailed trial results to be announced next week

Cytokinetics have published a press release of their ‘top line’ results for their drug Tirasemtiv. Unfortunately, the results are disappointing.

The main aim of their most recent clinical trials was to see if there was a slower rate of progression (measured by a widely used MND clinical scale called the ALSFRS) in people with MND taking tirasemtiv compared to those taking the dummy drug. The researchers didn’t find any difference in ALSFRS scores. They’re currently analysing the results for beneficial effects on other symptoms of MND.

The full results will be presented at the American Academy of Neurology conference next week. Sam is working on a more detailed article after these have been presented.   More information about tirasemtiv can be found on our website. The press release announcing these ‘top line’ results can be found here.

Dr Belinda Cupid, Head of Research at the MND Association said: “MND clinical trials give us all new hope that something can be done to slow the progression of the disease, and it’s devastating when those trials don’t show any benefit. It emphasizes why we have to continue to fund research – finding the cause and source of the disease and then working out how to stop it.”