Biotechnology, Government, Health, Medical, Science, Society

Genomic medicine is being blocked by the NHS

NHS: GENOMIC MEDICINE

THIS MONTH the NHS will become the first health service in the world to offer whole genome sequencing to patients where clinically appropriate. Heralded by NHS leaders as “a new era of genomic health”, the goal is to use these data and new technologies to decode and treat previously intractable diseases, to move away from symptomatic treatments to cures and prevention.

The Prime Minister has said she wants the UK to lead the world in this new area of science – to continue a tradition of innovation in this country that will “extend horizons and transform lives”.

Theresa May’s ambition to lead the world in genomics and precision medicines is one that we should all want to support. Scientists and doctors know that pioneering precision medicines and their advances change lives, but they will also be aware of the challenges that must be overcome to realise its potential. This is not necessarily because the science is lacking, but because a fundamental shift in thinking is still needed by governments, regulators and policymakers in how they assess the value of this innovation.

Cystic fibrosis (CF) is an excellent example of this challenge. In 1989, when the cystic fibrosis gene was first identified, scientists did not know how mutations in the gene caused the condition. There was nothing to treat the underlying cause of the disease and people could only seek treatment for their symptoms.

After nearly 20 years of research and development by hundreds of scientists, and the design, synthesis and testing of more than 400,000 unique molecules, they have now done what was once thought impossible – discovered and brought to nearly half of all CF patients the first medicines to treat the underlying cause of this devastating disease. Today, multiple medicines approved by the EU and U.S. now exist, and there are more coming down the line. The ultimate goal is to cure CF once and for all.

For this remarkable cycle of innovation to be completed, Governments must now play their part, by providing patients with access to these medicines. Three years after approval of these medicines, this has still not happened because scientific innovation is outpacing the UK medicines evaluation system.

The evaluation criteria and processes used by the NHS and the National Institute for Health and Care Excellence (NICE) are currently preventing them from being made available to patients. Despite universal acceptance of the benefits that these medicines will bring, people in the UK have been waiting for access for more than 1,000 days, while thousands of people with CF in other countries in Europe and the US have been benefiting from them for years.

CF patients don’t really have the time to wait. Half of those with this cruel disease will die before they are 31. Science has delivered the breakthroughs, but the system is blocking access. The UK has the second largest number of CF patients in the world.

In 2016, the UK’s own chief medical officer recommended a fundamental shift in how new transformative medicines are developed and appraised for use in healthcare systems. The appraisal system in the UK needs to reflect that the genes and pathways underlying genetic diseases seldom respond to traditional pharmaceutical approaches, and so precision medicine requires risk-taking innovation.

The Life Sciences Industrial Strategy, a report made to the Government just last year, echoes many of these sentiments. It outlines the need for industry to take on bold, far-sighted ambitions in the life sciences with the intention of creating commercial success, underpinned by novel technology and higher-risk science. The strategy singles out a handful of successful biotech companies with highly innovative products. Yet, unlike in many other European countries, the NHS and NICE have not yet followed these recommendations and evolved their evaluation criteria for these types of transformative precision medicines.

The Government must surely need to act, not just for more than 10,000 people currently living with CF in the UK, but also for people suffering from many other kinds of genetic diseases.

Genomic medicine stands on the cusp of becoming an everyday reality. Those institutions at the cutting edge of gene therapy and gene editing need a system that is already thinking about the innovations of tomorrow. Such systems need to incentivise innovators to get medicines into the hands of patients as soon as possible.

Organisations involved in scientific advances will never give up on their ambition to cure serious diseases that today might still seem impossible to tackle. While they continue to deliver on the science, the UK Government must show its commitment to biomedical innovation if the genomic revolution is to be fully realised.

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Health, Medical, Research, Science

Lifestyle factors in keeping dementia at bay

MEDICAL RESEARCH

DOCTORS have identified seven lifestyle factors in middle age which have a significant influence on the risk of developing dementia in later life.

