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The search for treatments for Parkinson’s disease

The research requires resilience but pharma is urged to ‘keep innovating, keep trying’

The reactions to Pfizer dropping neuroscience research ranged from ‘disappointing’ to ‘demoralising’. The move was compounded by the company saying the decision was driven by science rather than cost, a shockwave signalling a forlorn future for the Parkinson’s disease community.

The underlying message was not that the multinational couldn’t afford a long, hard slog but that perhaps it could only see a journey without end. But the loss of a major player that had been backing new drug therapies and innovative monitoring technology has only served to redefine the Parkinson’s R&D landscape rather than devastate it.

Two years on, there is real conviction that transformative therapies, in the shape of disease-modifying drugs, gene therapy, growth factors, vaccines and repurposed drugs along with potential biomarkers offer a bright vision. The job of decoding Parkinson’s, which may have at least five sub-types, remains an intellectual and logistical challenge but the passion to provide a better future for ten million patients worldwide remains intense.

Dr Arthur Roach, director of research at the influential Parkinson’s UK charity, believes that genetic understanding is advancing at pace and that the collaborations between startups, academia, patient groups and big scale pharmaceutical companies provide a highfunctioning framework.

“My message to the pharmaceutical industry is to keep innovating and keep trying,” he said. “When I speak to people across the industry, it is clear they share our motivation and determination to bring genuine hope to patients.”

New mechanisms

The mood is anything but one of dismay and, although neuroscience is a field that requires resilience and the patience of a convention of saints, improved genetic understanding with fresh scientific intelligence is creating sharper targets and more clearly defined pathways.

“There is a great deal of positivity, particularly as we are getting original leads from genetic cases,” adds Dr Roach, who joined Parkinson’s UK in 2014 after academic research posts and a decade as senior director of neurodegenerative diseases research at Merck Serono where he served as its leader for in vitro pharmacology in neurology, including in multiple sclerosis, Parkinson’s and Alzheimer’s diseases. He was also president of the Geneva Pharma Network and founded the clinicalstage Swiss biotech Chord Therapeutics.

“We are discovering fundamentally new mechanisms with really good validation from human data and numerous companies are putting their muscle behind them, which is really exciting. We do not know the best programme in advance, but we do know there are really good ones out there.”

The number of people living with Parkinson’s is predicted to rise to more than 13 million by 2040, underscoring the need to halt or at least slow down its destructive impact on the central nervous system (CNS). In Europe, the burden is immense, with the European Parkinson’s Disease Association, which represents 45 member groups, estimating an annual cost of €13.9bn to care for the 1.2 million people living with the condition.

Snakes and ladders

Although Pfizer’s withdrawal was an initial body blow, it was not out of character as big pharma recalibrates to concentrate on core specialties while R&D dynamism is ignited by smaller biotechs, funded by venture capital, start-ups and academia. Pfizer itself established a venture capital organisation to fund early-stage projects. “CNS disease drug development is traditionally viewed as higher risk and more expensive,” added Dr Roach.” I do not have privileged internal information about Pfizer’s neurological programmes, but many big pharma firms are saying that ‘if you bring me a molecule with good phase 2 data, we would be very keen to license it’.

“Big pharma companies used to have their own research institutes that were very academic, along with departments doing relatively high-risk preclinical drug discovery but, with intensifying competition, they’re specialising more.

“What they do uniquely well are the later stages; the phase 3s, the approvals, the marketing and manufacturing. They do those things better than anyone else, so they are staying fully engaged in their core therapeutic areas and outside that are happy to in-license products for which there’s already phase 2 data.

“Pfizer is probably in that category, so its move was not totally deflating, particularly as, in virtually the same week, Takeda said it was getting into CNS and developing an antibody for Parkinson’s. It’s snakes and ladders.

“The world is not abandoning neuroscience. It is just recognising that it is tricky and difficult.”

The Critical Path for Parkinson’s – a consortium of ten pharmaceutical companies, academic institutions, charities, including Parkinson’s UK, and regulators – is continuing to smooth the path for smarter and faster clinical trials for a generation of disease-modifying drugs. This is vital as patient groups push for their use earlier in the disease journey.

“The old dogma was that these new treatments were used only in patients as a last resort but if safe, the aim has to be to use them earlier so there will be more benefits,” said Dr Roach. “We have to work with the FDA and EMA to find more appropriate ways to do tests to get these drugs approved.

“This could involve wearable data, which is a big area of excitement right now. The regulators haven’t yet gotten around to saying exactly what kind of results from wearable data they will consider, but you get the impression the FDA and EMA are open to hearing the evidence.”

The tightrope for Parkinson’s progress involves leaps of faith; the regulators have to balance flexible endpoints on existing, stringent standards while R&D has to juggle a series of diseasemodulating therapies rather than a chase for a blockbuster.

Improved symptom management

“There is a need to realise the great importance of symptomatic therapies and that great symptom management can achieve a lot,” Dr Roach said. “These drugs are effective and life expectancy is much higher than it was ten or 20 years ago. I think people living with Parkinson’s, and a lot of other conditions, will be very happy with two or more symptomatic treatments that improve their quality of life.”

The diversity of thinking coursing through the field is exemplified by a Glial Cell-Derived Neurotrophic Factor (GDNF) trial in Bristol, UK, which had clinicians collaborating with engineering company Renishaw to construct a device to deliver the therapy to precise areas of the brain in the hope of regenerating dying dopamine cells. The trial failed to meet its endpoints but did show promising results.

Dr Roach is encouraged by the high levels of innovation and commitment and the potential from genetic studies.

“People in the pharma industry share the same kind of motivation as research scientists and academia and the fantastic tools we have are the advances in biomedical science which are helping us solve really important issues; everyone wants to be a part of that,” he adds. “They want to apply their resources and knowledge to provide answers.

“Companies in turn are happy to receive help, support, guidance and ideas from anyone, including patient groups, which is a great attitude to adopt.

“I think we are getting better programmes of managing Parkinson’s and managing different sub-types at different stages with different interventions. It won’t be one thing that cures Parkinson’s but a number of things; a collection of drugs used in a personalised and flexible way that stop or slow its progression.

“I think the pieces are all there and we will look back at this time as the beginning of the time when we really started to slow progression to such an extent that people could have much better lives.”

For further information about Parkinson’s UK, visit www.parkinsons.org.uk

Danny Buckland is a journalist specialising in the healthcare industry

2nd July 2020

Danny Buckland is a journalist specialising in the healthcare industry

2nd July 2020

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