Today, UK Research and Innovation (UKRI), has announced £34 million investment in a ground-breaking project, BioFAIR, which aims to overhaul research data management across the nation.

The project, initially proposed by the ELIXIR-UK community, which is co-led by Professor Carole Goble from the University of Manchester, aims to establish a cohesive, UK-wide digital research infrastructure that bridges current gaps between researchers, digital research technical professionals, existing institutional digital research infrastructures, and the funder-community partnership.

It will deliver a step change in the UK’s capability to translate existing and future life science data assets into world leading research in response to some of society’s most pressing challenges.

ELIXIR-UK is the UK Node of ELIXIR, a European project to integrate life sciences data across the continent with the aim of facilitating the linking of data worldwide. Professor Goble has been co-leading on the business case and investment activity for the project in partnership with the Earlham Institute and UKRI over the last six years and has played an instrumental role in securing the award for the UK. She is also leading the architecture requirements development of the BioFAIR Commons.

BioFAIR will be a catalyst for innovation and discovery and over its five-year life span will:

• accelerate the adoption of findable, accessible, interoperable and reusable (FAIR) data principles across the UK life sciences, making it more useful and valuable to researchers than ever before
• unify the UK’s currently fragmented digital research landscape, fostering unprecedented opportunities for collaboration and coordination among the national life sciences community
• break down barriers to democratise data accessibility, giving UK researchers the resources and autonomy needed for innovation and discovery to flourish
• coordinate and deliver extensive training and support for practitioners at all levels, building critical workforce capacity and securing the UK’s position as a global leader in life sciences

Fundamental to the BioFAIR concept are its four key capabilities. Each will be assembled from existing data tools and services developed and deployed by the UK and international life science research communities.

Collectively, the four capabilities signify an important ethos of one community driving and sharing responsibility for the management and use of national assets to maximise accessibility, usability and impact.

The data commons will catalogue sources of existing datasets, making them easily accessible to life science researchers. It will support FAIR data management throughout the data lifecycle, from the point of collection to deposition and, crucially, to reuse.

The method commons will enable the collaborative use of shared computational workflows with a national workflow capability. It will feature a national repository of trusted and curated data methods and workflows, contributed by the life sciences research community, supporting reproducible data analytics and advancing

The community centre will provide a focal point for sharing expertise, best practice and troubleshooting within disciplines.

The knowledge centre will enable those driving the collection and curation of existing knowledge resources and training materials to advance best practice in research data management.

Together, the community and knowledge centres will create a collaborative environment that supports more effective dissemination of research data management knowledge and skills across the life sciences research community.

Mission critical 

Put simply, BioFAIR is mission critical to the future of UK life sciences research. At its core the project will deliver major efficiency gains by streamlining research data management.

By better connecting research teams and championing the reuse of data and methods, BioFAIR will help accelerate research, leading to faster scientific breakthroughs as a result.

But BioFAIR adds significantly more value than efficiency alone. It will:

• pioneer innovation, with its state-of-the-art tools and methods paving the way for future scientific success
• future-proof the UK life sciences ecosystem by integrating advanced computational tools and methods to set the stage for new innovations that can be translated and commercialised for maximum impact
• support economic growth and prosperity by upskilling the life sciences research data management workforce and enabling new opportunities for the UK’s scientific leadership

Community driven from the outset, the concept of BioFAIR originated as an idea submitted to BBSRC’s by the ELIXIR-UK team.

This collaborative ethos remains at the heart of BioFAIR, complemented by additional UK and international initiatives to ensure best practices are shared and interoperability across disciplines is promoted.

BioFAIR’s success heavily relies upon the combined ability and proven track record of the UK life science research community in developing and operating research data management tools and services. 

As the awarded hosts of BioFAIR’s coordinating hub, the Earlham Institute’s strengths will be complemented by a skilled and distributed network of UK partners responsible for project leadership and delivery.

Dr Sarah Perkins, Executive Director for Strategic Planning, Evidence and Engagement at BBSRC and the UKRI Senior Responsible Officer for BioFAIR, said: “Digital research infrastructure has fast become as critical to UK bioscience as physical infrastructure. 

