MIROCALS update

It’s seven weeks since our last update about the MIROCALS trial and, disappointingly, the wait for the final results of the trial, testing Interleukin-2 as a treatment for motor neurone disease (MND), continues. We recognise and share the ongoing frustration we know this is causing. 

During that time, we have continued to do everything we can to speed up the process, and to use the relationships and partnerships we have to positively influence where possible.

We’ve written again to one of the trial leads, asking for an update on when the final data will be published, and for a reason it is taking so long. While they hope the data will be published soon, they are still unable to give a firm date for when this will happen by. We are also still unclear on why it is taking so long.

We met last week with ILTOO Pharma, the pharmaceutical company engaged by the MIROCALS Consortium, and asked a number of questions on behalf of the community, particularly about the Managed Access Programme (MAP). 

They confirmed that in the UK, the MAP is only open to people with MND who were on the MIROCALS trial, at least until the final data is published and a scientific consensus emerges; this reflects the fact that, as things stand, this is still an unproven treatment. 

We were naturally disappointed to hear this news about the MAP , but feel it is only right to share it with you. We know how frustrating it will be for those who hoped the MAP might offer a way of accessing the treatment in the short term. 

We are also aware that a patent application has been published online that includes some more data than was included in the press release for the initial results, but this is still incomplete and doesn’t offer the clarity needed by the clinical community or regulatory authorities. This is a normal part of a development process and therefore something we were not surprised to hear. 

So the position now is the same as it was in June – we are still waiting for the data. Once it is published, there are three possible scenarios: 

1. The data provides strong enough evidence for the drug to be immediately put into the regulatory process.
2. The data suggests the drug has a positive effect, but isn’t, on its own, strong enough evidence for it be approved. In this case, it is likely another trial would be needed.
3. The data shows the drug has no benefit, or a negative effect. In this case, there would be no further action to take. The information collected during the trial will, however, be shared with and used by researchers across the world to inform further research.

Until the peer-review results are published, we do not know which of these will be the case. But we have prepared for all three outcomes and are ready to respond as soon as the results are published. 

One of our key roles is to work with our community and to keep you informed at each step. Getting medicines to the people who need them can be a frustrating and lengthy process, even when they have been shown to be effective. But if the results are positive, we are determined to do everything we can to influence the pharmaceutical company and the regulators to make that process go as quickly as possible. 

Our ambition remains for people with MND to have equitable access to safe, effective, proven treatments.