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Currently there’s no cure for rare types of cystic fibrosis, but researchers are making significant advances

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Current treatments for cystic fibrosis are not suitable for all patients. The lack of treatment options is distressing for people suffering from a rare type of this degenerative and life-threatening disease. But researchers are making major advances. 

A decade ago, few cystic fibrosis patients lived beyond their teens. Thanks to a breakthrough in treatment options, for most patients with access to modern medicines, cystic fibrosis (CF) is no longer the catastrophic disease it once was. However, for 15% of people with CF, the cellular defect that causes their disease remains untreatable. For these patients, drugs are available to treat some of the symptoms of CF, but the condition continues to wreak havoc with their organs, resulting in premature death.

An EU-funded research project HIT-CF aims to change this by improving both the quality of life and the disease prognosis for people with ultra-rare varieties of CF. In Europe, there are an estimated 5,250 people who fall into this category.

The project, launched in January 2018, brings together researchers, doctors, pharmaceutical companies and patient representatives, with the aim of developing drugs and drug combinations that are matched with a high degree of precision to a patient, regardless of the rarity of their form of the disease. Such personalised medicine is possible thanks to a new approach to drug testing involving the creation of mini-organs in the lab using a patient’s own stem cells. These ‘organoids’ are genetic replicas of organs found inside the patient’s body and can be used to test how responsive a person’s cells are to specific pharmaceutical compounds.

‘We’re effectively shifting therapeutic trials from patients to the laboratory,’ explained Kors van der Ent, professor in paediatric pulmonology at the University Medical Centre, Utrecht in the Netherlands, and coordinator of the multi-disciplinary HIT-CF project.

To date, scientists involved in the project have grown organoids from 500 European patients with ultra-rare forms of CF. Ultra-rare can mean that just one or two people worldwide share the same form of the disease.

Describing his team’s work with organoids, Professor van der Ent said: ‘We’ve asked pharmaceutical companies to hand over drugs from their development pipelines so we can test these compounds against the organoids. These drug candidates target the basic protein defect involved in cystic fibrosis.

‘What is special about this work is that it means we can create highly personalised treatments for patients with rare mutations. What’s also special is that we can mix and match compounds from different companies to see if patients are responsive to a certain combination of drugs.’

From April, the project’s clinicians will start testing compounds that have proven to be effective on organoids on real-life patients. ‘We expect these patients to respond well,’ said Professor van der Ent. ‘We hope that within five years, these patients will have new drugs.’

Targeting the cystic fibrosis gene

One in 35 people carries the faulty gene that causes CF – usually without knowing. Two people carrying the faulty gene have a 25% chance of having a child born with the disease. Without modern treatment, most people born with CF do not live long beyond their thirties.

Until recently, the only way to treat CF was with antibiotics to fight infection, steroids to reduce inflammation, physiotherapy to clear airways, exercise, nutrition and transplants (of the lungs, liver and sometimes other organs). Although the disease still remains incurable, since 2012, a new class of drugs called modulators has transformed treatment for many.

CFTR modulators target specific defects in the CFTR protein, thereby restoring healthy function of the protein so that chloride (which is present in salt) can flow across the cell surface. To date, four such modulators have reached the market, and these drugs are both transforming the quality of patients’ lives and lengthening their lifespan substantially.

‘Thanks to these drugs, in some patients there’s a lung-function improvement of 30-40% and life expectancy can increase from the age of 30-40 to 60-80,’ said Professor van der Ent. ‘In other words, there can be a normal life expectancy.’

One significant downside of CFTR modulators is their price: treatment costs up to €200,000 per patient per year. As a result, only patients in countries with a well-funded health service can access medication. Meanwhile, many patients in Eastern Europe, along with other less developed parts of the world, are missing out.

Another drawback – and one that HIT-CF aims to address – is that CFTR modulators are only being clinically tested in patients with well-described, common mutations of CF. There are up to 2,000 genetic mutations that lead to CF, but just 120 of these are responsible for 80-85% of disease occurrence. It is patients with these common forms of the condition who are able to benefit from the CFTR modulators currently on the market.

