Making a difference: Impact report 2017

Case Studies

Chapter Nine

Case Studies


Pharmacy Research UK

A review of the prescription and use of unlicensed medicines highlighted a lack of understanding about the use of these treatments and suggests new guidance is needed to protect both pharmacists and patients.

Unlicensed medicines (ULM) are medicines which have not been granted approval for use by the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK. Despite this lack of licencing they are frequently prescribed to patients, but MHRA guidance states this should only be done when no licenced product is available. The need to use a ULM is often because a patient requires an altered dose to the standard prescription for example children’s dosages, liquid forms of drugs for those that are unable to take solid doses and revised formulas for patients allergic to an ingredient in the licenced drug. ULM can also be medicines that are licenced in countries other than the UK and products classed as food supplements.

In 2014 Pharmacy Research UK collaborated with the UK Clinical Pharmacy Association to fund Gemma Donovan and colleagues at the University of Sunderland who were keen to investigate the use of ULM. The project explored the use of ULM in primary and secondary care, from the perspective of prescribers, pharmacists and patients and also included a review of the guidance on the use of ULM. There was a need to understand what influences the decision to prescribe and use unlicensed medicines and what checks and balances are in place to ensure they are used appropriately. It was also unclear what information patients are given about unlicensed medicines, what their understanding is around these medicines, and how they feel about using them. In late 2015 the team published a report in the British Journal of General Practice, which highlighted the importance of the research in this area.

The project results indicated inconsistencies in the use of ULM. Guidance was lacking, with no single clear strategy in place to ensure ULM are used appropriately. Many prescribers were unaware of the licencing status of the medicines they were prescribing so were not taking this into account in their decision making. Because of their unlicensed status liability for any adverse outcomes from the use of ULM remains with the prescriber and dispensing pharmacist.Pharmacy Research UK The lack of clear guidance is putting both these healthcare professionals and patients at risk. Patients themselves are generally unaware of these risks as they are often not informed that they have been prescribed a ULM and so are unaware of the implications that might have. It is hoped that the results of the research will lead to the development of training materials for healthcare professionals, information for patients, and clearer guidance on the use of unlicensed medicines. It may also inform the design of future interventions to deliver safe and effective use of these treatments.

“This project has provided a foundation for future research in this area, and shown what improvements might be needed to how we manage unlicensed medicines and the potential role of pharmacists. The Clinical Pharmacy Research Grant has not only allowed me to complete a really worthwhile study into unlicensed medicines, it’s also kick started my research career. I’ve been able to access training opportunities to develop my research skills and network with other researchers both inside and outside of the pharmacy profession.” Researcher Perspective



Leukaemia and Lymphoma NI

Repurposing existing drugs allows new treatments for disease to be brought to patients much more quickly and this approach is being successfully used by researchers funded by Leukaemia and Lymphoma Northern Ireland

Leukaemia is a cancer of the white blood cells and there are many different types depending on which type of white blood cell is affected and the speed of progression of the disease. Acute Myeloid Leukaemia (AML) starts in the bone marrow (the soft inner part of the bones, where new blood cells are made), but in most cases it quickly moves into the blood. “Acute” means that the leukaemia can progress quickly, and if not treated, would probably be fatal in a few months. It often develops from a set of pre leukaemia conditions called Myelodysplastic Syndromes (MDS), a diverse collection of conditions united by ineffective production of myeloid blood cells.

MDS and AML are predominantly diseases of the elderly with the median age of diagnosis being around 71 years old. Treatments do exist but elderly patients (older than 60 years) do not respond to therapy as effectively as younger patients with a similar type of disease and are often less able to tolerate intensive chemotherapy protocols. Leukaemia and Lymphoma NI are funding Professor Ken Mills at Queen’s University Belfast to try and identify different, and less intensive, therapies that may be beneficial particularly in the vulnerable elderly patient population.

In 2015 Professor Mills and his team published a paper showing that they had identified an existing licensed drug as a potential new treatment for MDS and AML. The team showed it was very effective against MDS and AML cell lines and patient samples, with no effect on normal bone marrow, and also worked in synergy with current treatments.Leukaemia and Lymphoma NI Clinical trials still need to be done to show whether the drug is truly effective in patients, but the team feel it has the potential to improve the quality of life and longer-term outcome for those MDS and AML patients for whom high-intensity chemotherapy regimens are not an option. Estimates suggest that on average it takes over 12 years to bring a new drug to market at a cost over £1 billion. Repurposing an existing licensed drug to treat a new condition in this way eradicates the majority of that time and financial burden making it a highly attractive and efficient approach to developing new treatments. Drug repurposing strategies like the one used in this project are now being used to try and identify new treatment options across the spectrum of human health conditions.

