Taking the Drug a Little Longer?

Motor Neurone Disease (MND) affects around 5000 people in the UK. Patients, with this condition, progressively lose their ability to control their voluntary muscles. This is because nerve impulses fail to activate their muscles's motor end plates. Consequently, MND sufferers lose the ability to walk, use their hands or even breathe (https://www.theguardian.com/society/2022/sep/21/experimental-drug-slowing-motor-neurone-disease). The Biogen company developed a drug, Tofersen. This targeted the SOD1 genetic mutation, accounting for circa 2% of MND cases. Initial trials with Tofersen were disappointing. A new study, however, extended the drug's treatment for an additional 6 months. There were then indications that Tofersen slowed the development of MND. Some patients showed apparently striking improvements in their ability to walk or write Christmas cards. This, of course, was not a double blind experiment (finding appropriate and willing subjects with the SOD1 mutation would be difficult). Psychological effects could account for some of the 'improvements'. These findings mean, however, that further trials are well worth doing. Perhaps other drugs, targeting different genes that impair motor end plate function, should also be explored.