So why are patients with rare mutations being left behind? The high cost of clinical trials means it simply does not make commercial sense for drug companies to focus their efforts on this sub-group of CF patients.

Step in organoid technology

Scientists involved in the HIT-CF project are taking tissue samples from the rectum of patients with rare forms of CF, isolating stem cells, and growing these to form mini-intestines.

There are two major advantages of using organoids to screen potential drugs: there are no safety considerations for the patient, and the screening process is highly efficient (any number of compounds from a library of potential drug candidates can be thrown at an organoid, and at speed). As a result, the potential cost savings are vast. For participants of the HIT-CF project, this is great news.

‘This study is giving people who have been excluded from clinical studies the chance to be recruited for a study and to find medicines that will tackle the causes of their disease,’ said Dr Elise Lammertyn, head of research at the European federation of national CF patient organisations Cystic Fibrosis Europe, a partner in the HIT-CF project.

‘There are quite a few (conventional) clinical trials going on in Europe for cystic fibrosis, but most of these are only open to those with the most common mutations of the disease, and the 10-15% of people with ultra-rare mutations are left out in the cold. This new study is about personalised medicine at its most innovative.’

Universal access to treatment

Prof. van der Ent, in partnership with other scientists involved in CF research, is set to launch Fair Therapeutics – a company that will set out to use organoid technology to bring CF drugs to market for patients with both common and rare mutations, at affordable prices.

‘In a sense we’ll be competitors to big pharma but actually we will all be working towards the same goal of reaching all CF patients,’ said Professor van der Ent.

First, however, the project scientists must acquire permission from the European Medicines Agency (EMA) to approve organoid testing so it can be used beyond the current study. ‘It will be very helpful to have a test in the lab that can be used in conjunction with less lengthy clinical trials to prove the effectiveness of drugs in small groups of people,’ said Professor van der Ent. ‘It will highly speed up the pipeline of new drugs for all kinds of diseases, not just CF. It could even be used as a predictive tool for cancer treatment: you do a biopsy of a tumour, add chemotherapy and other drugs to the organoid, and then use the most sensitive treatment on the tumour.’ 

The research in this article was funded by the EU. This article was originally published in Horizon, the EU Research and Innovation Magazine.

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Health & Wellness

World’s richest countries damaging child health worldwide

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Over-consumption in the world’s richest countries is creating unhealthy, dangerous, and toxic conditions for children globally, according to a new report published on Tuesday by the UN Children’s Fund (UNICEF).

“Not only are the majority of rich countries failing to provide healthy environments for children within their borders, they are also contributing to the destruction of children’s environments in other parts of the world,” said Gunilla Olsson, Director of the UNICEF Office of Research – Innocenti.

Urgent policy shift

The latest Innocenti Report Card 17: Places and Spaces compares how 39 countries in the Organization for Economic Co-operation and Development (OECD) and European Union (EU) impact children’s environments.

Indicators include exposure to harmful pollutants, such as toxic air, pesticides, damp and lead; access to light, green spaces and safe roads; and countries’ contributions to the climate crisis, resource consumption, and e-waste dumping.

The report states that if the entire world consumed resources at the rate of OECD and EU countries, the equivalent of 3.3 earths would be needed to keep up with consumption levels.

If it were at the rate at which people in Canada, Luxembourg and the United States do, at least five earths would be needed, according to the report.

Not in your own backyard

While Spain, Ireland and Portugal feature at the overall top of the list, all OECD and EU countries are failing to provide healthy environments for all children across all indicators.

Based on CO2 emissions, e-waste and overall resource consumption per capita, Australia, Belgium, Canada and the United States are among other wealthy countries that rank low on creating a healthy environment for children within and beyond their borders.  

Meanwhile, Finland, Iceland and Norway are among those that provide healthier environments for their country’s children but disproportionately contribute to destroying the global environment.

“In some cases we are seeing countries providing relatively healthy environments for children at home while being among the top contributors to pollutants that are destroying children’s environments abroad,” attested Gunilla Olsson, Director of UNICEF Office of Research

In contrast, the least wealthy OECD and EU countries in Latin America and Europe, have a much lower impact on the wider world.

Harmful exposures

Over 20 million children in this group, have elevated levels of lead – one of the most dangerous environmental toxic substances – in their blood.

