̽��ֱ�� of Cambridge - Cambridge Neuroscience

Adolescents who sleep longer perform better at cognitive tasks

cjb250 — Tue, 22 Apr 2025 15:00:23 +0000

But the study of adolescents in the US also showed that even those with better sleeping habits were not reaching the amount of sleep recommended for their age group.

Sleep plays an important role in helping our bodies function. It is thought that while we are asleep, toxins that have built up in our brains are cleared out, and brain connections are consolidated and pruned, enhancing memory, learning, and problem-solving skills. Sleep has also been shown to boost our immune systems and improve our mental health.

During adolescence, our sleep patterns change. We tend to start going to bed later and sleeping less, which affects our body clocks. All of this coincides with a period of important development in our brain function and cognitive development. ̽��ֱ��American Academy of Sleep Medicine says that the ideal amount of sleep during this period is between eight- and 10-hours’ sleep.

Professor Barbara Sahakian from the Department of Psychiatry at the ̽��ֱ�� of Cambridge said: “Regularly getting a good night’s sleep is important in helping us function properly, but while we know a lot about sleep in adulthood and later life, we know surprisingly little about sleep in adolescence, even though this is a crucial time in our development. How long do young people sleep for, for example, and what impact does this have on their brain function and cognitive performance?”

Studies looking at how much sleep adolescents get usually rely on self-reporting, which can be inaccurate. To get around this, a team led by researchers at Fudan ̽��ֱ��, Shanghai, and the ̽��ֱ�� of Cambridge turned to data from the Adolescent Brain Cognitive Development (ABCD) Study, the largest long-term study of brain development and child health in the United States.

As part of the ABCD Study, more than 3,200 adolescents aged 11-12 years old had been given FitBits, allowing the researchers to look at objective data on their sleep patterns and to compare it against brain scans and results from cognitive tests. ̽��ֱ��team double-checked their results against two additional groups of 13-14 years old, totalling around 1,190 participants. ̽��ֱ��results are published today in Cell Reports.

̽��ֱ��team found that the adolescents could be divided broadly into one of three groups:

Group One, accounting for around 39% of participants, slept an average (mean) of 7 hours 10 mins. They tended to go to bed and fall asleep the latest and wake up the earliest.

Group Two, accounting for 24% of participants, slept an average of 7 hours 21 mins. They had average levels across all sleep characteristics.

Group Three, accounting for 37% of participants, slept an average of 7 hours 25 mins. They tended to go to bed and fall asleep the earliest and had lower heart rates during sleep.

Although the researchers found no significant differences in school achievement between the groups, when it came to cognitive tests looking at aspects such as vocabulary, reading, problem solving and focus, Group Three performed better than Group Two, which in turn performed better than Group One.

Group Three also had the largest brain volume and best brain functions, with Group One the smallest volume and poorest brain functions.

Professor Sahakian said: “Even though the differences in the amount of sleep that each group got was relatively small, at just over a quarter-of-an-hour between the best and worst sleepers, we could still see differences in brain structure and activity and in how well they did at tasks. This drives home to us just how important it is to have a good night’s sleep at this important time in life.”

First author Dr Qing Ma from Fudan ̽��ֱ�� said: “Although our study can’t answer conclusively whether young people have better brain function and perform better at tests because they sleep better, there are a number of studies that would support this idea. For example, research has shown the benefits of sleep on memory, especially on memory consolidation, which is important for learning.”

̽��ֱ��researchers also assessed the participants’ heart rates, finding that Group Three had the lowest heart rates across the sleep states and Group One the highest. Lower heart rates are usually a sign of better health, whereas higher rates often accompany poor sleep quality like restless sleep, frequent awakenings and excessive daytime sleepiness.

Because the ABCD Study is a longitudinal study – that is, one that follows its participants over time – the team was able to show that the differences in sleep patterns, brain structure and function, and cognitive performance, tended be present two years before and two years after the snapshot that they looked at.