“The BioFAIR project will provide the backbone for ground-breaking research, enabling researchers to tackle key societal challenges head-on. By democratising access to crucial data and methods, BioFAIR ensures that the UK life science community can innovate faster and more effectively than ever before.”

Gerry Reilly, Interim Director of BioFAIR, said: “Our vision is to create a powerful federated digital research infrastructure that revolutionises UK life science research. By leveraging established best practices and capabilities, we will build a national platform that ensures the effective adoption of FAIR principles and drives efficiency across all UK life science research institutions. 

“Developed by the research community for the research community, BioFAIR will transform the future face of the UK life sciences.”

or email your questions to info@biofair.uk.

is a spin-out company from the Catalan Institute of Nanoscience and Nanotechnology () and the Catalan Institution for Research and Advanced Studies (), partners of - and supported by - the European Commission’s programme.

INBRAIN’s work involves the decoding of brain signals by implanting innovative, flexible nanoscale graphene electrodes, developed in conjunction with researchers at 91ֱ��’s Nanomedicine Lab and the  (NGI).

These signals may then be used to produce a therapeutic, personalised response for patients with epilepsy, Parkinson’s and other neurological disorders.

This new investment is co-led by Barcelona-based venture capitalists Asabys Partners and Alta Life Sciences, joined by: Vsquared Ventures, a deep tech-focused early-stage venture capitalist based in Munich; TruVenturo GmbH, Germany’s most successful internet company builders; and CDTI, the Spanish Ministry of Science and Innovation.

Fruits of long collaboration

Kostas Kostarelos, Professor of Nanomedicine at The University of Manchester , the NGI and co-founder of INBRAIN Neuroelectronics, said: ‘’This investment for INBRAIN is a testament that graphene-based technologies and the properties of 2D materials have a unique set of propositions to offer for clinical medicine and the management of neurological disorders.

“This did not happen suddenly, though, or by a stroke of good luck in the lab,” he added. “It is the culmination of many years of persistent and consistent work between at least three research institutions, one of which is the Nanomedicine Lab in 91ֱ��, the other two in Barcelona, all working closely and cooperatively under the critically important funding of the Graphene Flagship project.”

The Graphene Flagship is the European Commission’s €1bn research funding spearhead and a key partner of ICN2, ICREA and Graphene@91ֱ��, with a mission is to accelerate advanced 2D materials research and commercialisation.

High costs of brain disease

The high incidence of brain-related diseases worldwide and their huge annual cost - around £700bn in Europe alone, according to a 2010 study by the European Brain Council - call for greater investments in basic research in this field, with the aim of developing new and more efficient therapeutic and diagnostic tools.

Existing brain interfaces are based on metals such as platinum and iridium, which significantly restrict miniaturisation and signal resolution, and are therefore responsible for considerable side effects.

As a consequence, there is a 50% rejection rate of these implants in candidate patients. INBRAIN Neuroelectronics has a disruptive technology proposition, based on the novel material graphene, that overcomes the current limitations of metal-based neural interfaces.

Graphene electrodes allow miniaturisation to nanoscale, with the potential to reach single-neuron resolution. The extraordinary properties of graphene - which is light, biocompatible, flexible and extremely conductive - are harnessed in much smaller devices, which are safer to implant and can be programmed, upgraded and recharged wirelessly.

Driven by artificial intelligence, the implant can learn from the brain of the specific patient and trigger adaptive responses to deliver a personalised neurological therapy. In addition, the use of big data management will permit remote monitoring of the device and data processing.

Better patient outcomes

Carolina Aguilar, founder and CEO of INBRAIN (pictured centre with team, above), said: “Patients with chronic conditions are alone with their diseases, at most they see their physician 1-4 times per year for a follow-up. With less invasive and more intelligent neuroelectronic therapies, we aim to provide safer and real-time adaptive therapies to empower them and improve the outcomes that matter to them.