In Iceland, Latvia, Portugal and the United Kingdom, one in five children is exposed to damp and mould at home; while in Cyprus, Hungary and Turkey, that number rises to more than one in four.

Many children are breathing toxic air both in and outside of their homes.

More than one in 12 children in Belgium, Czech Republic, Israel and Poland and are exposed to high pesticide pollution, which has been linked with cancer – including childhood leukaemia – and can harm vital body systems.

We owe it to ourselves and to future generations to create better places and spaces for children to thrive,” Ms. Olsson said.

Improve children’s environments

Children in poor families tend to face greater exposure to environmental harm –entrenching and amplifying existing disadvantages and inequities.

Mounting waste, harmful pollutants and exhausted natural resources are taking a toll on our children’s physical and mental health and threatening our planet’s sustainability,” said the UNICEF official.

As such, UNICEF has urged national, regional, and local governments to improve children’s environments by reducing waste, air and water pollution, and ensuring high-quality housing and neighbourhoods.

Children’s voices count

Governments and businesses must immediately honour their commitments to reduce greenhouse gas emissions by 2050. And climate adaptation should also be at the forefront of action across various sectors – from education to infrastructure.

Child-sensitive environmental policies must ensure that children’s needs are built into decision making and that their perspectives are considered when designing policies that will disproportionately affect future generations.

UNICEF’s report outlines that although children are the main stakeholders of the future and will face today’s environmental problems for the longest time, they are the least able to influence the course of events.

“We must pursue policies and practices that safeguard the natural environment upon which children and young people depend the most,” Ms. Olsson said.

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Boosting brain function in later life through singing

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Ask anyone in a choir why they enjoy it, and they will tell you about the euphoric effects singing has on their mental health. A team of neuroscientists and clinical psychologists based at the University of Helsinki (Finland) believe these benefits could extend to improving brain function and treating aphasia.

Professor Teppo Särkämö is studying how ageing affects the way singing is processed by the brain, which could have important therapeutic applications. ‘We know a lot about speech processing, but not much about singing. We’re exploring how different singing related functions might be preserved in many neurological diseases,’ he explained.

For people with aphasia, a condition which severely impairs communication and is commonly caused by stroke, communication can be almost impossible as they struggle to sound out the right words. Yet, through a technique known as ‘melodic intonation therapy’ – whereby people are asked to sing an everyday sentence instead of speaking it – quite incredibly they often find a voice.

Coordinator of the PREMUS project, Prof Särkämö and his team are using similar methods, scaling-up the approach through specially-run ‘senior choirs’ that involve aphasic patients and their families. The scientists are exploring how singing could play an important rehabilitative role for cases of aphasia and might prevent cognitive decline too.

Hitting the right notes

The PREMUS study is coordinated with a local aphasia organisation in Helsinki and involves around 25 people per choir, both aphasia patients and their family caregivers. Results of the trial show encouraging results.

‘Ultimately, the aim through our work with persons with aphasia is to use singing as a tool to train speech production and eventually enable them to communicate without singing. But through the choirs we are beginning to see how this approach is translating to people’s daily life as an important communication tool,’ said Särkämö.

Alongside an aphasia choir, the team has also carried out extensive fMRI brain scans of young, middle aged and older adults who participate in choirs to understand why singing is so important at different life stages. Their results indicate that as we age, the brain networks involved in singing undergo fewer changes than those that process speech, suggesting that singing is more widespread in the brain and more resilient to ageing.

Their studies also suggest that being actively engaged in singing, as opposed to listening to choral music for example, is crucial. ‘When you’re singing, you are engaging in the frontal and parietal systems in the brain where you regulate your own behaviour, and you use more of your motor and cognitive resources in terms of vocal control and executive functions,’ said Särkämö.

Early results from a longitudinal study, which compared neurocognitive functioning between members of senior choirs and healthy older adults (who do not sing) showed the positive effects of singing on cognitive and auditory functioning and the importance of the social interaction it brings, which may help delay the onset of dementia.