Senior author Dr Wei Cheng from Fudan ̽��ֱ�� added: “Given the importance of sleep, we now need to look at why some children go to bed later and sleep less than others. Is it because of playing videogames or smartphones, for example, or is it just that their body clocks do not tell them it’s time to sleep until later?”

̽��ֱ��research was supported by the National Key R&D Program of China, National Natural Science Foundation of China, National Postdoctoral Foundation of China and Shanghai Postdoctoral Excellence Program. ̽��ֱ��ABCD Study is supported by the National Institutes of Health.

Reference

Ma, Q et al. Neural correlates of device-based sleep characteristics in adolescents. Cell Reports; 22 Apr 2025; DOI: 10.1016/j.celrep.2025.115565

Adolescents who sleep for longer – and from an earlier bedtime – than their peers tend to have improved brain function and perform better at cognitive tests, researchers from the UK and China have shown.

Even though the differences in the amount of sleep that each group got was relatively small, we could still see differences in brain structure and activity and in how well they did at tasks

Barbara Sahakian

harpazo_hope (Getty Images)

Teenager asleep and wrapped in a blanket

̽��ֱ��text in this work is licensed under a Creative Commons Attribution-NonCommercial-ShareAlike 4.0 International License. Images, including our videos, are Copyright © ̽��ֱ�� of Cambridge and licensors/contributors as identified. All rights reserved. We make our image and video content available in a number of ways – on our main website under its Terms and conditions, and on a range of channels including social media that permit your use and sharing of our content under their respective Terms.

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̽��ֱ��Cambridge Awards 2024 for Research Impact and Engagement

zs332 — Mon, 03 Feb 2025 10:27:01 +0000

Meet the winner of the Cambridge Awards 2024 for Research Impact and Engagement and learn more about their projects.

Loneliness linked to higher risk of heart disease and stroke and susceptibility to infection

cjb250 — Fri, 03 Jan 2025 10:00:00 +0000

Researchers from the UK and China drew this conclusion after studying proteins from blood samples taken from over 42,000 adults recruited to the UK Biobank. Their findings are published today in the journal Nature Human Behaviour.

Social relationships play an important role in our wellbeing. Evidence increasingly demonstrates that both social isolation and loneliness are linked to poorer health and an early death. Despite this evidence, however, the underlying mechanisms through which social relationships impact health remain elusive.

One way to explore biological mechanisms is to look at proteins circulating in the blood. Proteins are molecules produced by our genes and are essential for helping our bodies function properly. They can also serve as useful drug targets, allowing researchers to develop new treatments to tackle diseases.

A team led by scientists at the ̽��ֱ�� of Cambridge, UK, and Fudan ̽��ֱ��, China, examined the ‘proteomes’ – the suite of proteins – in blood samples donated by over 42,000 adults aged 40-69 years who are taking part in the UK Biobank. This allowed them to see which proteins were present in higher levels among people who were socially isolated or lonely, and how these proteins were connected to poorer health.

̽��ֱ��team calculated social isolation and loneliness scores for individuals. Social isolation is an objective measure based on, for example, whether someone lives alone, how frequently they have contact with others socially, and whether they take part in social activities. Loneliness, on the other hand, is a subjective measure based on whether an individual feels lonely.

When they analysed the proteomes and adjusted for factors such as age, sex and socioeconomic background, the team found 175 proteins associated with social isolation and 26 proteins associated with loneliness (though there was substantial overlap, with approximately 85% of the proteins associated with loneliness being shared with social isolation). Many of these proteins are produced in response to inflammation, viral infection and as part of our immune responses, as well as having been linked to cardiovascular disease, type 2 diabetes, stroke, and early death.

̽��ֱ��team then used a statistical technique known as Mendelian randomization to explore the causal relationship between social isolation and loneliness on the one hand, and proteins on the other. Using this approach, they identified five proteins whose abundance was caused by loneliness.