“This way patients can better deal with their condition between follow-up visits, by getting the right therapy and support when they need it.”

The technology has already been validated in vitro and in vivo, with extensive biocompatibility and toxicity tests mainly performed in 91ֱ�� using preclinical models. This significant investment will be dedicated to bring the technology to human patients, with the execution of multiple clinical trials in collaboration with key neurosurgical and neurological groups in Europe, including various NHS hospitals.

 is one of The University of Manchester’s  - examples of pioneering discoveries, interdisciplinary collaboration and cross-sector partnerships that are tackling some of the biggest questions facing the planet. #ResearchBeacons

A new study reveals Italian-speaking children pick up some language rules faster than English-speakers.

Learning a language is more than just knowing the words – it’s knowing the rules too. A new paper from researchers at has investigated the speed at which Italian-speaking infants are able to pick up rules around singular and plural forms of words. They found that infants had learnt this distinction by 12 months.

The study is published in the journal

In contrast, previous research on this subject has found that English-speaking infants begin to understand singular/plural distinction at around 20 months.

The reason for this might be that the rules for pluralising words in Italian make more sense than those in English. For instance, changing “the yellow giraffe” to “the yellow giraffes” in Italian requires changes in three spots (“la giraffa gialla” becomes “le giraffe gialle”). While this might seem more complex than English, consider goose/geese versus moose or sheep!

In addition, the greater number of changes required between singular and plural in Italian may help reinforce the rule, improving how quickly it is learned.

“We know that all languages have words and that word learning seems to happen in the same way across the world.” Said Dr Alissa Ferry, one of the researchers involved in the study.

She continued; “But all languages have different rules about how words are put together and when children start to figure out those rules does seem to vary depending on the language.”

To investigate the speed at which infants pick up the Italian rules, the researchers had children aged either 12-, 18- or 24-months play a game. They were shown two pictures on a computer screen with either one or two people in each picture and then heard the word for singular (for example, la bambina, the girl) or plural (le bambine, the girls).

How long the infants spent looking at the correct picture was used to gage their understanding of language rules.

The results showed that, by 12 months, the Italian infants could correctly distinguish between girl or girls, depending on which form they heard. This means they were able to pick up the rules around eight months earlier than their English-speaking counterparts.

“We know that all languages have different rules, and these findings show that those rules can shape when the infants start to figure them out,” Said Dr Ferry, adding; “Rules that are harder to find, like the English plural system, take a bit longer to figure out that rules that are easy to find, like the Italian plural system.”

“Twelve to 24-month-olds can understand the meaning of morphological regularities in their language” is published in Developmental Psychology

14 researchers from are some of the most highly cited in their field, in a new list from the released this week.

They include Prof Sir Andre Geim and Prof Sir Kostya Novoselov, the co-discovers of graphene at the University in 2004, for which they won the Nobel Prize for Physics in 2010. Also on the list is fellow graphene researcher, Prof Irina Grigorieva, as well as Prof Jorgen Vestbo, a researcher in respiratory medicine, and Prof Frank Geels, and expert in energy and sustainability.

The list identifies scientists and social scientists who produced multiple papers ranking in the top 1% by citations for their field and year of publication, demonstrating significant research influence among their peers.

The methodology that determines the who’s who of influential researchers draws on the data and analysis performed by bibliometric experts from the Institute for Scientific Information at the Web of Science Group.

The data are taken from 21 broad research fields within Essential Science Indicators, a component of . The fields are defined by sets of journals and exceptionally, in the case of multidisciplinary journals such as Nature and Science, by a paper-by-paper assignment to a field based on an analysis of the cited references in the papers. This percentile-based selection method removes the citation advantage of older papers relative to recently published ones, since papers are weighed against others in the same annual cohort.

Listed University researchers;

Prof Sir Andre Geim, Dr Artem Mischenko, Prof Christian Klingenberg, Prof David Denning, Dr Donald Ward, Prof Frank Geels, Prof Irina Grigorieva, Prof Jorgen Vestbo, Prof Judith Allen, Prof Sir Kostya Novoselov, Prof Rahul Nair, Prof Richard Bardgett, Dr Roman Gorbachev, and Prof Zhiguo Ding.