Choir members performed better in neuropsychological tests, reported fewer cognitive difficulties, and had higher social integration. Electroencephalogram measurements of the same groups suggest that the choir singers had more advanced higher-level auditory processing abilities, especially for combining pitch and location information in frontotemporal brain regions, something Särkämö attributes to the complexity of the sound environment in choir singing.

The next step will be to replicate and expand this work with senior choirs for patients diagnosed with Alzheimer’s and to develop a large-scale clinical trial to test the effect. The challenge, however, is likely to be different with Alzheimer’s: whereas patients may remember songs from their past, Särkämö is unsure to what extent they can learn and retain new lyrics.

He is both optimistic and realistic about this work. ‘This is all about trying to stimulate the remaining networks in the brain. We believe singing could help to regain some of those functions, but of course with Alzheimer’s it’s a brutal, progressive disorder so it’s a matter of buying more time and trying to slow down the pattern of decline happening already.’

Same song sheet

Someone else firmly focused on responding to the challenges of an ageing population is Christian A. Drevon, Professor of medicine at the University of Oslo (Norway). Drevon is a specialist in biomarkers and is now using his expertise to understand the different factors affecting neurocognitive function in the EU-funded Lifebrain project.

’Most studies about Alzheimer’s are cross-sectional where you take a group of people, look at a certain time and associate certain things with those who have the disease and those who don’t,’ he explained. ‘However, this is often not causal; you can’t tell if it’s the reason for the disease or if it’s just a consequence of it.’

To really understand what’s happening with Alzheimer’s and dementia, data are needed for individuals spanning periods both when they are healthy and when they are not, to tease apart what has gone wrong. Unpicking this question is the primary aim of Lifebrain, coordinated by psychologists Professors Kristine Walhovd and Anders Fjell.

By pooling pre-existing MRI brain scan data from people right across Europe, the Lifebrain project has analysed the significance of a range of different factors on cognition when we age and how this might vary between individuals.

To analyse over 40 000 brain scans from more than 5 000 people aged 1880 across seven countries, the first challenge was to harmonise the data. Do MRI scans in Sweden and Spain produce the same results? To ensure they do, Lifebrain sent eight participants around Europe to be scanned and to adjust equipment accordingly.

All psychological tests (including cognitive tests) and other collected data (body weight; demographic; genetic; and lifestyle data, including sleep and diet) were harmonised.

Next, the team linked MRI data with additional databases which uncovered new insights about how where you live and what access you have to green space might help lower dementia risk. Conversely, it also helped to reveal how education and sleep may be less important for future risk of dementia than previously assumed.  

‘Lots of studies have claimed education is really important for reducing the risk of dementia. But if you follow people longitudinally through life there’s actually no association,’ said Drevon. ‘That doesn’t mean education isn’t important; it means it’s probably not true that education will prevent you from developing dementia. We have to search for other factors of importance.’

Given the expense of MRIs, Drevon suggests tiny blood samples (dried blood spots) could be taken by finger-prick without professional support to provide individual insights in the future. Analysed in an advanced laboratory like Vitas Ltd – Lifebrain partner – this could be a game-changer in providing tailored, online advice about individual risks.

‘If you really want to improve lifestyle, you probably have to personalise it. You have to measure several factors on an individual level across the life course,’ he said. ‘Our best chance of fighting cognitive decline and dementia will come from early preventative measures using this lifespan data approach.’

Work out songs

In time Prof Drevon hopes these personalised insights could help delay or potentially eradicate certain aspects of dementia. In the meantime, what about singing to stave off cognitive decline as proposed by Särkämö through the PREMUS project? Does he agree singing could be an important preventative step?

‘Well, the brain is like a muscle. If you train it, you make it fit, and if you use your brain for singing, it’s complicated, there are a lot of processes, it’s about remembering. Of course, there are other ways of training the brain, but singing is a very good example of how you can help to improve brain function.’  

The research in this article was funded by the EU. This article was originally published in Horizon, the EU Research and Innovation Magazine.  

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Health & Wellness

The Benefits Of Feeding Your Baby Organic Formula

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There are many benefits that come from feeding your baby formula milk, not to mention that it is much easier and allows for mothers to be more flexible with their schedule. There is no need for mom to worry about what she is and is not putting in her body and not breastfeeding means that dad can share the feeding duty as well.

