“This community deserves better, time is something the MND community doesnt have, so to wait almost two years for trial results is unnaceptable” - Dean Fox, Founder of the Dean Fox MND Foundation

Introduction:

A 21 month trial was conducted using a low dose Proleukin drug, which is currenlty in use for treating cancer patients. The headline results were released in December 2022 and showed very psotive and encouraging results, with an substantial and meaningful redced risk of death ofbetween 40 and 70% acroos the period of the MIROCALS trial.

However, thes results remain as interim (unvalidated) until the full results are released from the company.

1. What could this mean for MND Patients?

 Whilst this isnt a cure it did show a real meaningful slowing of symtpoms that could extend patient life my months or even years in some cases and would be the first major treatment available for many years.

2. So what are we waiting for?

In 2022 the pharmaceutical company ILTOO based in France, acquired the full rights to the Mirocals trial data, so far they have yet to release that data to the MND community despite calls from the MND Association and other majr charities.

The MND community is still waiting for the outcome of these trial results.

Its further complicated by the fact that the drug used in the trial, Proleukin, is already in use by the NHS for treatment of cancer patients. ILTOO have suggested they will be developing a new version of the drug to submit for regulatory approval, however this process could take months or even years befoer approval is granted.

3. Are there any other options?

As this regulatory process could be extremely lomg, we feel its important to explore all other avenues to seek access for twhat could be the most exciting and promising treatment this century. The MND community doesnt have the luxury of time!

So how might patients get early access to the drug?

As Proleukin,which was the drug used in the trial, is an existing drug available on the NHS, this opens up two other routes in addition to a new drug regulatory application.

1. Off-label prescribing for individual patients (NHS Specials).

2. NHS repurposing with national guidance for all eligible patients.

4. Is there anything we can do while we wait?

Its worth noting that the drug to be put forward by ILTOO for regulatory approval is NOT the drug formulation used in the trial and this could significantly extend the time take for approval

We are also aware of some patients in the UK being prescribed Proleukin privately and obviously tht comes at a cost and without the standard NHS monitoring protocol.

Alongside other foundations and charities within the MND community we are urging the 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.

Finally we are aware that the trial results, when finally released, could be negative and if that happens the community will simply move on and keep fighting. This community deserves to know so they can either have hope or move on!!