Update: Access to emerging treatments for motor neuron disease

22 September 2023

My Name’5 Doddie Foundation is receiving daily requests for new information on the data from the MIROCALS trial of low dose interleukin-2. We want keep you up to date as we continue to push hard for progress with MND research and give hope to all those living with MND.

The Foundation is not a member of the MIROCALS Consortium, which is responsible for managing the data, and therefore the information we have is limited. But there’s much we can do and are doing, working hard to get early insight into the data and clarifying options for those living with MND. While not indicating a cure, the initial data presented seems to show slower disease progression in some patients.

We understand that some people living with MND are able to purchase interleukin-2 and are having it compounded into a lower dose to reflect the dose used in the trial. Although we wouldn’t encourage this in the absence of the trial data, we absolutely understand how urgently people want access to this drug. Those living with MND don’t have time on their side.

Inequity like this is unfair. We believe that, if results of the MIROCALS trial are as positive as we all hope, everyone who is eligible should be given access to the drug without delay. In the interim this could be via Proleukin®, the brand of interleukin-2 that’s currently available in the UK for the treatment of advanced renal cell carcinoma, albeit in a higher dose than was used in the MIROCALS trial. Proleukin can be compounded and reformulated into a lower dose, which was done in the UK for the MIROCALS trial itself. We’ve been involved with communication to the NHS Repurposing team to help try and make this happen as soon as the results are available.

We recognise there will be an unavoidable gap between the release of the trial data and availability of a fully licenced product for MND (currently called ILT-101), in the event that the trial data are positive. We’re planning to meet with ILTOO Pharma, the commercial partner chosen by the MIROCALS Consortium, as soon as possible to get a best estimate on how long this period will be. We believe it would be unacceptable for people living with MND to continue waiting for access to the drug, while the commercial company navigates the licence and health technology appraisal process, which could take years.

We believe a suitable temporary measure to fill that interim gap between data release and a licenced product is to allow access to low dose interleukin-2 that’s already available, provided this is via safe and approved channels.  We continue to work with anyone who can help us to achieve this goal.

Whilst we’re not members of the MIROCALS Consortium, we’re proud to have supported three research projects in the UK and Italy, which analysed samples donated by participants of the MIROCALS trial and aim to understand why some people responded better to the treatment than others. This ‘secondary analysis’ is still ongoing and the results are not needed for assessment of the overall efficacy of the drug in the trial. It’s part of our desire and focus to better understand MND and do all we can to find effective treatments and ultimately a cure.