![Tris Dyson, founder of Challenge Works](/https/www.telegraph.co.uk/content/dam/global-health/2023/07/25/TELEMMGLPICT000343819198_16902895347880_trans_NvBQzQNjv4BqRo0U4xU-30oDveS4pXV-Vv4Xpit_DMGvdp2n7FDd82k.jpeg?imwidth=680)
![Tris Dyson, founder of Challenge Works](/https/www.telegraph.co.uk/content/dam/global-health/2023/07/25/TELEMMGLPICT000343819198_16902895347880_trans_NvBQzQNjv4BqRo0U4xU-30oDveS4pXV-Vv4Xpit_DMGvdp2n7FDd82k.jpeg?imwidth=680)
I knew something was wrong. I was getting slower, I felt tired more quickly and then one day I couldn’t give a thumbs up.
Maybe it was lack of sleep – I’d just had a baby daughter. Maybe it was stress. Maybe it was just middle age rearing its head.
In March came the diagnosis that has upended my reality: motor neurone disease (MND).
When you’re a new dad in your 40s, it’s the absolute last thing you expect to hear. Five months on, I still haven’t fully come to terms with the news.
The signals from my brain are progressively unable to reach my muscles. In the next couple of years, I will lose the ability to walk, talk, eat, swallow and look after myself. Finally, the muscles in my chest will stop functioning and I will suffocate. There is no cure. The only treatment available may extend my life by a couple months.
Knowing I will miss being a part of my daughter’s life is the most gut-wrenching aspect, ahead of reconciling with the burden I am placing on my partner, now and when I’m gone.
A rude but enlightening education
MND is uncommon, but not rare. Up to three people in every 1000 will develop MND in their lifetime. However, compared to many other conditions, there is a much smaller active patient community – a result of the short lifespan that comes after diagnosis.
Standard diagnosis is shockingly basic – a doctor observed how much I twitched, prodded me to test reflexes and discounted other conditions with blood tests. This means diagnosis is mostly only possible when MND is about 60 per cent advanced.
As a result, we usually have two or three years before we die.
For 10 years, I’ve run Challenge Works – part of the innovation foundation Nesta. We design challenge prizes to fast-track innovations that solve some of the world’s biggest problems. I have been astounded by the lack of support for MND innovation when so many families are impacted.
That’s not to say nothing is happening. The last few months have been a rude but enlightening education.
I’ve been exploring how a challenge prize for MND could make a difference to developing effective treatments. I’ve met with many organisations including the new MND Research Institute, MND Association and My Name’5 Doddie Foundation. They are run by extraordinary people, all of whom are striving for new drugs and treatments.
They are constrained by a limited – though growing – pot of funding in a world where new drugs require hundreds of millions of pounds to be developed.
I can tell you though, when you’re terminally ill, progress on new drugs generates only so much optimism when you know they won’t be available for a decade – far too late for me.
Hope in existing drugs?
Maybe we don’t just need new drugs – maybe some already exist, but aren’t being used to treat MND.
Taking a drug has multiple impacts on the body. To bring a new drug to market as quickly as possible, a drug company usually focuses on one beneficial impact and without noticing or exploring other potentially advantageous effects.
For example, aspirin was for nearly a century prescribed for pain relief, before being repositioned in the 1980s to also prevent cardiovascular conditions. It’s now thought it could lower the risk of cancer too.
And yet a side effect for treating one disease could have a positive impact for another. Interleukin 2 (IL-2) is an immunotherapy drug for kidney cancer. At high doses it impacts cell growth and stimulates the immune system to produce regulatory T cells that attack tumours.
T cells can also reduce inflammation at low doses – particularly in the brain and spinal cord – which is thought to contribute to the progression of MND.
In Phase 2 trials at very low doses, it has been reported to decrease the risk of death after 21 months for a significant population of people with MND by 40 per cent. If it gives me nearly two more years with my family, I’ll take those chances. And yet, despite knowing it is safe and tolerable for people to take, I cannot find someone to prescribe the drug.
Clinical trials follow a necessarily strict and arduous regulatory process, with results being peer reviewed. IL-2 is licensed to treat kidney cancer, but is not yet licensed for MND. Therefore it cannot be used to treat me, regardless of how informed and willing I am to try it in light of the Phase 2 trial results. IL-2 is not the only drug with the potential to offer hope to people if it was repurposed for MND.
I could rail against my doctors and hospital, but they are working within a system that makes them solely accountable and leaves them exposed to litigation (clinical negligence claims cost the NHS more than £13 billion in 2021). We need to re-engineer the system whereby terminally ill patients and their families can take full accountability when the stakes are so high.
When the clock is ticking, we need to get these drugs out of small-scale trials and to the people that could benefit from them – especially when we are informed and willing.
When you are diagnosed with MND, you don’t have enough days left to risk taking placebos or waiting for an existing drug to one day make it to the front of the approval queue. We don’t have time – and nor do I.
- Tris Dyson is the founder of Challenge Works
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