Dr Chun Shen from the Department of Clinical Neurosciences at the ̽��ֱ�� of Cambridge and the Institute of Science and Technology for Brain-Inspired Intelligence, Fudan ̽��ֱ��, said: “We know that social isolation and loneliness are linked to poorer health, but we’ve never understood why. Our work has highlighted a number of proteins that appear to play a key role in this relationship, with levels of some proteins in particular increasing as a direct consequence of loneliness.

Professor Jianfeng Feng from the ̽��ֱ�� of Warwick said: "There are more than 100,000 proteins and many of their variants in the human body. AI and high throughput proteomics can help us pinpoint some key proteins in prevention, diagnosis, treatment and prognosis in many human diseases and revolutionise the traditional view of human health.

" ̽��ֱ��proteins we’ve identified give us clues to the biology underpinning poor health among people who are socially isolated or lonely, highlighting why social relationships play such an important part in keeping us healthy.”

One of the proteins produced in higher levels as a result of loneliness was ADM. Previous studies have shown that this protein plays a role in responding to stress and in regulating stress hormones and social hormones such as oxytocin – the so-called ‘love hormone’ – which can reduce stress and improve mood.

̽��ֱ��team found a strong association between ADM and the volume of the insula, a brain hub for interoception, our ability to sense what's happening inside our body – the greater the ADM levels, the smaller the volume of this region. Higher ADM levels were also linked to lower volume of the left caudate, a region involved in emotional, reward, and social processes. In addition, higher levels of ADM were linked to increased risk of early death.

Another of the proteins, ASGR1, is associated with higher cholesterol and an increased risk of cardiovascular disease, while other identified proteins play roles in the development of insulin resistance, atherosclerosis (‘furring’ of the arteries) and cancer progression, for example.

Professor Barbara Sahakian from the Department of Psychiatry at the ̽��ֱ�� of Cambridge said: “These findings drive home the importance of social contact in keeping us well. More and more people of all ages are reporting feeling lonely. That’s why the World Health Organization has described social isolation and loneliness as a ‘global public health concern’. We need to find ways to tackle this growing problem and keep people connected to help them stay healthy.”

̽��ֱ��research was supported by the National Natural Sciences Foundation of China, China Postdoctoral Science Foundation, Shanghai Rising-Star Program, National Key R&D Program of China, Shanghai Municipal Science and Technology Major Project, 111 Project, Shanghai Center for Brain Science and Brain-Inspired Technology, and Zhangjiang Lab.

Reference
Shen, C et al. Plasma proteomic signatures of social isolation and loneliness associated with morbidity and mortality. Nat Hum Behav; 3 Jan 2025; DOI: 10.1038/s41562-024-02078-1

Interactions with friends and family may keep us healthy because they boost our immune system and reduce our risk of diseases such as heart disease, stroke and type 2 diabetes, new research suggests.

More and more people of all ages are reporting feeling lonely. We need to find ways to tackle this growing problem and keep people connected to help them stay healthy

Barbara Sahakian

Noah Silliman

Person looking out through window

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Boost your life in 2025: Top tips for a healthier body and mind from Cambridge experts

jg533 — Thu, 02 Jan 2025 09:16:07 +0000

Five Cambridge experts share their top tips on ways to boost your body and mind, backed up by their own research

Magnetic field applied to both sides of brain shows rapid improvement for depression

cjb250 — Mon, 28 Oct 2024 15:23:23 +0000

̽��ֱ��treatment – known as repetitive transcranial magnetic stimulation (TMS) – involves placing an electromagnetic coil against the scalp to relay a high-frequency magnetic field to the brain.

Around one in 20 adults is estimated to suffer from depression. Although treatments exist, such as anti-depressant medication and cognitive behavioural therapy (‘talking therapy’), they are ineffective for just under one in three patients.

One of the key characteristics of depression is under-activity of some regions (such as the dorsolateral prefrontal cortex) and over-activity of others (such as the orbitofrontal cortex (OFC)).

