5 February 2025 Research
Over the last few weeks, there has been discussion in the MND community following media coverage of research into a drug, or compound (NVG645), called ellorarxine. This coverage has suggested that this is an exciting development in MND research. Following this, some people in the community have asked whether we should be funding further research into it or be campaigning to get the Government to do so. So we thought it would be helpful to give an update.
We first published news about the early stage testing of this compound in the laboratory on 20 September 2024. The research published so far has shown that in healthy mice, and mouse and rat cells in a dish, the compound had an effect that led researchers to hope that, if the drug were to have a positive effect in motor neurone disease in humans, it might be a potential treatment for MND.
We really hope this turns out to be the case. As a charity, we exist to improve the lives of people affected by MND, so any discovery of an effective treatment would be incredibly exciting.
Ellorarxine is a newly created compound and we don’t know if it is safe for humans to take. If it is, we don’t know whether it will have an effect on motor neurones in humans. In science, there are many, many examples, of drugs that seemed promising in the laboratory but then did not then have a beneficial effect in humans.
Ellorarxine has shown potential effects that will require further investigation. But from the data published so far, it’s one of the many drugs or compounds in the drugs pipeline worldwide, rather than being an exceptionally exciting one. Indeed, the failure of many promising drugs in the past led the MND community, working with the government-funded Medicines Discovery Catapult, to generate new guidance to improve standards of preclinical testing.
To respond to the question about asking the government to fund further research into this particular compound - we are no stranger to campaigning for government investment in MND research. Three years ago, we secured £50m of funding over five years through our collaborative campaign with people with MND and other MND charities. This investment is now starting to generate new research, including the recent launch of a new rapid drug prioritisation trial for MND in the UK. But to campaign for government funding, we have to be confident any money would be targeted at the thing offering the highest possible chance of benefit to people with MND. From the information and published data we’ve seen so far, it’s still very early days for ellorarxine. And so we’re not currently planning to make the case to government for extra funding for this.
This judgement is based only on the publicly available data. It may be that Nevrargenics, the private, shareholder-owned company behind ellorarxine, has more data. We would welcome seeing that. If the company showed compelling evidence that gave us confidence there was a high chance of this becoming a treatment we would, of course, review our position.
We are the UK’s largest charitable funder of MND research and, as with any organisation carrying out research, Nevrargenics is welcome to submit a formal application for funding to us. Every application we receive goes through robust review processes, involving a diverse panel of multidisciplinary experts, to ensure we are spending the money our supporters have generously raised to fund the very best and most promising research.
We hope that sets out our position on ellorarxine. Given the interest in this within the community, we will share future developments when we have them.