Chief Executive update: Access to emerging treatments for motor neurone disease (MND)

We want to bring you up to date on access to emerging treatments as we appreciate there is a level of anger and frustration among the MND community and we are committed to sharing the progress being made with you on a more frequent basis.  

We know how important this is to our community and understand the frustrations felt by many about the length and complexity of the processes behind accessing new and emerging treatments. We are currently managing the response to a number of potential treatments with a view to getting proven treatments to patients via the fastest means possible.  

This statement focuses on low dose interleukin-2.  

As you may already be aware, in December 2022, the preliminary findings from the MIROCALS study were announced, which trialled the use of low dose interleukin-2 (IL-2) for ALS. These early findings were encouraging. However, further research and an analysis of the data was still needed.   

Since then, a French company, called ILTOO Pharma, has bought the commercial rights to low dose IL2. ILTOO is currently awaiting the release of the data. While the MND Association has limited influence or control over this process, we are doing what we can to press for the final set of data to be published as quickly as possible.   

A drug called Proleukin was used as the low dose interleukin-2 in the trial for ALS, and as you may know is already available in a high dose for treating types of cancer. The effects on the body of high doses of the drug are very different from those of low doses and indeed high doses could be detrimental to people with MND. For these reasons, the drug is considered ‘unlicensed’ and must be evaluated and approved by the regulatory bodies.

At the moment we are unable to influence access to low dose Proleukin for patients with ALS as the data needs to be presented in the form of a Clinical Study Report (or Regulatory Package) before any regulatory review begins. The data required for the Clinical Study Report has to be rigorously analysed to determine if the drug is safe and effective.  

This process is, of course, frustratingly slow and upsetting.    

An alternative option, and the one that we are mainly focussed on now, is to pursue early access routes that can be used for unlicensed drugs in exceptional circumstances.   

We are working hard to identify the best and quickest routes to gain early access to low dose Proleukin so that we are prepared for the data publication, which we hope will be positive.  We are also preparing the ground in other areas including contacting clinical directors of the 22 MND Care Centres around the UK to see what we can do to support them once the data is published as a research paper and available to the medical community across the world.

We are looking at NHS England’s Drug Repurposing Scheme, provision of the drug on a named-patient basis, and what is called the ‘NHS Specials’ Route.  These are all areas in healthcare where we are having conversations to ensure we can move forward when we have the data. We are also talking with Government contacts to ensure this challenge is front and centre of their minds.

Please note that while we will continue to look at the longer-term regulatory process, we are focussing most of our efforts on early access to Proleukin. This does of course depend on the final data being positive. The trial investigators are working hard to complete their analysis.

We are producing an infographic explaining the process that all new drugs in the UK need to go through before being approved and being made available on the NHS. We will share this with you very soon. We will also be providing an update on our website on a weekly basis.  

You can read further information about the MIROCALS trial and interleukin-2 here. 

Tanya Curry
Chief Executive