MIROCALS: our latest update

We’re now a month on from our previous update about the MIROCALS trial testing Interleukin-2 as a treatment for motor neurone disease. We remain in regular contact with other consortium members but, disappointingly, there is not yet any confirmation of a definitive publication date. 

However, we aren’t standing still. We have continued to prepare for all potential outcomes of the trial and continue with our commitment to work collaboratively with other MND charities to pursue every avenue available to us to ensure people with MND have equitable access to proven treatments as quickly as possible. Depending on the outcome of the trial, that may include us working to influence the regulatory process, pushing for access for people with MND through the NHS Repurposing and NHS Specials routes, or ensuring people in the UK can take part in any further trial that may be deemed necessary.

An important part of that preparation has been maintaining relationships with people who are key decision makers in organisations, including the regulatory bodies, so we can have an open and helpful dialogue going forward. Like all of us, they continue to wait for the publication of the full results before being able to take any further steps.
In the meantime, we have received a number of questions from people in the MND community about our part in the MIROCALS Consortium and the role we have to play now. We have addressed most of these in the past and hope it’s helpful to pull the responses together here so there is one point of reference.

Q: Why did you endorse the choice of a small pharmaceutical company when a far larger company, with the global rights to market the trial drug, already existed.
As we have made clear previously, we would be in breach of the confidentiality agreement we signed as a member of the Consortium if we were to share details of the discussions and voting around the choice of ILTOO Pharma. We simply are not permitted to do that. 
We have previously shared that we voted for ILTOO Pharma because we believed it offered people with MND the best chance of access to the drug as quickly as possible, in the event of it being shown to be effective. 

 
Q. Will you admit you have made an error on the commercial arrangements? If not, please tell us why you made the right choice?
Nothing has happened since to make us believe we made the wrong decision. As above, we voted in the way we did believing ILTOO Pharma was the right choice of pharmaceutical partner, given the information we had at the time. 
While we were involved in the vote, as shared previously, we had a very limited role in the Consortium. It wasn’t within our remit to be involved in discussions and decisions around the commercial arrangements put in place following that vote.

Q. Why do you continue to highlight the bio similar version of low dose Interleukin-2 to the exclusion of the trial drug. Proleukin, readily available on NHS pharmacy shelves? Why do you not support access to this drug?
As we have always said, if the trial data shows this is an effective treatment, we are committed to doing everything in our power to ensure patients are able to access the drug as soon as possible. We will support the pursuit of every viable route to ensure that happens.  This includes the regulatory process for ILT-101 (ILTOO Pharma’s version of the drug) and the NHS Specials and Repurposing routes for Proleukin (the drug used in the trial). 

Q. Why do you continue to push for the fully regulated access route, rather than routes that could be explored in the interim, even though they may prove difficult to navigate i.e. NHS specials and medicines repurposing?
As above, we are preparing for and have been developing relevant and useful relationships and partnerships for all possible routes, including NHS Specials and NHS Repurposing. The suggestion that we don’t see these as important potential routes is simply incorrect. For all potential routes, we are clearly being told we must have the data to support the process – we simply cannot progress now without it. 

Q. Why do you continue to hide behind confidentiality and non disclosure agreements when it is a publicly and charity funded trial?
We are not hiding – we are abiding by a contractual obligation. Confidentiality agreements are standard practice when entering into a contract or partnership like this one where we are a member of a Consortium. All charities, and indeed businesses and organisations, have confidentiality agreements written into a wide range of contracts they hold. As an Association, we certainly have them in a number of contracts across all areas of our work. 
To breach a confidentiality agreement would leave us open to legal challenge. In this instance, that could come from other Consortium members, the trial sponsor, the European Commission, and any of the industry partners involved in the trial. This would mean us having to direct a large amount of charity funds and resources to managing a costly and lengthy legal situation, taking money away from further research and support. It would also risk damaging the trust other organisations have in us as a reputable charity, making them less likely to enter into partnerships with us in the future. 
We all believe that breaching this confidentiality agreement would be going against the best interests of the MND community.

Q. Why will you and other members of the MIROCALS Consortium not approach the current worldwide rights holder to Proleukin, Iovance, as has been suggested?
We have previously met with Iovance and remain open to meeting with them and any other individuals, organisations or bodies which can facilitate access to proven treatments. However, once again we do need to stress, nothing can happen without the trial data.

Next steps
We have taken the decision to respond to the questions that we know have been circulating both on and offline and in private correspondence, even though we have previously provided comprehensive information about our role in the Consortium, ways of working and our preparation for the results.
We completely understand and share our community’s deep frustration over the length of time it has taken for the final results of the MIROCALS trial to be published, it is not acceptable on any level. But we have been open and transparent throughout, and so it is disappointing for these questions to be circulated in a way which suggests we have something to hide. We don’t. 
Most of the information above we have already shared publicly, and it is difficult to imagine any charity like ours would consider breaking a commercial confidentiality agreement in the way that the wording of these questions implies that we should.
As we have done throughout, we will continue to publish updates when we have new information to share. Our work on our mission and purpose for the whole MND community is of paramount importance, and we must continue to focus on and deliver that for everybody living with and affected by MND.

Tanya Curry
Chief Executive