“This type of research into repurposed drugs has the potential to have an impact across cancers and many other types of diseases. The benefits to the patient are that more effective therapies become available whilst the cost the health care provider is reduced. The funding from LLNI has been essential in enabling this research to be undertaken and has been used as a springboard for our other studies, in Belfast, on repurposed drugs” Researcher Perspective



Action for A-T

Developing of the first mouse model of Ataxia Telangiectasia that mimics the neurological features seen in patients has allowed researchers to drive forward research into the causes of the disease.

Ataxia Telangiectasia (A-T) is a rare, genetic degenerative disease of childhood, which affects multiple systems of the body including the nervous and immune systems. One of the aspects that has the greatest effect on a patients quality of life is a loss of motor coordination (ataxia), which generally leads to children being confined to a wheelchair and needing assistance with everyday tasks by around age 10. There is currently no cure for A-T and no treatments exist to slow the progression of this devastating disease.

Developing treatments for any condition hinges on scientists having the tools in place to investigate what is going wrong. Mouse models are one of a range of vital tools researchers use to investigate human diseases when there are no viable alternatives.Action for A-T It’s essential that the model accurately mimics the aspect of the human condition being studied but existing models for A-T were failing to show the neurological components of the condition, blocking urgently needed research from progressing. Recently Action for A-T funded researchers have successfully completed a project to develop a new mouse model, which does have the neurological hallmarks of A-T.

This is a huge step forward for A-T researchers, who can now use this tool to investigate the underlying causes of the ataxia seen in A-T. The scientists who developed the tool have already used it to show that cells in specific regions of the brain are firing irregularly in A-T, which they believe may be a cause of the ataxia seen in these mice and possibly also in patients. Advancing understanding of the root causes a disease in this way is the first crucial step towards treatments and ultimately cures.



Parkinsons UK

The global Parkinson’s research community has access to a continually updated database describing the function of all the genes shown to be linked to the development of the condition.

Every hour, someone in the UK is told they have Parkinson’s, a degenerative neurological condition, which currently has no cure. The main symptoms of the condition are tremor, slowness of movement and rigidity. It affects 127,000 people in the UK, around one in 500 of the population. It is not yet known exactly why most people get Parkinson’s, but researchers think that in the vast majority of people a combination of genetic, lifestyle and environmental factors lead to its development. Many genes have now been identified that may affect how likely it is for an individual to develop Parkinson’s.

In 2014, Ruth Lovering and colleagues from UCL were awarded a project grant by Parkinson’s UK to create a database summarising the cellular roles of the human genes that may contribute to Parkinson’s. Using information gathered from scientific papers, the genes linked to Parkinson’s are ‘annotated’ using a defined vocabulary of terms (Gene Ontology) which can be ‘read’ by computers. These annotations describe gene functions and the biological processes they contribute to, making this information searchable.

Parkinsons UK  The team releases their annotations each month and they are freely available. The annotations are shared through initiatives such as the Gene Ontology (GO) Consortium dataset – a bioinformatics initiative that consolidates information about gene products and their biological functions. The sharing of the annotations across all the major online biological knowledge bases ensures high visibility of the gene annotations allowing researchers worldwide to quickly find information relevant to their work. The team hopes that researchers will use this resource to help identify additional genes, find potential targets for treatment and develop tests to identify people at risk of Parkinson’s.

“Our team hopes that researchers will use our database of described genes to help identify additional genes, find potential targets for treatment and develop tests to identify people at risk of Parkinson’s.” Researcher Perspective



Asthma UK

Research into the support of self-management in Asthma has contributed to the establishment of Spoonful of Sugar, a service that’s helping ensure patients use the medicines they are prescribed in the most effective way.

Every ten seconds in the UK someone has a potentially life threatening asthma attack. Not all of these are preventable but many could be avoided with better self-management by patients, including regular use of prevention inhalers. In 2010 Asthma UK funded Professor Robert Horne at the UCL to investigate why patients often fail to properly control their Asthma, building the evidence which would allow effective interventions to be developed to support self-management.