A major study suggests each of the seven elements – weight, diet, exercise, cholesterol, blood sugar, blood pressure and smoking – contributes to the chance of getting dementia.

Scientists at the University of Bordeaux tracked more than 6,600 French people from Bordeaux, Dijon and Montpellier.

The participants, who were at least 65 at the start of the study, were tested for the seven elements and then monitored for an average of eight and a half years.

For each of the seven tests that they “passed” as healthy, the risk of developing dementia over the following years went down by 10 per cent.

The scientists, whose findings were first reported and published in the JAMA medical journal, said their study demonstrates the link between cardiovascular health and the resilience of the brain.

“These findings may support the promotion of cardiovascular health to prevent risk factors associated with cognitive decline and dementia,” they wrote.

Experts in Britain say the findings from the research highlight the fact that the risk of dementia can be reduced with a healthy lifestyle.

A spokesperson for Alzheimer’s Research UK, said: “Although age is the largest risk factor for dementia, the condition is not an inevitable part of getting older and there are things we can all do to help reduce our risk.

“This large study of over 6,000 older people in France adds to a wealth of existing evidence indicating that what is good for the heart is also good for the brain.

“We know that the diseases that cause dementia can begin to develop in the brain up to 20 years before symptoms show so it’s never too early to take steps to reduce your risk.

“As well as these seven aspects of healthy living, drinking within recommended guidelines and staying mentally active and socially engaged have also been linked to better brain health in later life.”

Dr Doug Brown, chief policy officer at the Alzheimer’s Society, said: “Of all the diseases in the UK dementia is now the biggest killer, so exploring potential factors which could reduce the risk of developing this devastating condition is fundamental to beating it.

“Taking care of your cardiovascular health… may help slow cognitive decline and reduce your risk of getting dementia.

“Although this study stopped short of examining whether participants had healthy hearts in earlier life – which might have contributed to fewer of them getting dementia in later life – everyone should take steps from an early age such as eating a balanced diet, avoiding smoking and heavy drinking, and exercising regularly.”

The Seven Lifestyle Factors

. Weight – Have a body mass index (BMI) of less than 25

. Diet – Eat fish twice a week and fruits and vegetables at least three times a day

. Exercise – Walk more than two hours a day or take part in intensive sport more than twice a week

. Cholesterol – Levels need to be less than 200 mg/dL (milligrams per decilitre)

. Blood Sugar – Should be less than 100 mg/dL

. Blood Pressure – Should be less than 120/80 mmHg

. Smoking – Not smoking


MANY adults put off going to see their GP because they do not like being told what to do, research suggests.

Data from 4.300 Britons saw nearly one in five admit they had delayed making an appointment to avoid being lectured about lifestyle changes.

Experts say patients stay away for fear of giving away control – even when they are already showing worrying health symptoms – and warns this poses a considerable threat to public health.

The report, by experts at University College London, the University of Bedfordshire and thinktank 2020 Health, says many people also have a “fear of finding out” they are unhealthy and so avoid their doctor.

This can lead to major issues in the long term because minor problems missed at an early stage can escalate into far more severe issues. Many people – especially the rising number who know they do too little exercise or are overweight – do not report health complaints for fear of being lectured about their lifestyle.

GPs are encouraged by the NHS to ask patients about their weight and how much exercise they do, and are often given financial incentives to give weight-loss advice.

A spokesperson for 2020 Health, said: “It’s easy to think that consumers are wilfully refusing to change unhealthy habits, but the truth is, we are bombarded by conflicting health advice and mixed messages everywhere we turn.

“This is both confusing and dangerous as it can lead to important, evidence-based health information and guidance being lost or overlooked.

“Not only are lifestyle changes undermined, but the importance of getting specific symptoms checked out early are obscured.” Some 18 per cent of participants in the study – nearly one in five – said they had delayed an appointment because they were worried they might be pressured to change their lifestyle.

With 68 per cent of men and 58 per cent of women in Britain now overweight or obese, many may have reason to fear being lectured.