A brave new world of immune system therapies – harnessing the body’s own defences – could help treat all kinds of different illnesses, according to one of the UK’s leading immunologists.

But in his new book The Beautiful Cure: Harnessing your body’s natural defences, Professor Daniel M. Davis, from The University of Manchester, says this also raises vital new issues for society, not least being how we cope with the expense of new medicines.

The book, published by Random House, describes the scientific quest to understand how the immune system works, and how it is unlocking a revolutionary approach to our fight with disease.

American immunologist Professor James P. Allison, he writes, was one pioneer who, by studying how our immune system works, has found a way to help treat some types of cancer.

The journey to a modern understanding of the immunity can be said to have begun in 1989, when Charles Janeway expanded our understanding of innate immunity, the body's first line of defence against infection.

Then followed a global adventure of digging into cells and molecules, leading to discoveries about how immune cells switch on and off in their fight with disease.

“Take one example,” Davis says: “Immunologists have learnt how to switch off a brake on the immune system – to unleash its power more forcefully in fighting cancer.”

“Another example is how anti-TNF therapy was developed for arthritis and inflammatory bowel disease.

“But these successes are probably still just the tip of the iceberg. All kinds of different diseases could feasibly be tackled with immune system therapies: cancers, viral infections, arthritis, and a range of other conditions.

“There are many other break receptors in the immune system which can switch off specific types of immune cells. We must now test whether or not blocking these, alone or in combination, can unleash immune cells to tackle different types of disease.”

He added: “We also know that stress has a vital influence on the immune system. This raises crucial questions about whether practices that reduce stress, like tai chi and mindfulness, can help our fight with disease.

“A new detailed knowledge of how our immune system works has unlocked a revolutionary new approach to medicine and well-being.”

A scientist and historian from The University of Manchester has unearthed a document which throws light on a long-forgotten controversy in scientific publishing from the 1960s.

Writing in the journal PLOS Biology, Professor Matthew Cobb tells how he discovered the document – a letter to Francis Crick – in the archives of Cold Spring Harbor Laboratory, New York.

The letter invited Crick to join an experiment designed to encourage information sharing for biologists, which was called the ‘Information Exchange Groups’. Crick initially declined, although he eventually changed his mind.

The system,which was forced to shut down in 1967 following opposition from the established publishers, eventually involved over 3,600 researchers around the world and saw the production of over 2,500 different preprints, on millions of pages of paper.

Scientists have long complained it can take months or even years for a scientific discovery to be published, because of the slowness of peer review.

To cut through the problem, researchers in physics and mathematics now use “preprints” – preliminary versions of their scientific findings published on internet servers for anyone to read.

But it wasn’t until In 2013 that biologists were able to share preprints in this way.

The growth in the Information Exchange Groups and their possible extension into physics provoked systematic opposition from journal publishers such as Nature and Science.

Vitriolic editorials were published as a number of journals refused to consider articles that had been circulated as preprints. That helped put an end to the Information Exchange Group experiment.

The widespread circulation of preprints in physics really took off in the 1990s with the appearance of the World Wide Web and a server called arXiv.

Biology continued to lag behind, and a further attempt to launch preprints in 1999 met with similar hostility from publishers and learned societies and was soon abandoned.

It is only recently that biology preprints have been widely accepted by scientists and by journals.

Professor Cobb said: “Although well-intentioned, the Information Exchange Groups became hugely controversial.

“Journal publishers claimed only they were able to guarantee the accuracy and probity of scientific findings, and that the widespread adoption of preprints threatened their existence.

“But I suspect the real issue was the potential threat to publishers’ income and prestige.”

He added: “Scientists are completely unaware of this controversy, but it illustrates how some vested interests have opposed the open circulation of knowledge in the name of money and prestige.

“It also shows how even old-style technology was able to bypass the traditional gate-keepers of science and the barriers they created.”