Asthma UK	 Failure to take prescribed medications is a problem across the spectrum of disease not just for Asthma, as it is thought that between a third and a half of all medicines prescribed for long‑term conditions are not taken as recommended. In 2011 Professor Horne began tackling this widespread issue, taking the knowledge he gained from projects such as that funded by Asthma UK and founding Spoonful of Sugar, a consultancy supporting healthcare providers and the pharmaceutical industry to develop patient-centred support materials facilitating optimal use of medicines.

SoS won both Small Consultancy or Health Team of the Year and Specialist Consultancy of the Year at the 2015 Communiqué Awards which reward excellence and best practice in healthcare communications. Communiqué judges commented that “Spoonful of Sugar’s behavioural change is steeped in academia to provide credible, measureable outcomes and provides an industry changing model”



National Osteoporosis Society

A novel assay is allowing researchers to identify osteoporosis patients that have a newly discovered autoimmune form of the condition that may require different treatment strategies.

Osteoporosis is a condition which weakens bones so they break easily and it changes people’s lives. It occurs when the struts that make up the inside of our bones become thin, causing them to become fragile and break easily following a minor bump or fall. Every year, people in the UK suffer more than 300,000 fragility fractures,. One in two women and one in five men over the age of 50 will break bones as a result of poor bone health. In all of us bones begin to thin in later life as the amount of bone produced falls below the level of the amount of new bone being made. The majority of osteoporosis develops due to a combination of environmental and genetic factors exacerbating this natural bone thinning. Drug treatments and lifestyle changes are both currently used to decrease the risk of fractures in people diagnosed with osteoporosis.

National Osteoporosis SocietyResearchers funded by the National Osteoporosis Society have recently discovered a novel cause of osteoporosis in which bones are attacked by a person’s own immune system. Patients with this autoimmune for osteoporosis have raised levels of antibodies against a protein called osteoprotegerin. In healthy bones, osteoprotegrin acts to increase bone mineral density and bone volume. An increase in antibodies against osteoprotegerin blocks this process leading to bone thinning. At the start of this study there was no reliable way to assess a patient’s level of osteoprotegerin antibodies so the researchers developed an assay known as an ELISA which can quickly and accurately make these measurements.

The ELISA assay is being used to study a cohort of over 1500 patients with osteoporosis to establish the prevalence of autoimmune osteoporosis in the population. If a significant percentage of those with osteoporosis do have raised osteoprotegerin antibody levels this could have a significant impact on both diagnostics and treatment strategies and the ELISA assay may become a key part of the diagnostic pathway for all patients suspected to have osteoporosis. This would allow patients to be stratified for more personalised treatment based on the specific form of the disease they have, which is both more cost effective and, more importantly, much more beneficial to patients.

“Getting funding for research that translates new disease understanding into new tests or treatments is becomingly increasingly hard to come by, with many major funding agencies moving away from this kind of research altogether. Funding from charities such as the National Osteoporosis Society has been vital in ensuring that this work continues” Researcher Perspective



Ovarian Cancer Action

A clinical trial for a new drug combination was shown to restore response to treatment in a subset of patients with recurring ovarian cancer that were not responding to standard treatments.

Ovarian Cancer is the sixth most common cancer in women with around 7,000 new cases diagnosed in the UK each year. Most women with ovarian cancer are initially treated with surgery and/or chemotherapy drugs based on modified forms of platinum. These drugs are initially very effective, in general, but there is still a 70% chance the cancer will come back. When a patient relapses it is common for the tumour to be less susceptible to platinum based therapy. If a patient relapses within six months of their last round of platinum based chemotherapy their cancer is designated platinum resistant which significantly reduces the treatment options available.

Ovarian Cancer ActionIn 2012, Ovarian Cancer Action funded a study into platinum resistance, part of which included a clinical trial with GSK for a new drug treatment for women resistant to platinum chemotherapy. The trial examined a drug called Afuresertib, a cancer growth inhibitor that blocks the signals that cancer cells use to divide and grow. Researchers believed Afuresertib could help restore the response to platinum chemotherapy in resistant patients. Patients whose ovarian cancer recurred within six months of receiving carboplatin and paclitaxel treatment were recruited to the study. In this population only 10-15% of people would be expected to respond to a second course of treatment; however, the addition of Afuresertib was shown to lead to a far higher response rate of 39%.

