This page summarises our ‘understanding’ of the current status of the Mirocals trial and the potential Low Dose Interleukin 2 (LD-IL2) treatments arising from it, Proleukin & ILT-101. Please check back here regularly for updates.

It is presented as 6 questions and answers below.

Our high profile patients/supporters Rob Burrow, Stephen Darby, Ed Slater, Marcus Stewart and their partners along with Geoff Burrow on the 22/04/2024 wrote this powerful open letter to the Mirocals consortium expressing the frustration of the community about the continuing delay to releasing the results and publically asking for transparency about the choice of ILTOO to produce a new form of IL2 when Proleukin is available on pharmacy shelves if the results are positive. You can add your support by signing and writing your personal message using this form.

We continue to await the final data from the Mirocals trial. Please read this page for a full updated synopsis. The MND Association, MND Scotland and My Name5 Doddie Foundation posted this statement back in February 2024 which we thank them for. It both endorses/supports our contention that the NHS available drug Proleukin should be the drug that should be focused on in the event of successful trial. So we say a thank you to our leading charities. However, we are still waiting.

We would like to thank Filipe Morais for raising the delay of the results during the March of the day MND fundraising event in his BBC Breakfast interview. You can watch it here.

We have, however, still received no update from the MNDA and the wider consortium on plans to prepare for a positive outcome. We have asked for a clear statement which you can read here. The charities have issued this statement on Tuesday, January 9th. It is disappointing to say the least in that it is a general statement about work which should be underway already and even then uses phrases such as “coming years”. While we must await the results (and continue to question why they are delayed) we believe the scientific confidence on the outcome of the trial is sufficiently positive to PREPARE proactively to reduce the time taken to get the drug into patients’ bodies via various pathways after a positive announcement. It is also necessary to PREPARE to campaign for access, by notifying relevant government bodies of the likelihood of this drug proving to have positive benefits and its immediate availability at a reasonable price in NHS pharmacies.

1) What is the current status of Mirocals trial and the treatment candidate Low Dose Interleukin 2 (LD-IL2)?

We are still awaiting the full results of the Mirocals trial. This status remains unchanged and it’s now over a year and half since the very positive top line (provisional) results were released in December 2022! Any results remain interim (unvalidated) until finally confirmed with the full results.

2) What do we hope Low Dose Interleukin 2 (LD-IL2) might do for MND patients?

The provisional, unconfirmed, results show a substantial reduced risk of death of between 40% to 70% within the trial period of 21 months.

What could this mean to us?

If the results are confirmed as positive, it is NOT a cure, however, it is possibly a significant and meaningful slowing of the disease. It would likely mean months of extra life for many patients and possibly years for some. But there will be a lot of unknowns. For example, we might not know how long for or why etc. Only longer use might show us, if the drug is approved

3) How might UK patients obtain access to the drug if the results are confirmed as positive?

Typically for a new drug, an application to regulators around the globe, eg FDA in the USA, EMA in Europe and the MHRA/NICE in the UK needs to be made. This can typically take 18 months to several years.

Low Dose Interleukin 2 (LD-IL2) will be no different. 

It is envisaged & expected that the pharma ILTOO, who acquired the exclusive rights to the Mirocals data to produce a NEW formulation of LD-IL2 known as ILT-101, will be applying to the regulators when the data is ready.

But, in a unique aspect of this trial, the active component of LD-IL2, is NOT actually a new drug to the UK healthcare market. It is already approved for some forms of cancer, albeit at a much higher dose. It is available as the branded drug Proleukin for these uses. Proleukin is supplied in vials as a powder and is on the NHS price books at an agreed price with the manufacturer. 

Importantly, Proleukin was the drug used within the trial and was diluted/compounded to the low dose levels required.

4) How might patients gain access before the formal approval?

As the regulatory process could be lengthy, it is vitally important to explore all other avenues to seek access for this most exciting and promising treatment this century. Patients don’t have the time to wait. How might early access happen?

As Proleukin, the drug used in the trial, is an existing drug available on the NHS price books, this opens up two specific access pathways in addition to a new formulation drug regulatory application.

1. Off-label prescribing for individual patients (NHS Specials).
2. NHS repurposing with national guidance for all eligible patients.

We have encouraged and offered to work with the UK MND charities in preparing for widespread off-label prescription. Watch this space.

We have also been asking the charities to work hard for NHS national repurposing guidance on prescription so that no patients miss out. We await news on the progress of the MND Association’s application to NHS repurposing.

We received on (2/11/2023) an acknowledgement by the NHS of the Medicines repurposing application but have been informed that until the data is published in a peer reviewed scientific paper that it will be held in abeyance and placed in monitor status.

IILTOO have also announced a Managed Access Program (MAP) which promises to provide some early access to patients to the new formulation, ILT-101. However, this MAP appears only to be potentially available for ex Mirocals trial participants and patients have received confusing and inconsistent responses from both their consultants and ILTOO.

The three major UK charities, at our request, have also formally written to ILTOO on the 13th October 2023 to inquire on how patients may access the product and how long it is likely to take.

No response has yet been received.

As NO response had been received from ILTOO requesting clarity on their Managed Access Plan (MAP), on the 2/11/2023 the charities wrote again to ILTOO in November – We understand that NO response has been received from this additional letter as yet.

We are also aware of specialist MND Consultants writing to ITLOO and similarly receiving NO response as yet.

5) It is confusing, what’s this about two products, Proleukin and ILT-101?

Proleukin is the form of IL2 used in the trial and for treating some forms of cancer. It has, however, stability issues and presents some manageability challenges. If it were to be prescribed after proof of efficacy it would need recompounding (just as it was within the trial), require chilled delivery in syringes (or be administered in hospitals/care centres) and only be usable for 7 days. But it is a perfectly viable option in patientsunited2endMND’s opinion.

ILT-101 is an entirely new form of LD-IL2 under development by ILTOO pharma, that is claimed will be delivered in patient ready syringes that will last up to 36 months.

However, Patientsunited2endmnd and others have concerns about the maturity*, potential availability of, and above all whether the EMA or MHRA will deem that further testing of this new formulation will be required. This latter concern would be considerably problematic if it were to delay patients receiving LD-IL2 when Proleukin is readily available, even with its manageability issues.

We acknowledge these issues may not materialise, but, without clarification, we remain very concerned at this time.

*By maturity we are referring to bio-availability, bio-equivalence, stage of development, manufacturing capability and manufacturing quality.

6) What can we do whilst we wait?

The drug to be put forward for regulatory approval is NOT the drug formulation used in the trial.

We are now aware of a number of patients in the UK being prescribed Proleukin privately and managing the administration of the drug as a skin injection 5 days a month.

As patients, it is not for us to recommend or encourage such prescriptions, but we empathise and sympathise with patients facing our disease prognosis.

We therefore urge our scientists and the MND Association to do all they can to expedite the release of the results so patients do not have to take on such risks, and the associated financial hardship, by accessing the drug privately and having to manage its administration without mainstream NHS monitoring and assistance. There is also the health inequality issue, with the vast majority not having the means to take up such an option.

To end this update, we acknowledge that the final results from the trial may be negative. If this were the case, we and the whole MND community will accept and move on. Move on and keep up the fight.

PatientsUnited2EndMND