United2EndMND Statement on MIROCALS results

It’s now been nearly three years since completion of the MIROCALS Phase II trial and two and a half years from the announcement of the top line results in December 2022.

Finally, the long awaited peer reviewed paper, detailing the results and conclusions, was published yesterday (9/5/2025) in the highly regarded international scientific journal, The Lancet.

The MIROCALS trial used a low dose of interleukin 2 (aldesleukin). Specifically, this was reconstituted from the off-the-shelf NHS brand, Proleukin. However, after the trial a commercial arrangement was agreed between the MIROCALS Consortium and a fledgling French pharmaceutical company, ILTOO, enabling them to produce a bio-similar, but effectively new drug, known as ILT-101.

The promising top line results gave some hope to the community. However, this hope was replaced by disappointment at the long delay in publication of the final results. This has challenged the way in which clinical trials initiated by the leading MND clinician researchers and funded by charity donors and taxpayers are organised. Please see our previous articles on MIROCALS.

We are, and will be, looking out for any signals that a specific formulation choice may delay the arrival of the active drug for our patient community.

Has the journey towards finding treatments for MND entered a new phase with these results? We all hope so, but have to remember this is just the beginning. We must continue to refuel the metaphorical ‘research tank’ and put the pedal to the metal.

We sincerely hope that today marks a new start for our community and our dedicated scientists. It has to! We have to hope that lessons will be learned by everyone involved, and that we can continue to move forward with zest and, above all, positivity.

The results appear very promising indeed. However, we must first hear the opinions of our lead MND clinician researchers and await their consensus statement. It is only with a consensus that any treatment might reach patients.

Many questions will be at the forefront of patients’ minds today including…

• Given our exceptionally high unmet need for treatments, can these results justify some form of accelerated access now?

• What will the ‘further research’ described in the paper entail?

So where do we go from here?

Patients United is now asking our lead MND clinician researchers to meet urgently to

1. Form a consensus on the results and
2. Decide if they will support some form of early access for patients whilst the pharmaceutical company (ILTOO) submits a formal regulatory application to the MHRA, EMA and/or FDA for a licence, a process which will take several years.

Given the very favourable safety profile of low-dose aldesleukin, shown now in two trials (MIROCALS and IMODALS studies) should access now be prioritised? How might this happen?

We can envisage two routes forward (either/or/both):

1. ILTOO offers patients a new Managed Access Program (MAP) for ILT-101. Any such programme should be at sufficient scale to enable the drug to be accessed by as many patients as appropriate.

2. Access to low dose aldesleukin reconstituted from Proleukin, already on NHS pharmacy shelves, under NHS Specials. As previously mentioned, this was the formulation used in the MIROCALS clinical trial.

With both routes it would be important to gather real-world data to feedback into any regulatory application and further research.

We therefore ask:

Charities, MND clinicians, NHS authorities and government to step up to the challenge and ensure those living with MND receive the therapy as quickly as possible if the consensus confirms benefits for patients.

We look forward to working with them all on the next steps and hope a confirmed positive consensus will bring a welcome and much-needed boost to our community.

This is just the start. The researchers’ job is not complete. This is not a cure, but it might give many some more time. We must continue our endless campaigning for government and industry partners to invest in research.

United2endMND