As a result of the preclinical and clinical data collected during the study, together with the significant improvement in response rate, further discussions around the development of future clinical studies with Afuresertib are ongoing.

As part of the original study, translational research was conducted including the collection of comprehensive tissue and blood samples. Scientists hope that they can utilise this valuable resource towards research that will help predict who may benefit from this combination in order to personalise treatments so they can be individually tailored for women who will benefit most.



Cancer Research UK

BOADICEA software can calculate an individual’s risk of breast and ovarian cancer and is used by clinicians worldwide to help patients understand and manage those risks.

Cancer Research UK funds the Centre for Cancer Genetic Epidemiology at the University of Cambridge, one of the world’s leading centres for research into cancer genetics. The aim of this centre is to identify and understand the genes that predispose people to cancer, calculate the risk that having certain changes in these genes will lead to a person developing cancer and investigate how that may differ in response to a person’s lifestyle or environment.

The team have developed multiple pieces of software based on their research that are now being used in clinical settings to help patients understand their risk of getting cancer. One of the most widely used is BOADICEA (Breast and Ovarian Analysis of Disease Incidence and Carrier Estimation Algorithm). This computer programme allows doctors to accurately calculate the risks of breast and ovarian cancer based on a woman’s family history and genetic background.

Cancer Research UKAs the world’s first polygenic breast cancer risk model, available to the global healthcare community through a web-tool, BOADICEA has influenced the management of breast cancer on a global scale. It is recommended as a risk assessment tool in UK NICE clinical guidelines and is also incorporated in the guidelines of several other countries, including The USA, Canada and Australia for the management of familial breast cancer. BOADICEA is now used by more than 5700 healthcare professionals in more than 60 countries worldwide. The information provided by BOADICEA has allowed thousands of people with a predisposition to cancer to make informed choices about what strategies they should put in place to minimise their chances of developing the disease.

“BOADICEA is the culmination of basic and applied research, based on data from large local and national epidemiological studies. These efforts have been made possible through the long-term support by CR-UK of more than 20 years. BOADICEA is used in clinical practice for identifying women who would benefit from mutation screening in the high-risk genes BRCA1 and BRCA2, from enhanced breast cancer screening, chemoprevention or other interventions” Researcher Perspective



The Brain Tumour Charity

Research shows that a clinical guideline and public awareness campaign has more than halved the time a UK child waits for a Brain Tumour diagnosis.

Each week in the UK, around ten children or teenagers are diagnosed with a brain tumour. A 2006 study by The Children’s Brain Tumour Research Centre, funded by The Brain Tumour Charity, identified that it took a lot longer for children with brain tumours in the UK to receive an accurate diagnosis than in other parts of the world. As a result a new guideline advising healthcare professionals on the identification, assessment and investigation of children presenting with symptoms that could be caused by a brain tumour was developed. An associated awareness campaign, HeadSmart, was also launched which produces a wide array of information materials on brain tumour signs and symptoms for health professionals, parents and the public. The HeadSmart website receives around 13,000 visits per month and the team at The Charity is also actively raising awareness using a variety of social media platforms.

The Brain Tumour CharityThe key aim of developing both the guideline and the HeadSmart campaign was to significantly reduce the time between the onset of symptoms and diagnosis, a measure known as the total diagnostic interval (TDI). In order to help assess whether this aim had been achieved, data on TDI from children and young people newly diagnosed with a brain tumour was collected across 18 centres in the UK.

This data has been analysed to investigate the success of the guideline and campaign. Since the 2006 survey which prompted the development of the new guideline the TDI has been more than halved, from a median of 14 weeks to a median of 6.7 weeks. This huge improvement in the time taken for diagnosis of brain tumours is giving children across the UK the best chance of getting the appropriate treatment in time to make a full recovery. The successful use of this strategy to accelerate brain tumour diagnosis has been acknowledged by a number of NHS and charity awards for excellence. Being able to produce strong quantitative research evidence showing the success of the campaign has led to it being replicated at an international level.



British Lung Foundation

Evidence from a countrywide investigation into respiratory disease was presented to the APPG on respiratory health and was incorporated into the government’s inquiry into respiratory deaths which made many recommendations for improving awareness, research and care.