Whales and dolphins (Cetaceans) live in tightly-knit social groups, have complex relationships, talk to each other and even have regional dialects – much like human societies.

A major new study, published today in (Monday 16^th October), has linked the complexity of Cetacean culture and behaviour to the size of their brains.

The research was a collaboration between scientists at The University of Manchester, The University of British Columbia, Canada, The London School of Economics and Political Science (LSE) and Stanford University, United States.

The study is first of its kind to create a large dataset of cetacean brain size and social behaviours. The team compiled information on 90 different species of dolphins, whales, and porpoises. It found overwhelming evidence that Cetaceans have sophisticated social and cooperative behaviour traits, similar to many found in human culture. The study demonstrates that these societal and cultural characteristics are linked with brain size and brain expansion – also known as encephalisation.

The long list of behavioural similarities includes many traits shared with humans and other primates such as:

• complex alliance relationships – working together for mutual benefit
• social transfer of hunting techniques – teaching how to hunt and using tools
• cooperative hunting
• complex vocalizations, including regional group dialects – ‘talking’ to each other
• vocal mimicry and ‘signature whistles’ unique to individuals – using ‘name’ recognition
• interspecific cooperation with humans and other species – working with different species
• alloparenting – looking after youngsters that aren’t their own
• social play 

Dr Susanne Shultz, an evolutionary biologist in 91ֱ��’s School of Earth and Environmental Sciences, said: “As humans, our ability to socially interact and cultivate relationships has allowed us to colonise almost every ecosystem and environment on the planet. We know whales and dolphins also have exceptionally large and anatomically sophisticated brains and, therefore, have created a similar marine based culture.

“That means the apparent co-evolution of brains, social structure, and behavioural richness of marine mammals provides a unique and striking parallel to the large brains and hyper-sociality of humans and other primates on land. Unfortunately, they won’t ever mimic our great metropolises and technologies because they didn’t evolve opposable thumbs.”

The team used the dataset to test the social brain hypothesis (SBH) and cultural brain hypothesis (CBH). The SBH and CBH are evolutionary theories originally developed to explain large brains in primates and land mammals.

They argue that large brains are an evolutionary response to complex and information-rich social environments. However, this is the first time these hypotheses have been applied to ‘intelligent’ marine mammals on such a large scale.

Dr Michael Muthukrishna, Assistant Professor of Economic Psychology at LSE, added: “This research isn’t just about looking at the intelligence of whales and dolphins, it also has important anthropological ramifications as well. In order to move toward a more general theory of human behaviour, we need to understand what makes humans so different from other animals. And to do this, we need a control group. Compared to primates, cetaceans are a more “alien” control group.”

Dr Kieran Fox, a neuroscientist at Stanford University, added: “Cetaceans have many complex social behaviours that are similar to humans and other primates. They, however, have different brain structures from us, leading some researchers to argue that whales and dolphins could not achieve higher cognitive and social skills. I think our research shows that this is clearly not the case. Instead, a new question emerges: How can very diverse patterns of brain structure in very different species nonetheless give rise to highly similar cognitive and social behaviours?”

Reference: 'The social and cultural roots of whale and dolphin brains, Nature Ecology & Evolution (2017). '

A new study finds that unlike humans, Chimpanzees are entirely selfish creatures who act in their own interests, turning conceived wisdom on its head.

The team from the universities of Manchester, Birmingham, and St Andrews, and the Max Planck Institute for Evolutionary Anthropology, say humans were unlikely to have inherited the trait of kindness from their primate cousins.

The animals, they find, are unlikely to take an interest in each other unless there is an anticipated benefit.

Previous research implying helpful behaviour in chimps was likely to be a by-product of the way experiments were designed, they argue in .

The team worked with a group of 16 Chimpanzees at the , in Uganda.

Using two ingenious experiments, the team discovered the chimpanzees were no more likely to help feed each other as they were to block access to a box of peanuts.

