Professor Andrew Lees: Parkinson’s needs tailored disease management
PD in Practice
sponsored by EVER Neuro Pharma
Author: Peter MansellPublished: 9 April 2015
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Parkinson’s is a chronic and progressive disease that makes strenuous demands on people with Parkinson’s (PwPs), their families, carers and health systems. It also manifests through a variety of symptoms, both physical and psychological, that change over time. These changes can be both unpredictable and life-altering. Here, Parkinson’s Life speaks to Professor Andrew Lees (pictured above, left), one of the foremost authorities on Parkinson’s, about the vital importance of tailored disease management in Parkinson’s.
Effective diagnosis and measurement of Parkinson’s are difficult enough. Therapeutic intervention, particularly in the advanced stages of the disease, then has to be carefully gauged to achieve optimal trade-off between symptom management and sometimes problematic side-effects.
Patient-centred care is something of a political mantra in 21st century medicine. In Parkinson’s disease, it really is ground zero. The condition itself is multi-faceted, calling for a range of medical and societal inputs. It is further complicated by the heterogeneity of responses to diagnosis and treatment, and by the different weight PwPs and health systems accord to symptoms as various as constipation, depression or cognitive impairment.
This may appear to slot usefully under the catch-all banner of ‘personalised medicine’, but for Andrew Lees, Professor of Neurology at the UK’s National Hospital for Neurology and Neurosurgery and at University College London, the term is a distraction. Good doctors have always practised personalised medicine, he insists. It is just that a medical culture increasingly hidebound by guidelines has “hijacked evidence-based medicine”.
As Professor Lees notes, treatment decisions in Parkinson’s are inevitably personalised by the variable and complex nature of the disease – and “you can’t reduce 30 years of experience to an algorithm.”
Professor Lees has spent some 45 years working at the coal face of neurological disease and is the world’s most cited researcher on Parkinson’s.
Among other achievements, he was responsible for the introduction of apomorphine as a treatment for advanced complications of Parkinson’s disease, including L-dopa-induced ‘off’ periods and dyskinesias.
Optimism and realism
Encouragingly for someone with his depth of experience in an often intractable field of medicine, Professor Lees remains both realistic and optimistic.
On the realistic side of the ledger sit the pressing need for a “game-changer” in drug therapy and the “continual frustration” of trying to address PwPs’ non-pharmaceutical needs, such as physical therapy, within the sometimes unyielding machinery of a national health system.
On the optimistic side, the management of Parkinson’s disease is already in a “much better place” than other neurological conditions such as Alzheimer’s, with treatments that genuinely improve quality of life (QoL), Professor Lees points out.
That said, much remains to be done in optimising Parkinson’s management to the benefit not just of PwPs and carers, but of health systems that will take on the growing logistical and budgetary challenges of a fast-expanding Parkinson’s population.
Some, but not all, of this relates to societal ageing. As Professor Lees observes, 15-20% of PwPs will have the disease before they are 45 years old. In any case, the implications for healthcare provision, support services and societal costs, both financial and otherwise, are daunting.
“Good doctors have always practised personalised medicine”
With no new drug treatments for Parkinson’s licensed for seven years now – the largest gap since the 1960s – most existing therapies are genericised. So while a breakthrough treatment is enormously desirable, it is also going to come at a price.
Moreover, if disease-modifying treatment is the holy grail of Parkinson’s, existing therapeutic options for some of the symptoms associated with the disease, such as problems with balance or cognitive impairment in the elderly, remain sub-par.
Recognising Parkinson’s for what it is
Not that PwPs always have access to even effective symptomatic treatment. The challenges of tailoring therapy to individual patient characteristics start with ensuring that the condition is recognised for what it is.
Instead, Professor Lees says, healthcare professionals and fundholders tend to “lump together” Parkinson’s with other neurological diseases such as Alzheimer’s.
In the UK at least, politics and money have exacerbated a ‘postcode lottery’ in the availability of Parkinson’s-disease treatment, with decisions taken on the basis of “science not humanity.”
Diagnosis is also problematic, especially in the early stages of the disease. According to Professor Lees, techniques have improved but specialists still make the wrong diagnosis in 10-15% of cases.
Even if pointers such as reduced dopamine production or lost sense of smell are judged sufficient evidence of early Parkinson’s, it is arguable whether pharmacological intervention at this stage – in the absence of disease-modifying therapy – will really improve quality of life, Professor Lees adds.
What early diagnosis may offer PwPs is some clarity over the future –although many will remain in denial over their diagnosis for up to two years. Fear of treatment is another barrier to intervention.
If identifying Parkinson’s – and doing so at the right time for PwPs and health systems – is complicated enough, measuring disease progression accurately and objectively in a condition clouded by co-morbidities is a significant barrier both to effective disease management and effective clinical trials of potential new therapies.
While new approaches such as blood biomarkers, analysis of spinal fluids or brain imaging are coming into play, neurologists remain largely reliant on “semi-quantitative” ratings scales.“We’ve been using self-scoring diaries since the 1970s,” says Lees.
The emerging field of objective measurement in Parkinson’s should address some of these deficiencies, enabling pharmaceutical companies to target drug candidates more productively in clinical development and healthcare professionals to shape real-world therapy more closely to patient responses, enhancing both treatment effects and cost-effectiveness.
All the same, Professor Lees cautions, even biomarkers “don’t measure how people feel.” Historically there has been a “preoccupation” with impaired motor function in Parkinson’s, while other symptoms are relatively neglected.