Repetitive transcranial magnetic stimulation applied to the left side of the dorsolateral prefrontal cortex (an area at the upper front area of the brain) is approved for treatment of depression in the UK by NICE and in the US by the FDA. It has previously been shown to lead to considerable improvements among patients after a course of 20 sessions, but because the sessions usually take place over 20-30 days, the treatment is not ideal for everyone, particularly in acute cases or where a person is suicidal.

In research published in Psychological Medicine, scientists from Cambridge, UK, and Guiyang, China, tested how effective an accelerated form of TMS is. In this approach, the treatment is given over 20 sessions, but with four sessions per day over a period of five consecutive days.

̽��ֱ��researchers also tested a ‘dual’ approach, whereby a magnetic field was additionally applied to the right-hand side of the OFC (which sits below the dorsolateral prefrontal cortex).

Seventy-five patients were recruited to the trial from the Second People’s Hospital of Guizhou Province in China. ̽��ֱ��severity of their depression was measured on a scale known as the Hamilton Rating Scale of Depression.

Participants were split randomly into three groups: a ‘dual’ group receiving TMS applied first to the right- and then to the left-hand sides of the brain; a ‘single’ group receiving sham TMS to the right-side followed by active TMS applied to the left-side; and a control group receiving a sham treatment to both sides. Each session lasted in total 22 minutes.

There was a significant improvement in scores assessed immediately after the final treatment in the dual treatment group compared to the other two groups. When the researchers looked for clinically-relevant responses – that is, where an individual’s score fell by at least 50% – they found that almost half (48%) of the patients in the dual treatment group saw such a reduction, compared to just under one in five (18%) in the single treatment group and fewer than one in 20 (4%) in the control group.

Four weeks later, around six in 10 participants in both the dual and single treatment groups (61% and 59% respectively) showed clinically relevant responses, compared to just over one in five (22%) in the control group.

Professor Valerie Voon from the Department of Psychiatry at the ̽��ֱ�� of Cambridge, who led the UK side of the study, said: “Our accelerated approach means we can do all of the sessions in just five days, rapidly reducing an individual’s symptoms of depression. This means it could be particularly useful in severe cases of depression, including when someone is experiencing suicidal thoughts. It may also help people be discharged from hospital more rapidly or even avoid admission in the first place.

“ ̽��ֱ��treatment works faster because, by targeting two areas of the brain implicated in depression, we’re effectively correcting imbalances in two import processes, getting brain regions ‘talking’ to each other correctly.”

̽��ֱ��treatment was most effective in those patients who at the start of the trial showed greater connectivity between the OFC and the thalamus (an area in the middle of the brain responsible for, among other things, regulation of consciousness, sleep, and alertness). ̽��ֱ��OFC is important for helping us make decisions, particularly in choosing rewards and avoiding punishment. Its over-activity in depression, particularly in relation to its role in anti-reward or punishment, might help explain why people with depression show a bias towards negative expectations and ruminations.

Dr Yanping Shu from the Guizhou Mental Health Centre, Guiyang, China, said: “This new treatment has demonstrated a more pronounced – and faster – improvement in response rates for patients with major depressive disorder. It represents a significant step forward in improving outcomes, enabling rapid discharge from hospitals for individuals with treatment-resistant depression, and we are hopeful it will lead to new possibilities in mental health care.”

Dr Hailun Cui from Fudan ̽��ֱ��, a PhD student in Professor Voon’s lab at the time of the study, added: “ ̽��ֱ��management of treatment-resistant depression remains one of the most challenging areas in mental health care. These patients often fail to respond to standard treatments, including medication and psychotherapy, leaving them in a prolonged state of severe distress, functional impairment, and increased risk of suicide.

“This new TMS approach offers a beacon of hope in this difficult landscape. Patients frequently reported experiencing ‘lighter and brighter’ feelings as early as the second day of treatment. ̽��ֱ��rapid improvements, coupled with a higher response rate that could benefit a broader depressed population, mark a significant breakthrough in the field.”