One of the principle investigators, at The University of Manchester, said, “The evolution of social behaviour, and what drives individuals to act altruistically, is an important and active area of debate.

“There has been an appealing suggestion that the roots of human altruism extend down at least as far as our common ancestor with chimpanzees.

“However, the results of this study challenges that view. ‘Helping’ might have formerly arisen in previous studies as a by-product of interesting tasks.”

Dr Claudio Tennie, from the University of Birmingham, said: “The results of these experiments combined demonstrate that the chimpanzees did not act in a manner that would produce benefits for others in a task where there was no perceived benefit to themselves.

“Indeed, given that the participants were just as likely to prevent access to food as they were to permit access, chimpanzees are no more altruistic than they are spiteful.

“Even after they demonstrated a clear understanding of the consequences of their actions, they remained indifferent to any effects these actions may have on others. If true, this would mean that prosocial behaviour has developed late in evolution, after our split with the other apes.”

For the first time, the precise way organs and tissue develop in early pregnancy has been mapped – providing new insight into how developmental disorders can occur during the crucial first two months of pregnancy.

The research, carried out by a team from the University of Manchester and Central 91ֱ�� University Hospitals NHS Foundation Trust, is published in the current issue of the journal eLIFE.

Concentrating on the period know as organogenesis – the point at which organs and tissue develop out of the newly established germ layers of ectoderm, mesoderm and endoderm – the study provides the very first insight into the human embryo development.

The computational model developed during the study has not only correctly identified many genes that were already known to cause developmental problems when faulty, but more importantly it discovered many other genes which could lead to developmental defects in an unborn child. The identification of these potentially damaging genes has led the research team to believe that not only genetic diagnoses will improve in terms of identifying development disorders at an earlier stage but also the potential to treat these disorders in utero.

Neil Hanley, Professor of Medicine at The University of Manchester’s School of Medical Sciences and lead author of the report, said the research findings will give a major boost to the understanding of early human development.

“Until now, remarkably little has been known about organogenesis – the assembly phase for human organs and tissues. Any errors in this stage of development can result in miscarriage or serious birth defects. Our research has opened a window to this crucial phase, and hopefully will allow the scientists who follow us to develop methods to eliminate or significantly reduce organ and tissue disorders.”

Working on the established knowledge that genes control the way organs and tissues develop – certain genes switch on or off in complex patterns, downloading DNA code into RNA molecules - the team used RNA-sequencing to identify the RNAs instrumental in organ development.

The computational model developed by the team used the identified organ-developing RNAs to decode the precise patterns of gene activity in the tissues. The sequencing uncovered both normal patterns leading to healthy development as well as a significantly large and previous unknown number of genes which lead to defective development.

Academics at The University of Manchester have dismissed the long-held argument that the ancient Egyptian queen Cleopatra was killed by a snake bite. 

Andrew Gray, Curator of Herpetology at 91ֱ�� Museum, says venomous snakes in Egypt -  Cobras or Vipers - would have been too large to get unseen into the queen’s palace.

He was speaking  to Egyptologist Dr Joyce Tyldesley in a new video which is part of a new online course introducing ancient Egyptian history, using six items from the Museum’s collection.

According to Dr Tyldesley, the ancient accounts say a snake hid in a basket of figs brought in from the countryside, and was also used to kill one or two of her serving maids.

But according to Andrew Gray, Cobras are typically 5 to 6 feet long but can grow up to 8 feet – too big to hide very easily.

There would also be too little time to kill 2 or 3 people-  because snake venom kills you slowly-  with in any case only a 10 per cent chance of death.

He said: “Not only are Cobras too big, but  there’s just a 10 per cent chance you would die from a  snake bite: most bites are dry bites that don’t inject venom.

“That’s not to say they aren’t dangerous: the venom causes necrosis and will certainly kill you, but quite slowly

“So it would be impossible to use a snake to kill  2 or 3 people one after the other. Snakes use venom to protect themselves and for hunting – so they conserve their venom and use it in times of need.”