1 in 5 people in the UK have been diagnosed with a lung disease. These include asthma, chronic obstructive pulmonary disease, lung cancers and many other conditions. 115,000 people a year die from lung disease in the UK which equates to 1 person every 5 minutes. This makes lung disease is one of the biggest killer diseases in the UK. Whilst mortality rates from cardiovascular disease, another of the top killers, have fallen roughly 15% over the last ten years, mortality rates for lung disease have remained static highlighting the need for increased research to improve prevention and treatment.

British Lung FoundationThe statistics above stem from a large scale review on the respiratory health of the nation funded by the British Lung Foundation. The study examined the overall extent and impact of lung disease across the UK and also took a closer look at the impact of 15 major lung conditions. The resulting report, entitled the Battle for Breath, was made publicly available and has an accompanying website that gives a clear overview of all the key facts and figures.

Data from this study was presented directly to the government’s All Party Parliamentary group (APPG) on Respiratory Health by the lead researcher Professor David Strachan. Professor Strachan’s evidence fed into a 12 month enquiry the APPG was conducting on respiratory deaths. The inquiry asked about why premature mortality from respiratory disease remains so high and how death rates could be improved. The final report stemming from the enquiry made a number of key recommendations including increased investment into respiratory medical research, so that it is proportional to the burden posed by respiratory diseases, lung function testing to be included as part of the NHS Health Check for everyone over 40 and an awareness campaign on the symptoms of lung disease for the public and healthcare professionals.



Great Ormond Street Hospital Childrens Charity

An evaluation of long term outcomes for children born with specific congenital heart defects fed into an ongoing NHS England review of national children’s heart services

Congenital heart disease (CHD) affects approximately 5600 children born annually in England and Wales. CHD encompasses a wide range of heart conditions, some of which are very complex. These complex cases often require a number of procedures and operations. While much is known of the immediate and short term impact and outcomes of these interventions, there is a lack of corresponding information relating to the long term outcomes for patients. Further understanding of these long term outcomes is of great importance to patient families and could influence decision making about the management strategies used for patients.

Researchers at Great Ormond Street Hospital obtained funding from Great Ormond Street Hospital Children’s Charity and the NIHR to undertake research in this area using British national audit data and information from patients treated at Great Ormond Street Hospital.Great Ormond Street Hospital Childrens Charity As part of this work they have created a new analysis method that links all of the separate procedure records from each individual patient and then groups together all the patients based on their underlying congenital heart condition. This approach enables assembly of a complete picture of what happened to all the patients with one particular congenital heart disease.

Their work has also already begun to influence policy as a publication from year one of the study has been used by NHS England as a reference document during the ongoing review process for children’s heart services nationally. This paper showed that, early outcomes (30 days after intervention) for patients have improved, however the ‘case mix’ has become more complex over time, meaning that longer term outcome monitoring has become all the more important. The work has contributed to subsequent research projects funded by NIHR including one project on outcomes of infant heart disease and one ongoing study of important early morbidities linked to paediatric cardiac surgery.

The work has also been influenced by the partnership that the research team set up with the main patient and family user group at Children’s Heart Federation (CHF). The user group has provided valuable input into research materials such as information sheets and they have contributed to the direction of the research.



Alzheimer’s Society

A project designed to improve the early identification of dementia in the signing Deaf community catalyzed the formation of a targeted clinic specifically for this patient population alongside a training programme for healthcare professionals and updated Department of Health guidance.

There are many challenges associated with identifying and diagnosing dementia and these are more complex in the Deaf community where conventional testing is not possible and access to information and knowledge about dementia is poor. In 2010 Alzheimer’s Society funded a project, led by the University of Manchester, on overcoming the obstacles to the early identification of dementia in the signing Deaf community. This was run in partnership with City University London, (DCAL) University College London and the Royal Association for Deaf People.

Alzheimer’s SocietyThe project was the first of its kind and made important steps to improve the early identification of dementia in people who communicate through British Sign Language (BSL). Through Deaf-led consultation, the project identified culturally-tailored approaches to increasing knowledge and awareness about dementia that went beyond simple translation into BSL of pre-existing information. For the first time, Deaf people with a diagnosis of dementia have described their experiences, strategies for living well and best practice in their support. This work has been further developed into a sign-bilingual information hub with their stories re-told by Deaf actors and additional information conveyed in BSL. Another strand of the project created the first cognitive, language and memory assessment tools that use BSL.