Just under a half (48%) of participants in the dual treatment group reported local pain where the dual treatment was applied, compared to just under one in 10 (9%) of participants in the single treatment group. However, despite this, there were no dropouts.

For some individuals, this treatment may be sufficient, but for others ‘maintenance therapy’ may be necessary, with an additional day session if their symptoms appear to be worsening over time. It may also be possible to re-administer standard therapy as patients can then become more able to engage in psychotherapy. Other options include using transcranial direct current stimulation, a non-invasive form of stimulation using weak electrical impulses that can be delivered at home.

̽��ֱ��researchers are now exploring exactly which part of the orbitofrontal cortex is most effective to target and for which types of depression.

̽��ֱ��research was supported by in the UK by the Medical Research Council and by the National Institute for Health and Care Research Cambridge Biomedical Research Centre.*

Reference
Cui, H, Ding, H & Hu, L et al. A novel dual-site OFC-dlPFC accelerated repetitive transcranial magnetic stimulation for depression: a pilot randomized controlled study. Psychological Medicine; 23 Oct 2024; DOI: 10.1017/S0033291724002289

*A full list of funders is available in the journal paper.

A type of therapy that involves applying a magnetic field to both sides of the brain has been shown to be effective at rapidly treating depression in patients for whom standard treatments have been ineffective.

Our accelerated approach means we can do all of the sessions in just five days, rapidly reducing an individual’s symptoms of depression

Valerie Voon

TheDigitalArtist

Digital image of a brain

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Cambridge joins forces with ARIA to fast-track radical new technologies to revolutionise brain health

Anonymous — Wed, 09 Oct 2024 12:57:22 +0000

̽��ֱ��collaboration, which includes researchers from the ̽��ֱ�� of Cambridge, aims to accelerate progress on new neuro-technologies, including miniaturised brain implants designed to treat depression, dementia, chronic pain, epilepsy and injuries to the nervous system.

Neurological and mental health disorders will affect four in every five people in their lifetimes, and present a greater overall health burden than cancer and cardiovascular disease combined. For example, 28 million people in the UK are living with chronic pain and 1.3 million people with traumatic brain injury.

Neuro-technology – where technology is used to control the nervous system - has the potential to deliver new treatments for these disorders, in much the same way that heart pacemakers, cochlear implants and spinal implants have transformed medicine in recent decades.

̽��ֱ��technology can be in the form of electronic brain implants that reset abnormal brain activity or help deliver targeted drugs more effectively, brain-computer interfaces that control prosthetic limbs, or technologies that train the patient’s own cells to fight disease. ARIA’s Scalable Neural Interfaces opportunity space is exploring ways to make the technology more precise, less invasive, and applicable to a broader range of diseases.

Currently, an implant can only interact with large groups of neurons, the cells that transmit information around the brain. Building devices that interact with single neurons will mean a more accurate treatment. Neuro-technologies also have the potential to treat autoimmune disorders, including rheumatoid arthritis, Crohn’s disease and type-1 diabetes.

̽��ֱ��science of building technology small enough, precise enough and cheap enough to make a global impact requires an environment where the best minds from across the UK can collaborate, dream up radical, risky ideas and test them without fear of failure.

Professor George Malliaras from the ̽��ֱ�� of Cambridge’s Department of Engineering is one of the project leaders. “Miniaturised devices have the potential to change the lives of millions of people currently suffering from neurological conditions and diseases where drugs have no effect,” he said. “But we are working at the very edge of what is possible in medicine, and it is hard to find the support and funding to try radical, new things. That is why the partnership with ARIA is so exhilarating, because it is giving brilliant people the tools to turn their original ideas into commercially viable devices that are cheap enough to have a global impact.”