However, when it comes to using formula milk, the last thing any parent wants is to be feeding their beloved child with one that contains lots of nasty and / or potentially harmful chemicals that impact on both their growth and their overall general health /wellbeing.

Organic baby formula is the perfect choice then in order to ensure that your baby gets everything that they need to grow up and develop into a happy and healthy child. There are many brands nowadays that produce organic baby formula milk and you can buy it online from MyOrganicCompany; learn more about the company and its products by clicking the link.

There are many different benefits to feeding your baby organic formula milk with some of the most significant ones listed below for you to read in detail.

It does not contain any synthetic ingredients

As opposed to conventional baby formula milk, the organic stuff is free from any ingredients that are synthetic. Synthetic ingredients should not be going anywhere near the insides of a delicate newborn baby. In addition to this, none organic baby formulas typically contain artificial sweeteners that can seriously damage a young child’s health.

When consumed regularly, many babies build up an intolerance to the synthetic and artificial ingredients found in conventional baby formula. Over time, they may experience certain gastrointestinal health issues as a result. This includes things like constipation and / or diarrhea. By feeding your baby a formula milk that is free of synthetic ingredients, you do not have to worry about it happening to them.

It contains lots of nutrients

While a formula milk tasting nice can encourage a baby to feed, the main goal here is to get all of the required nutrients and minerals into them so that they can grow into a strong and healthy child. With organic baby formula, being dense in nutrients is the main priority and so typically it is jam packed full of the correct blend of vitamins, such as Vitamin E, that are necessary for the healthy functioning of the red blood cells, immune system, and organs of your baby. The great thing about Vitamin E is that it is also an antioxidant and so it works to protect your child’s body from being attacked by free radicals.

This is why you should feed your baby organic formula milk rather than the none organic stuff. The most nutrient rich organic baby formulas are in fact those from European countries as they pay extra close attention to what good stuff goes into the formula milk.

It is advantageous to mental development

Organic baby formula milk is also much better for the mental development of your baby. As well as having way more nutrients, this type of formula milk also contains the perfect amount of polyunsaturated fatty acids, including Omega 3 fatty acids. These nutrients are particularly beneficial to your baby’s health as they help significantly in mental development (as well as physical development).

Mental issues, such as depression and anxiety, are often things that affect people for the whole of their life and so it is, of course advisable to dry and avoid these things from happening in the very first place. Getting a good amount of both Omega 3 fatty acids and all other types of polyunsaturated fatty acids is therefore essential for preventing mental development issues from occurring.

Getting these nutrients can also go some way to making your child more communicative and more intelligent. Additionally, they can prevent certain behavioral issues from arising also. Finally, if your baby has lots of these types of nutrients in them, it makes them more protected from conditions, such as autism, attention deficit hyperactivity disorder (ADHD), and even cerebral palsy.

It does not contain any GMOs

Genetically modified organisms (GMOs) are in a large proportion of the foods that we eat nowadays. These organisms are not good for adults to consume and so are even worse for developing babies to feed on. It has been estimated that in the United States, almost as much as 75 percent of all food items available in grocery stores have some amount of GMO ingredients in them. By using organic baby formula milk, you can ensure that your little bundle of joy is not consuming any of these harmful GMOs or any synthetic ingredients at all.

When it comes to what we put into our bodies and that of our offspring, natural is always the best option to go with. Nowaday cows are constantly being pumped full of various different pesticides, antibiotics, and so much more and this will eventually end up coming out in the milk they produce. If you feed this milk to your baby then no doubt they will be consuming some of these harmful chemicals.

Rather than having this worry, it is good to know that the organic baby formula milk that you are bringing your child up on is free from all of these harmful things, whilst at the same time being full of all of the good stuff that they need to thrive.

It comes with lots of choice

It may be the case that your baby has specific dietary requirements that need to be met in order for them to get all of the nutrients and goodness that they need. They may even be suffering from certain health conditions, such as constipation and / or gas and organic baby formula milk can help to alleviate some of the symptoms associated with these things.

There also exist a wide range of organic formulas to treat common infant / childhood allergies.

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