The project has acted as a catalyst for establishing a dementia clinic specifically aimed at supporting Deaf people, the first of its kind anywhere in the world. It has spawned a training programme for health professionals and contributed guidance to the Department of Health. The project has highlighted the importance of increased dementia awareness within the Deaf community to address stigma, loss of cultural affiliation and to make the community more dementia friendly. The research team achieved further funding from several sources to develop this work to make neighbourhoods more dementia friendly, to increase the involvement of Deaf carers and people living with dementia in policy and practice, to develop a Deaf cultural life-story telling programme and to train psychologists across the UK to deliver the new BSL cognitive assessments.



Marie Curie

Research funded by Marie Curie led the Scottish Government to fast track support plans for individuals acting as carers for friends or relatives with a terminal illness.

Around 1 in 10 people in the UK are acting as carers for family members or friends and 20% of these carers have to give up work as a result. As the age of the general population increases these numbers will rise. Care provided by family or friends reduces pressure on the health service but acting as someone’s carer is both physically and psychologically demanding and carers regularly experience poor health themselves. This is especially true for those caring for people with a terminal illness who face the imminent loss of the cared for person. Support for carers is available but carers are often ambivalent about their own needs and are reluctant to identify themselves as a caregiver in need of support.

Funded by the Dimbleby Marie Curie Research Fund, a joint funding initiative between Dimbleby Cancer Care and Marie Curie, Professor Scott Murray at the University of Edinburgh set out to explore better ways for health and social care services to identify carers. He worked with the Marie Curie Hospice, Edinburgh, NHS Lothian, and the support organisation Voice of Carers Across Lothian.Marie Curie The study found that carers often prefer to think of themselves as a wife or a son rather than a ‘carer’, which can mean they don’t ask for help until they are struggling. Carers can become overwhelmed, managing both their own wellbeing and that of the person they are looking after. GPs and nurses should be alert to asking their patients if they have a relative with a serious illness, and carers should mention this to their GPs. With appropriate support, carers will experience greater resilience, enabling them to maximise the quality of care they can provide and their own quality of life.

The research findings were used to inform Marie Curie’s response to the Scottish Government’s Carers (Scotland) Bill and were promoted to Members of the Scottish Parliament (MSP) through a motion of support by MSP Michael McMahon. MSPs received a parliamentary briefing highlighting the key findings from the study. Following Marie Curie’s engagement with MSPs, the Scottish Government has committed to amending the Bill so that the newly proposed adult carers support plans, which all carers will be entitled to, will be fast-tracked for those caring for people with a terminal illness. The team’s work led to media coverage across Scotland, including features in The Scotsman and The Herald newspapers. Dr Emma Carduff, who was the lead researcher on the study, is now Research Lead at the Marie Curie Hospice, Glasgow



Kidney Research UK

A Kidney Research UK funded researcher is increasing our understanding of E.Coli infection in the hope that new treatments can be developed before bacteria become totally resistant to current antibiotics.

Urinary tract infections (UTIs) are one of the most common forms of infection and affect approximately 50% of all women in the UK. If not properly treated UTIs can spread to the kidneys and cause permanent kidney damage. In rare cases the infection can even become life-threatening. The majority of UTIs are caused by the bacteria E.coli which are becoming increasingly resistant to antibiotics meaning these common infections are becoming progressively harder to treat. The World Health Organisation found that resistance to one of the most widely used antibacterial medicines for the treatment of urinary tract infections caused by E. coli–fluoroquinolones–is very widespread. In the 1980s, when these drugs were first introduced, resistance was virtually zero. Today, there are countries in many parts of the world where this treatment is ineffective in more than half of patients.

Growing resistance to antibiotics is one of the biggest public health threats of modern times and designing new treatments to take over from the current classes of antibiotics is vital. To begin to tackle this problem for UTIs Kidney Research UK is funding Dr Rachel Floyd at Liverpool University to investigate which genes and processes are essential for E.coli to infect humans and cause UTI’s.Kidney Research UK Dr Floyd hopes that her research will identify specific processes that drug companies could explore when developing new treatments for UTIs. Without such new treatments the number of serious kidney infections caused by E.coli will begin to rise rapidly.