Cambridge’s partnership with ARIA will create a home for original thinkers who are struggling to find the funding, space and mentoring needed to stress-test their radical ideas. ̽��ֱ��three-year partnership is made up of two programmes:

̽��ֱ��Fellowship Programme (up to 18 fellowships)

Blue Sky Fellows – a UK-wide offer - we will search the UK for people from any background, with a radical idea in this field and the plan and personal skills to develop it. ̽��ֱ��best people will be offered a fellowship with the funding to test their ideas in Cambridge rapidly. These Blue Sky Fellows will receive mentorship from our best medical, scientific and business experts and potentially be offered accommodation at a Cambridge college. We will be looking for a specific type of person to be a Blue Sky Fellow. They must be the kind of character who thinks at the very edge of the possible, who doesn’t fear failure, and whose ideas have the potential to change billions of lives, yet would struggle to find funding from existing sources. Not people who think outside the box, more people who don’t see a box at all.

Activator Fellows - a UK-wide offer - those who have already proved that their idea can work, yet need support to turn it into a business, will be invited to become Activator Fellows. They will be offered training in entrepreneurial skills including grant writing, IP management and clinical validation, so their innovation can be ready for investment.

̽��ֱ��Ecosystem Programme

̽��ֱ��Ecosystem Programme is about creating a vibrant, UK-wide neurotechnology community where leaders from business, science, engineering, academia and the NHS can meet, spark ideas and form collaborations. This will involve quarterly events in Cambridge, road trip events across the UK and access to the thriving online Cambridge network, Connect: Health Tech.

“This unique partnership is all about turning radical ideas into practical, low-cost solutions that change lives,” said Kristin-Anne Rutter, Executive Director of Cambridge ̽��ֱ�� Health Partners. “Cambridge is fielding its best team to make this work and using its networks to bring in the best people from all over the UK. From brilliant scientists to world-leading institutes, hospitals and business experts, everyone in this collaboration is committed to the ARIA partnership because, by working together, we all see an unprecedented opportunity to make a real difference in the world.”

“Physical and mental illnesses and diseases that affect the brain such as dementia are some of the biggest challenges we face both as individuals and as a society,” said Dr Ben Underwood, Associate Professor of Psychiatry at the ̽��ֱ�� of Cambridge and Honorary Consultant Psychiatrist at Cambridgeshire and Peterborough NHS Foundation Trust. “This funding will bring together different experts doing things at the very limits of science and developing new technology to improve healthcare. We hope this new partnership with the NHS will lead to better care and treatment for people experiencing health conditions.”

Cambridge partners in the project include the Departments of Engineering and Psychiatry, Cambridge Neuroscience, the Milner Therapeutics Institute, the Maxwell Centre, Cambridge ̽��ֱ�� Health Partners (CUHP), Cambridge Network, the Babraham Research Campus, Cambridgeshire and Peterborough NHS Foundation Trust, and Vellos.

A team from across the Cambridge life sciences, technology and business worlds has announced a multi-million-pound, three-year collaboration with the Advanced Research and Invention Agency (ARIA), the UK government’s new research funding agency.

Science Photo Library via Getty Images

Illustration of human brain

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CamFest Speaker Spotlight: Dr Anna Moore

zs332 — Tue, 26 Mar 2024 13:42:26 +0000

Assistant Professor of Child Psychiatry at the ̽��ֱ�� of Cambridge and programme lead, Dr Anna Moore, discusses the NIHR BioResource’s national childhood health research programme, D-CYPHR. ̽��ֱ��groundbreaking programme is a world first and aims to be the largest ever DNA research programme involving children aged 0-15.

Clinical trial underway to treat ultra-rare genetic disease with possible link to leader of mutiny on the Bounty

jg533 — Thu, 21 Mar 2024 09:00:00 +0000

A clinical trial to look at repurposing the UK-licensed medicine deferiprone for patients with the ultra-rare genetic disease neuroferritinopathy has launched today at the ̽��ֱ�� of Cambridge.

Neuroferritinopathy is a progressive and incurable brain disorder caused by changes in a gene that produces a specific protein - ferritin light chain protein. This change leads to the build-up of iron in the brain. ̽��ֱ��disease usually appears in middle-aged adults and causes severe symptoms that impact on day-to-day life, eventually resulting in the loss of speech and swallowing. There are currently no effective treatments.

