SHEFFIELD, England (Commonwealth Union) – Motor neurone disease (MND) are usually characterized by slurred speech, difficulty swallowing, cognitive changes, clumsiness and many more. Researchers are optimistic of a new genetically-targeted treatment for MND could become a major step forward for patient care, after the findings of a Phase 3 clinical trial demonstrated important physical benefits for patients following 12 months.
Scientists from the Sheffield Institute for Translational Neuroscience (SITraN) saw patients with a faulty SOD1 gene responsible for 2 per cent of MND cases, have seen that the advancing of their symptoms slowed down 12 months after the administration of the investigational medicine tofersen.
108 MND patients who had the faulty SOD1 gene participated in the innovative Phase 3 clinical trial funded by biotechnology firm Biogen Inc. Although a vital clinical improvement was not found at the primary endpoint of the study at 28 weeks, when the trial was extended, marked differences in patients’ motor function and lung function were noted.
Findings of the trial, demonstrate that biomarkers in patients’ spinal fluid indicated a reduction in the SOD1 and neurofilament protein levels after the tofersen administration for 6 months, suggesting that the treatment favorably hits the therapeutic goal and lowers loss of motor neurones which may permit them to begin regenerating connections with muscles in the body. But more time was taken for patients to witness the physical improvements in their findings.
“What we have found is that we can reduce or slow damage from happening biologically, but it takes more time for the motor neurones to heal and regenerate their connections with the muscles. So, the motor system needs time to heal before we see a physical and clinical change,” said Professor Dame Pamela Shaw, Professor of Neurology and Director of SITraN at the University of Sheffield.
