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Dexpramipexole clinical trial enrols first participant

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We’re aware that thing’s may seem quiet in terms of ‘big’ MND research moments… almost too quiet…

Well, today, the pharmaceutical companies Biogen Idec and Knopp Biosciences announced that the first person has been recruited into their clinical trial to test the effectiveness and safety of a drug called dexpramipexole.

       Please note: UK trial recruitment has not yet opened.

This is exciting news as it marks an important milestone of a drug reaching a new level in its development. It’s the equivalent of the drug going to University after going to primary school, secondary school and college and passing all its exams with flying colours. It is only by successfully completing these previous phases that the drug can be tested in a large Phase III clinical trial.

With a whopping 804 people with MND set to be recruited into this trial from 11 countries around the world, in three continents, it’s certainly a big trial. Although each centre is set to recruit approximately 10 people, it’s a fantastic opportunity for people with MND from around the world to be involved in this collaborative research effort to find the answer to whether this drug is effective for MND.

With strict inclusion criteria, we are aware that a lot of people will be disappointed that they cannot participate – more information on this can be found in our news in research article on our website.

The official Biogen Idec press release can be found here.

6 thoughts on “Dexpramipexole clinical trial enrols first participant

  1. Good news but what’s preventing the UK trial going ahead? Why delay the PI meeting until May? With modern communication methods (video conferencing etc) it could be held tomorrow.

    1. Hi Jacqui, the UK part of the trial is still awaiting ethical approval and so cannot start. As soon as they get this, I believe they can begin recruitment. I was unaware that the PIs are meeting in May but it is possible that the meeting will be arranged as a teleconference rather than face-to-face – especially as the UK centres are quite far apart.

    1. Hi John, trial centres in Australia are Caulfield South, Herston, Randwick and Westmead. If you’re interested, then talk to your neurologist about the inclusion/exclusion criteria stated on www.clinicaltrials.gov and what taking part in a trial involves. I hope this helps.

  2. Hi Kelly,
    My friend is one of the people as you say above “who will be disappointed that they cannot participate in the trial”.. The doctors are saying he has had MND for 2 year, although just diagonised in May 2011. Any idea if there is any way to appeal, to help get him on the trial? Or is there any private routes we could consider fund raising for to get him on a trial over and above the standard UK tests??

    1. Hi Jan,

      Thank you for commenting on our blog article,

      I’m sorry to hear that your friend cannot take part in this trial. Unfortunately, the doctor’s decision cannot be appealed, nor can the barriers to the trial be bent.

      The reason why they have to use strict criteria for including people in the trial is so that the drug stands the best chance of demonstrating its safety and effectiveness in a controlled, unbiased situation. Also, as dexpramipexole has shown in a previous trial that it may be more effective if given at an earlier stage of disease, then the 24 months from symptom onset criterion is vital to ‘give it its best shot’. In the long run, this will mean that if it proves itself to be beneficial in this trial, then it will be licensed for use. If the trial were to open its doors to people who have had MND longer, then the results may not show its ‘true’ beneficial value and subsequently may fail the trial.

      Your friend’s case is a classic example of why we need to speed up the process of diagnosing MND by identifying MND specific ‘fingerprints’, called biomarkers which may be found in brain scans, blood tests, spinal fluid samples etc. That is why we are funding research to identify biomarkers for MND. You can find out more about these projects on our research we fund section of our website if you are interested in reading more about these.

      By finding a biomarker that can be used in clinics, MND can be diagnosed quicker, meaning that in the future people like your friend, will not have to wait two years to be diagnosed and can therefore take potentially beneficial treatments earlier on in their progression when they are thought to be most effective.

      Although your friend isn’t able to take part in this trial, there are other studies that he may be able to take part in that can lead us to understanding more about the causes and finding biomarkers, which will inevitably lead us closer to finding a better treatment for MND. You can find out more about these studies and more on our ‘get involved with research’ section of our website.

      Best wishes,

      Kelly

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