Following the publication of the MIROCALS trial data in the Lancet on Friday, 9 May, MND Scotland welcomes the prompt response from the Clinical Studies Group (CSG). This group brings together the UK’s leading neurologists working in the area of MND.

We believe the CSG meeting is the first time the UK MND clinical community has formally come together to look at a trial publication together. This collaborative approach is very much welcomed by MND Scotland, and we would like to thank the CSG for convening at short notice to discuss the MIROCALS trial data.

The publication of clinical trial results is always an important moment, and we would like to thank the MIROCALS consortium and authors for delivering a rigorous trial, testing low dose interleukin 2 (IL-2) on survival of people with MND, and making the data publicly available via a leading international journal.

The Clinical Studies Group statement is available to read here.

The publication in the Lancet can be accessed here.

Read our initial response to the trial results here.

While the CSG felt the trial results were promising, ultimately there was no overall effect of the treatment (low dose IL-2) on the number of people who had died by the end of the trial, 21 months.

A sub-analysis, which grouped people into those with low levels of a biomarker compared to high levels, showed a significant reduction in the risk of death in those with low levels, but no effect for those with high levels, when receiving low dose IL-2. It should be noted that this did not halt the disease and people receiving the treatment still experienced disease progression and died during the trial.

Considering all this information, the CSG concluded that more data is needed to determine whether this treatment effect in the 70% of trial participants who had low levels of the biomarker was real.

There are a number of ways that additional evidence can be collected in order to definitively determine the efficacy of a possible new treatment. One would be to run a larger phase 3 trial – this trial was a Phase 2b – and this is the option the trial authors propose.

Another option would be real world data collection, for example via a managed access programme (MAP). Although this might be a quicker option than a Phase 3 trial, such an approach will not only require funding but also agreement from the clinical community and the NHS.

The three main MND charities are reaching out to ILTOO, the company that manufactures low dose interleukin 2 (IL-2), to determine their plans in the UK. We will also continue to work with the CSG and the regulators to progress making proven new treatments available to people living with MND.