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Exploring the future of neurodegenerative disease research

Increasingly topical area discussed in enlightening LSX panel session

The recognition of ageing populations' impact on global health, and growing awareness of mental health issues, have ignited a renewed focus on neurodegenerative disease in recent years. Last month’s LSX Congress saw an expert panel, moderated by Evaluate Vantage’s Madeleine Armstrong, discuss the investment needed to overcome obstacles in neurodegenerative disease research. The session also explored the future of financing and commercialisation in this field, paving the way for innovative solutions.

Making up the panel were:

· Hugh Nuthall, Senior Director, Search & Evaluation Neuroscience, Eli Lilly

Understanding the challenges

Laurence Barker noted that while research in this area is in a better place than when he started, it still receives ‘only a fraction of capital’ compared to other therapeutic areas. This is perhaps unsurprising: due to notorious difficulties crossing the blood-brain-barrier, and, in line with recent headlines, identifying the right biomarkers, research in neuroscience is still considered high-risk. After a few years of scrutiny, the level of regulatory flexibility in CNS is still a constant point of interest: ‘I think that there is a willingness to allow biomarker endpoints to play a bigger role,’ said Hugh Nuthall.

‘One big, well-run study could be enough, whereas two, maybe three would have been standard in the past.’

Regulatory effects

Smaller companies are seeing this, too. David Reynolds notes that while flexibility from regulators is generally welcome, whether it will translate into benefits for patients remains, as yet, unproven. He mentioned his previous role as Chief Scientist at Alzheimer’s Research UK, where understanding patient perspectives was key. Linking research to patient benefits is crucial; Reynolds spoke of his hope that accelerated moves on biomarkers would ‘lead fairly quickly to follow-up studies that get much stronger data and help a whole sector move forward.’

While regulatory flexibility may appear to risk flooding the market with substandard drugs, in terms of outcomes Nuthall stated that products which have ‘bigger treatment effects, better safety profile, and more convenient dosing’, will make their way through eventually. Patient recruitment systems remain an area of concern; if a less effective drug becomes standard, it risks making clinical trials more expensive, while it can be difficult to encourage experimentation among sufferers of rarer diseases. Nonetheless, going through these processes is often necessary in the journey towards creating the most efficacious treatments.

Investment updates

Although we are far from fully understanding Alzheimer’s trials, Nuthall spoke of heavy investment in biomarkers which has helped to achieve an incredible amount of progress. While he also mentioned exciting Phase 3 developments in Alzheimer’s research, the picture is more varied across other neurodegenerative diseases. Huntington’s, for example, is difficult to treat – nonetheless, Reynolds noted that he is impressed by ‘the number of companies that are working in this space’, with recent research helping in understanding the disease’s natural history. ‘There’s a huge amount of interest in this area, which is borne out by the sheer number of biotechs and pharma playing this space and really working hard. And I’m sure some of those will be successful,’ he said.

For Jenny Barnett, meanwhile, Monument Therapeutics’ being a new, small company means there is extra pressure to show generalist investors ‘that neuroscience isn’t a dead end’. She knew the necessity of patient stratification from previous experience in pharma; having seen the success of stratification in oncology, Barnett is all the more convinced of the need to apply this to neuroscience.

‘It's just not how the brain works that you can give one drug and it's going to affect everyone in the same way.’

It's no secret that the biotech market is tricky at present. Venture capital funds have been taking longer than usual to decide where to invest and are pooling their money into fewer companies. However, the funds are there – and we expect investment to increase over the next months as the market stabilises.

What does the future hold?

Armstrong later queried Barnett’s assertion that neuroscience would be to this decade what oncology was to the last. We are at the beginning of that journey now: ‘It’s perhaps more accurate to say that neuroscience is where we were twenty years ago in oncology,’ Barnett said. There is a huge commercial opportunity to generate interest from seed funding, while the science has begun to mirror cancer treatment’s shift ‘to treat the genetic profile’ of a tumour, rather than targeting individual body parts. Still, while Barker notes that we may be ‘getting there’, there is still a long way to go.

‘I think we need we need some of these recent successes to now play through with more investment – more pharmas – to come back into the field.’


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