A quarter of participants said they had avoided a doctor because they did not want to be physically examined. A fifth said they were anxious about treatment that might be required, and another fifth were worried about the impact a diagnosis may have on family members.

The report, funded by pharmaceutical firm AbbVie, said patients who go to their doctor when they have a problem actually have a smaller impact on the NHS.

It said: “The model of the ‘good patient’ [is] one who adopts a healthier lifestyle, sees the doctor promptly if they notice what could be the early signs of illness and turns up to routine health checks and screening appointments.”

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Health, Medical, Research, Science

Could a new ‘universal vaccine’ stop all strains of flu virus?

MEDICAL RESEARCH

A NEW universal long-lasting vaccine could prevent the need for yearly flu jabs.

Currently, scientists have to predict every year what the new strain of flu will look like, but by the time the vaccine has been made, the strain of virus may already have mutated and changed.

Recently released figures reveal the vaccine given to ten million over-65s, children and at-risk adults last year offered little protection against the killer H3N2 strain, known as Aussie flu, which put unprecedented pressure on the NHS during the winter months.

Public Health officials have admitted the jab had “no significant effectiveness” in preventing people from being struck down – and was deemed only 15 per cent effective overall.

UK researchers are now working on a “universal” vaccine to protect against a number of strains.

The cells of the flu virus are like spherical cushions with lots of pins sticking out. Flu vaccines currently work by triggering an immune response to antigens – the heads of the pins – on the cell’s surface.

The immune system creates antibodies, which are then primed to attack and block the real flu virus when it comes along: the antibodies recognise the virus by its antigens.

The new vaccine, developed at the University of Oxford, protects the body against flu in a way that makes it more universal.

 

SCIENTISTS have found that while flu strains vary, all flu viruses contain epitopes (parts of the antigen to which antibodies attach), which vary much less than previously thought. Targeting these epitopes through vaccination would protect against all strains of the virus.

The new vaccine is designed to home in on these common epitopes and help the immune system create antibodies to fight them.

So far they’ve identified specific epitopes for two main types of flu – influenza A virus (subtypes H1 and H3) and influenza B. Researchers say a vaccine using this new approach could provide immunity without the need for yearly vaccinations, and could work against many types of flu even if the virus mutates.

Two or three injections would give long-term protection against different strains, they say.

Sunetra Gupta, a professor of theoretical epidemiology who led the research, says: “We believe our methods can be applied to produce vaccines against all subtypes of influenza, thereby providing the opportunity to develop not only a more effective vaccine against endemic influenza, with lower healthcare costs, but also better protection against potential influenza pandemics.

“The same strategy can also be used to produce vaccines for swine and avian influenza, which will have significant economic consequences, and the control of which will reduce the probability of new lineages emerging with pandemic potential.”

Vaccination is the most effective way to protect against the virus and is given annually in the UK to people at risk, including the over-65s, children aged two to nine, pregnant women, and people in long-stay residential care homes.

But the problem with existing vaccines is that the flu virus frequently mutates in two ways.

The first, known as antigenic drift, involves small changes in the genes of influenza viruses as the virus replicates.

These small genetic changes usually produce viruses that are closely related to one another, and share the same antigenic properties, so an immune system exposed to a similar virus will usually recognise it and respond.

But these small changes can accumulate over time and eventually the immune system may not recognise them and respond.

The less common route is antigenic shift – an abrupt, major change in the influenza A virus, and most people have little or no protection against the new virus.

 

TO ENSURE vaccines are available when needed, six months before the flu season, scientists try to predict the new strain. However, as the latest figures show, they don’t always get it right.

Commenting on the research, Professor Wendy Barclay, the Action Medical Research Chair in Virology at Imperial College London, says: “There are lots of different ideas about to make a universal flu vaccine and how universal it would actually be.

“This work from the Oxford group would mean we don’t have to update the flu vaccine yearly, but if a new pandemic came along, chances are this type of vaccine wouldn’t work against that.

“But it would mean we don’t have to chase after the virus as it constantly drifts and we try to keep up.”

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