Research updates

Tofersen given green light to enter Phase 3 trial

Recent reports from a Phase 1/2 trial of tofersen revealed a significant reduction of the toxic SOD1 protein in participants with SOD1-related MND who took the drug for three months.

Tofersen is an antisense oligonucleotide (ASO), designed to prevent the faulty disease-causing SOD1 protein from being made. Proteins, the building blocks of the body, are created from our genetic information (DNA) via its photocopy (RNA). If a piece of DNA is damaged, the RNA will also be damaged, leading to the formation of a faulty protein and creating issues in the body.

Tofersen is a synthetic RNA designed to ‘stick to’ the faulty RNA preventing it from making faulty proteins. Although signs of slower disease progression were also observed in the early trial, this now must be investigated in a longer and larger Phase 3 trial, which is already recruiting participants in the USA.

In a separate clinical trial, a similar drug is also being investigated for its safety, and eventually its effectiveness, in people with C9ORF72-related MND – this trial plans to recruit people in the UK in the future. It is thought that this type of treatment has the potential to treat other types of MND, including the sporadic form. You can read more about this trial on our blog.


Antibiotics, stem cells and more

Are antibiotics to blame in MND? Antibiotics are known to significantly change the composition of gut bacteria and studies in animal models have previously suggested that these changes may be associated with MND.

Researchers in Sweden found people with MND had used antibiotics more than the general population and concluded that antibiotic use, particularly repeated use, was associated with a subtly increased risk of MND but cautioned that more studies are needed to prove a direct cause-and-effect relationship. You can read more in this article.

In other research, a Phase 1 trial using human neural stem cells (hNSCs) injected into the spinal cord of people with MND was found to be safe and well tolerated. During treatment, and over a 2-year follow up, no participants reported severe adverse effects. These results support progression into a Phase 2 trial to continue to monitor the safety of hNSCs and to assess their effectiveness on lung function and disease progression. Find out more here.

A Phase 2 trial of reldesemtiv, a successor to the unsuccessful drug tirasemtiv, yielded mixed results and failed to show a statistically significant difference in slow vital capacity (SVC) after 12 weeks of treatment. However, those who received reldesemtiv still declined less than those who received placebo, making the drug a potential candidate for slowing down lung function decline and overall disease progression in people with MND. This will be tested further in follow up trials. You can read more about reldesemtiv in the press release.

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