Results from a recent clinical trial into MND, involving people with MND in England, have been announced and show the trial drug to be ineffective at treating the disease.

The disappointing news was announced by Trophos SA who had been conducting the Phase II/III clinical trial across Europe into TRO19622, also known as olesoxime, developed as part of the MitoTarget project.

    Olesoxime was tested for safety and effectiveness (efficacy) in 512 patients with ALS across Europe. While it has been shown to be safe and well tolerated, it did not demonstrate a significant increase in survival (compared to patients receiving the placebo along with riluzole). Full results of the trial will be published in peer reviewed scientific journals and conference presentations in 2012.

    Damian Marron, Chief Executive Officer of Trophos, said: “The results of this study in ALS are disappointing, above all for the ALS community, who urgently require new therapies that can prolong survival and improve function. We are genuinely proud to have worked closely with this community and our international partners in the MitoTarget project on this important and very well run study.”

    Olesoxime had demonstrated protective effects on motor neurones grown in the laboratory and had been shown to extend survival in mice. The drug exerted its effects through its interactions with structures called mitochondria, which are found within motor neurones and all other cells. Mitochondria have roles in several processes that are thought to go wrong in MND, including the ‘self destruct’ mechanism that ultimately destroys a sick motor neurone. Olesoxime appeared to prevent the mitochondria from triggering this self-destruct process.

    Disappointingly the results that were seen in the laboratory and in the Phase I and Phase II clinical trials did not translate into meaningful results for patients during Phase III. Phase III clinical trials are important as they aim to show whether a drug has a beneficial effect on patients. This stage of testing usually involves hundreds of patients which is enough to allow a reliable assessment of a drug's effectiveness.

    The MND Association shares the sense of disappointment that will undoubtedly be felt across the international MND community as we work together to find effective treatments for the disease.

Commenting on these findings, Dr Brian Dickie Director of Research Development said: “Although these results are intensely disappointing after the initial promising findings from the lab, it is encouraging to know that Trophos are keen to ensure that the international research community is able to learn from these results, to help steer future research.  

    “These results also highlight the imperative need of a disease specific biomarker or ‘fingerprint’ for MND. One of the possible contributing factors suggested by the company for the lack of efficacy is the fact that the delay between symptom onset and diagnosis is still too long. With a biomarker, earlier diagnosis will mean potential treatments can be tested earlier in the disease, with a greater chance of showing a beneficial effect”

    Some people with MND are still taking olesoxime as part of the ‘open label’ phase of the trial. We know that the study co-ordinators are in the process of contacting them to give advice on what to do next. If you are still taking olesoxime, please contact the study co-ordinator for advice. It’s important to remember that while olesoxime is not beneficial, it has been found to be safe and well tolerated.