Ensuring Parkinson’s management is both as individualised and as comprehensive as it can be within the current state of the art means first acknowledging the complexity of QoL considerations in the disease.
Quality of life: it’s not that simple
Symptoms that may seem peripheral from the outside can be hugely important to some patients within the broader framework of coping with Parkinson’s on a day-to-day basis.
These may be non-motor problems such as pain, depression or constipation. As Professor Lees notes, not only are co-morbidities inadequately addressed in the more controlled and homogenised environment of clinical trials, they may also fall outside the scope of official guidelines.
Discussions on updating Parkinson’s disease guidelines drawn up by England’s National Institute for Health and Care Excellence (NICE), for example, have excluded non-motor symptoms unless these are shown to manifest otherwise than in a non-Parkinson’s context, Professor Lees (pictured, right) explains.
Professor Andrew Lees (right)
The problem here is that PwPs may present with “hundreds” of symptoms, some but not all of them associated with Parkinson’s, and others a natural consequence of ageing. Sifting out what is and is not Parkinson’s-related is part of what makes the disease such a challenge for everyone involved.
It also underlines how crucial a holistic approach to care is, one that can be applied consistently throughout PwPs’ many points of contact with health systems and carers.
Parkinson’s immediately draws people into its orbit – the rule of thumb being ‘one PwP, five affected.’ Yet the concept of joined-up care for the disease is “broken”, Professor Lees says.
PwPs’ experience of health systems is “really variable and can be terrible”, he comments. There have been improvements: the NICE (National Institute for Health and Care Excellence) guidelines, for example, already state that all PwPs should see a specialist with an interest in movement disorders.
Finding a specialist may not be so easy, though. The 600 or so neurologists in the UK compare poorly with Germany, where there are as many as 3,000, Professor Lees observes.
Shuttled around the system
What happens next can also be erratic. Too often, PwPs report seeing a consultant once and once only, while many GPs will say, “you’re with the hospital now.”
Being shuttled around the system is what pushes PwPs into the private-health sector – only to find that insurance companies are unlikely to cover Parkinson’s.
Many of the gaps in addressing Parkinson’s are now being filled by specialist nurses. This is an area of provision in which the UK runs ahead of its European counterparts. Even so, there can be problems with securing contracts for Parkinson’s nurses in the National Health Service, Professor Lees says.
Some pharmaceutical companies focused on Parkinson’s disease are already funding specialist nurses themselves. In future, industry may become more involved in knitting together disparate elements of the health system into an integrated network spanning primary and secondary care for Parkinson’s.
“The problem here is that PwPs may present with hundreds of symptoms. Sifting out what is and is not Parkinson’s-related is part of what makes the disease such a challenge for everyone involved”
With “big changes” in healthcare delivery expected over the next 10 years, Professor Lees envisages companies emerging as “healthcare providers” to offer a package of tailored services addressing the many needs of PwPs: anything from drug therapy to diet, exercise, physiotherapy, education, speech therapy or emotional support.
Technology should accelerate this process, with innovations in drug-delivery systems and remote, real-time monitoring shifting the focus of care from the clinic to the home.
In parallel, PwPs themselves, and the associations that represent them, are taking the initiative to get educated and play a more active role in reshaping the management of Parkinson’s.
One vitally important consideration in addressing the complex of needs in Parkinson’s is achieving stability. The symptoms of the disease are ‘predictably unpredictable’, while pharmacological intervention is complicated by side-effects such as compulsive behaviour, depression, nausea or confusion.
Gold-standard therapies like levodopa carry a sting in the tail, as prolonged exposure commonly leads to ‘wearing-off’ of drug effects, with associated dyskinesia or loss of motor control.
Something that may help to keep PwPs on an even keel is the evolution of continuous drug-delivery systems that can maintain dopaminergic stimulation and potentially control motor fluctuations.
The best way to achieve this remains open to debate, with some conflicting evidence from clinical trials.
In general, though, a “steady dribble” of dopamine into the brain’s basal ganglia, whether through emerging pump technology or, in the future, stem-cell implantation, should help to avoid complications further down the line, Professor Lees believes.
Towards a more systematic approach
The effective diagnosis and management of Parkinson’s disease calls for a more systematic approach that can align products, services, research, development and public policy in delivering seamless, integrated and individualised care across a broad spectrum of PwP needs.
As Professor Lees stresses, though, there also has to be leeway for personal intuition and insight. It takes 10 years to become a neurologist “and you do it at the bedside”, he comments. “Hunches” are intrinsic to good medicine, even in the face of new technology.
“Medicine’s not really a science,” Professor Lees comments. “Science is part of it, but it’s not like physics or computer technology. There are too many grey areas to get anywhere near that. Human beings are different.”
This article was sponsored by EVER Neuro Pharma. The information in this article is given for information purposes only and does not represent an endorsement by Parkinson’s Europe of any particular treatments, products or companies. This article is not a substitute for advice from your doctor, pharmacist or other healthcare professional. Parkinson’s Life makes no representations or warranties of any kind, express or implied, about the completeness or accuracy of information provided.
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EVER Neuro Pharma is a fully integrated niche pharmaceutical company based in Unterach, Austria and working in the field of neurological diseases and critical care. The company was founded in 1934 and now has a presence in more than 50 countries worldwide.
EVER Neuro addresses the clinical needs of Parkinson’s patients through tailored solutions, supplementing drugs and devices with support services such as daily disposables, specialist nurses and patient helplines.
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