Cleopatra is strongly associated with snakes, like many ancient Egyptian kings and queens of Egypt. In addition, Cleopatra also believed she was the embodiment of the Goddess Isis, who can take on the form of a snake.

Dr Tyldesley, who’s book Cleopatra: Egypt's Last Queen was a BBC Radio 4 book of the week, says one aspect of the accounts has proved to be correct. The ancient Egyptians believed snakes were good mothers.

“Very few snakes have a maternal instinct. However, the cobra is an exception: they sit on the nest and protect them until they hatch. So in this case, it seems the Egyptians were right,” agrees Dr Gray

The free Massive Open Online Course (MOOC), ‘A History of Ancient Egypt’, launches on 26 October.

Dr Tyldesley added: “The MOOC includes behind-the-scenes access at the Museum and detailed descriptions of many objects from our Egypt and Sudan collection.”

Researchers in 91ֱ�� have demonstrated for the first time the relative safety and effectiveness of treatment, eltrombopag, in children with persistent or chronic immune thrombocytopenia (ITP), as part of an international duo of studies. 

The results of the studies conducted in 91ֱ�� by the ITP Centre, in partnership with the (CRF), both based at Royal 91ֱ�� Children’s Hospital were published in and .

ITP is an autoimmune disorder where the immune system attacks platelets and blood fails to clot as it should.  Four in every 100,000 children develop the disorder each year globally.  The symptoms of ITP include bleeding and bruising more easily.  Frequent nose bleeds and bleeding from the gums can be common, and bruising often appears as purple patches or tiny red spots on the skin. On rare occasions bleeding can be life threatening.

The condition may resolve by itself, but for one in every four of the affected children, the condition becomes chronic persisting after primary intervention and lasting for more than twelve months.  For eight-year-old George, a patient from Royton near Rochdale who was involved in the study, having chronic ITP meant frequent visits to A&E for nose bleeds that wouldn’t stop and he would also miss out on social activities.

George’s mum, Joanne, explains: “George was diagnosed when he was four years old. I’d find myself chasing after George trying unsuccessfully to wrap a very active little boy in cotton wool.  On this new treatment, George can make the most of his childhood and he recently celebrated his eighth birthday with friends in style at a trampoline park.”

Historically, second-line treatment options for children with ITP have been scarce and, one of the earliest options, surgical removal of the spleen (splenectomy) was associated with a high risk of sepsis and thrombosis.  A better understanding about the underlying cause of ITP led to the development of the use of newer immunosuppressant agents, including rituximab.  More recently, thrombopoietin receptor agonists (e.g. eltrombopag) have been approved for the treatment of thrombocytopenia in adult patients with chronic ITP who have had an inadequate response or are intolerant to other treatments.

Chief investigator, , Consultant Paediatric Haematologist and Honorary Lecturer at The University of Manchester, explains: “The studies, funded by , provide clinicians with much needed evidence to help decide when eltrombopag would benefit paediatric patients and provide dosage regimens suitable for paediatric patients. 

“The studies demonstrate that eltrombopag is well tolerated and effective, consistently stabilising the platelet count to over 50 X 10⁹ per litre within 2–6 weeks for 40 per cent of children receiving the treatment, compared with 0 per cent on the placebo arm.”  

Life Sciences Minister George Freeman MP said: “This important research has the potential to bring real benefits to children suffering from rare and life threatening bleeding disorders. By investing more than £1 billion a year through the National Institute for Health Research we are giving our world leading life sciences sector the ability to trial the latest 21st century medical breakthroughs.”

Professor Nick Webb, Director of the NIHR / Wellcome Trust 91ֱ�� CRF adds: “The first ever patient to be recruited to a clinical trial at our clinical research facility in 91ֱ�� was also the first patient globally to be recruited into the ITP study.  This research will enable patients to be more actively involved in school and social activities, and is also an historical milestone for the clinical research facility – affirming the value of having a dedicated children’s research facility to test and tailor treatments to children’s needs.” 

On the back of these studies eltrombopag was licenced by the FDA for use in children >1 year old.  EU licencing for this application is currently in progress.