This is a historic moment for science: an experimental drug has succeeded in slowing the progression of the development of motor neuron disease (MND) in patients. A team of scientists from the University of Sheffield in England has been conducting clinical trials for over 6 months. They published their results in New England Journal of Medicine this Thursday 22 September. (source 1)
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The drug “Biogen Tofersen” works by targeting a genetic mutation called SOD1, which is the known cause of 2% of motor neuron diseases.
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These rare neurodegenerative diseases are characterized by a progressive loss of motor neurons that lead to motor disturbances et progressive paralysis. According to Santé Publique France, around 2,300 new cases of motor neuron disease are diagnosed each year, and 90% of these are amyotrophic lateral sclerosis, also known as Charcot’s disease.
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For 28 weeks, the researchers followed 108 patients with one of these neurodegenerative diseases and the SOD1 gene mutation. Two-thirds received the drug, while 36 of them took a placebo.
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Improved motor and lung function
After six months, the drug had significantly reduces the levels of the defective protein, but there was still no significant difference. However, the results were encouraging enough to allow the trial to be extended. Patients who had received a placebo were then switched to the real drug.
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It was still six months later that the researchers found a difference between those who started taking Biogen Tofersen early on and those who started it midway through the study. In fact, those who had taken it from day one showed up improved motor and lung function. This delay would reflect the time it takes for motor neurons to heal.
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Some patients had remarkable results. While using a wheelchair at the start of the clinical trial, one man was able to walk again without a cane. Another said he might write his Christmas cards again after receiving the treatment.
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“I think it’s really significant. I’ve done over 25 trials and never before have I heard patients report stabilization or improvement, ”study co-author Dame Pamela Shaw told the journal. The Guardian. (source 2)
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The Biogen Tofersen drug is not a miracle cure. However, it might allow slow down the progression of the disease significantly.
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The Food and Drug Administration in the United States is currently evaluating a marketing authorization application for the drug. Meanwhile, it is being offered to some UK patients as part of an early access program.
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