Funded by LifeArc, the new randomised placebo-controlled trial - DefINe - will be led by Professor Patrick Chinnery from the Department of Clinical Neurosciences. It aims to stop the progression of the disease by reducing the iron accumulation in the brain with an existing drug called deferiprone. Deferiprone is an affordable oral tablet that is already licensed for use in the UK to reduce iron levels in blood conditions like thalassemia. If successful, the trial could also open the possibility of deferiprone being used for other neurodegenerative conditions linked with build-up of iron the brain.

Professor Chinnery said: “Neuroferritinopathy leads to severe disability and currently has no cure. ̽��ֱ��DefINe trial will show whether we can stop the disease in its tracks by pulling iron out of the brain using a well-known medicine called deferiprone.

“By funding this study, LifeArc has given the first hope of a treatment for affected families. If successful, the trial will open the possibility of using a similar approach for other neurodegenerative conditions linked to the build-up of iron in the brain, including Parkinson’s disease.”

Neuroferritinopathy affects approximately 100 patients worldwide. Initial discovery of the condition came when a surprising number of individuals diagnosed found to live in the Lake District in Cumbria experienced similar symptoms with a series of incorrect diagnoses. Research into the ancestry of these families by Professor John Burn, a clinical geneticist at Newcastle Hospitals NHS Foundation Trust, discovered the genetic commonality and also found an interesting potential link to the past.

Professor Burn found that a rare mutation caused the progression of the condition and almost all the known cases were likely to be descended from the same ancestor. He traced it back to the 18th Century in Cockermouth in Cumbria and families with the surname Fletcher. Professor Burn suggested they could have shared common ancestry with Fletcher Christian (Fletcher being his surname), known for leading the mutiny on the Bounty in April 1789, given he was also from the region.

̽��ֱ��DefINe trial will involve 40 patients taking the drug for a year, who will undergo state-of-the-art 7T magnetic resonance imaging (MRI) scanning to monitor the iron levels in the brain throughout. ̽��ֱ��evidence collected will form the basis of an application for licensing in the UK under ‘Exceptional Circumstances’, which is often used for rare conditions where the number of people affected is low. This means, if the trial is successful the drug could go on to benefit all people with the condition more quickly.

Samantha Denison, a patient hoping to participate in the trial, said: “It came as such a surprise to be informed of the trial and to learn that we have not been forgotten about. To have the chance to be involved in the trial gives me such hope. If it can help to slow or stop the condition progressing, that would be a huge relief. Just to know that by taking part we could also be helping future generations, is amazing.”

LifeArc has contributed £750,000 to the project and Lipomed, a Swiss life sciences company, has offered to provide both a cost-effective generic form of deferiprone, Deferiprone Lipomed, and a placebo to the trial – a Gift in Kind worth £250,000.

Dr Catriona Crombie, Head of LifeArc’s Rare Disease Translational Challenge, said: “Drug repurposing trials like this are an increasingly effective way of taking treatments that have already been approved and applying them to new conditions and diseases. This will help unlock new treatments for conditions that currently have few, if any, available."

Dr Chantal Manz, Chief Scientific Officer Lipomed AG, Switzerland, said: “Lipomed is very excited to support this promising study concept in patients with neuroferritinopathy, by providing deferiprone 500 mg film-coated tablets and matching placebo tablets. We recognise the unmet clinical need and the potentially significant benefit of this orally active iron chelator. Deferiprone is able to penetrate the blood-brain barrier and may reduce cerebral iron accumulation in patients with this extremely rare, but devastating genetic neurodegenerative disorder, for which no alternative treatments are available.”

Adapted from a press release by LifeArc.

If successful, the trial will open the possibility of using a similar approach for other neurodegenerative conditions linked to the build-up of iron in the brain, including Parkinson’s disease.

Patrick Chinnery

Patrick Chinnery looks at brain scans

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