Throughout her project Dr Floyd communicated this important work in a wide array of ways. She was invited to speak at multiple scientific conferences and also gave talks to undergraduate and postgraduate student groups, health professionals and industry representatives. Her work was press released and covered in local and national newspapers and on local radio. She attended the launch of the Kidney Research UK Renal Report at the House of Lords at which parliamentarians were briefed on the report which includes her work. Dr Floyd has also won multiple awards for her communications skills at the annual Kidney Research UK fellows days. These annual events bring together all researchers currently or previously funded by the charity and have helped establish a well-informed, connected and collaborative and kidney research community in the UK. Dr Floyd has won prizes for her presentations from both scientific and lay audiences, highlighting her skill in communicating her research clearly. This widespread cross sector research communications effort is key for projects like this which could provide a piece of the puzzle needed to tackle the global antibiotic resistance crisis, a problem that can only be solved if we all work together.



Orthopaedic Research UK

Researchers investigating how stem cells could be used to treat common conditions such as lower back pain are engaging the public in an array of ways, including running interactive stem cell days for A-level students to inspire a new generation to become researchers.

Age-related diseases, such as osteoarthritis, osteoporosis and lower back pain, affect musculoskeletal tissues (cartilage, bone and intervertebral disc respectively) cause pain and limited mobility. The prevalence of these diseases is increasing as the population ages and they now represent a major socio-economic burden for countries around the world. For example, low back pain is the second most common cause of pain behind the headache and affects around 80% of adults at some point of their lives, with about 10% of these suffers being chronically affected.

Orthopaedic Research UK funded a project led by Dr Richardson at the University of Manchester to investigate the potential for stem cell therapies for these conditions. Adult stem cells are special cells found in bone marrow and fat tissue that have the unique ability to turn into most of the other cell types in the body including cells from musculoskeletal tissues.Orthopaedic Research UK This project focused on whether stem cells from people with age related diseases were still able to form new musculoskeletal tissue and whether this process could be done in the lab setting that would be required for the cells to be used as a therapy. If successful this could lead to an exciting range of stem cell-based regenerative medicine therapies to repair the damage caused by age-related musculoskeletal diseases.

During this project the researchers found a range of excellent ways to increase public understanding about stem cells. Firstly they set up a dedicated website to explain stem cell biology and the possible applications of stem cells in an accessible way. Next they took part in The Body Experience – and annual event at Manchester Museum where scientists from the university take over the Museum with interactive displays and activities all designed to increase understanding of human biology. Finally the research team ran frequent study days at Manchester Museum during which A-level students from across the region met with scientists to learn more about stem cells and regenerative medicine. Student feedback from the events was excellent with the vast majority of students saying they understood stem cell science better and that they had enjoyed working with the scientists. This is a fantastic example of how researchers can directly help inspire the next generation of scientists, something that is vital if we want to keep driving research forward in the longer term.



Action Medical Research

Improving brain imaging techniques for preterm babies has allowed an Action Medical Research funded team to successfully bid for a €15 million grant from the European Research Council.

Over 60,000 babies are born prematurely each year in the UK. These babies have greatly increased risks of developing a range of long term neurodevelopmental impairments including cerebral palsy and autism. The brain is going through a time of tremendous change in the three months leading up to and surrounding birth as it develops the precise web of connections between cells that are needed to correctly process information. Understanding how this development is disrupted in preterm infants, and being able to identify those most at risk of developing these conditions would allow clinicians to start early interventions to minimise those risks.

Action Medical Research funded two projects by Professor Daniel Rueckert and colleagues to investigate how the methods for assessing brain development in preterm infants could be improved, one of which was co funded by EPSRC. As part of these projects Professor Rueckert’s team developed new MRI based imaging and analysis techniques to produce complex maps of how brain development progresses in infants born prematurely.Action Medical Research Hundreds of these maps have been produced and are grouped into atlases which are freely available to researchers from the teams online database brain-development.org. Research teams from all over the world have made use of these atlases and they have been cited over 200 times. The maps have also enabled the researchers to build a prototype computer system that can learn patterns of normal and abnormal brain development and automatically interpret MRI scans to predict a baby’s risk of developing neurodevelopmental impairments. This is currently under development as a diagnostic tool.

The research team successfully applied for a prestigious €15,000,000 synergy grant from the European Research Council to continue this work of which a €3,250,000 share was awarded to Professor Rueckert. ERC launched synergy grants aim to enhancing collaborations between lead researchers whose complementary skills, knowledge and resources can realise results that would not be possible if they were working alone. The ERC received over 700 applications for synergy grants and have funded just 11 projects across the EU highlighting the extremely high quality of the team’s proposal.

The aim of the six year synergy grant is to produce a higher resolution dynamic map showing exactly when and where nerves are forming connections in the developing brain in the three months before birth and shortly afterward. The team hope that by understanding precisely how each area of the brain develops and connects they will be able to uncover why preterm babies often go on to develop diseases like cerebral palsy and autism, a finding which would significantly enhance efforts to develop preventative interventions and treatments.



Heart Research UK

Research funded by Heart Research UK contributed to a team winning an NC3Rs CRACK-IT challenge designed to produce a way to reliably test whether new drugs will be safe for the human heart without the use of animal models.

The process required to take potential new drugs from the lab to the clinic is long and complicated, with an average timescale of over 12 years at an average cost over £1.1 billion. An essential stage in this process is rigorous safety testing in cells and animal models before any drug is trialled on patients. This process currently requires ~500,000 procedures on rodents, rabbits, dogs and primates in the UK annually. One of the most common causes of drugs failing to reach clinical trials is that they are found to have adverse effects on the heart during safety tests, which could lead to clinical heart failure and even death in patients. All AMRC members are committed to the principles of the 3Rs in animal research which aim where possible to replace, reduce and refine the use of animals for scientific purposes. Developing a non-animal based system to reliably assess whether potential drugs would adversely affect the heart would be a big step towards reducing the number of animals used in the drug discovery process.

In 2013 Heart Research UK funded Professor Chris Denning at the University of Nottingham to evaluate potential new drugs for genetic heart conditions that change the rhythm of the heartbeat which can lead to fainting, seizures and cardiac arrest.Heart Research UK In this project patients donated a small skin sample which the researchers converted into stem cells that could then be pushed to become heart muscle cells known as cardiomyocytes. These human induced pluripotent stem cell derived cardiomyocytes (hiPSC-CMs) beat at the same rhythm as those in the patient’s heart, providing an excellent system to test whether a drug can return the heartbeat to a natural rhythm.

Chris used this experience in producing and using hiPSC-CMs for drug testing to successfully win further funding from the National Centre for the Replacement, Refinement and Reduction of Animals in Research (NC3Rs), an organisation dedicated to promoting the importance of the 3R’s. As part of their work NC3Rs work with industry and other funders to run frequent CRACK-IT challenges, project calls designed to tackle specific problems that hinder the 3Rs. Chris won both rounds of the In-Pulse challenge and was awarded a total of £1.1 million from NC3Rs. This brings together an international consortium comprising academic partners (Chris Denning, Nottingham; Christine Mummery, Holland; Thomas Eschenhagen, Germany; Godfrey Smith, Glasgow), small biotech companies (Clyde Biosciences, Glasgow; Pluriomics, Leiden) and an industrial sponsor, GlaxoSmithKline. This challenge asked applicants to design a robust 2D and 3D human cell based system that could be used to assess drug-induced changes in heart electrical, calcium and contractile activity. The team are using the hiPSC-CM method and are currently perfecting and validating their 2D and 3D model platforms. Once validated, these platforms could be used to screen all new drugs and provide earlier safety decisions, greatly reducing the number of animals used to test compounds that would ultimately fail in development and speeding up the drug development pathway.

“Cardiovascular disease is still a major cause of death in the UK but much of the new research is being funded by charities. The support Heart Research UK provided to my lab meant I could leverage seven times more funding from government and industry. This enabled new collaborations throughout Europe and the USA, which will accelerate our efforts to produce better drugs that relying less on animal use” Researcher Perspective



Chest Heart and Stroke Scotland

Experience gained during Chest Heart and Stroke Scotland funded trial of robotic therapy in stroke survivors contributed to a successful bid to NIHR HTA to fund a £3 million randomised controlled trial and developed capacity to deliver clinical trials of novel rehabilitation interventions.

Stroke occurs approximately 152,000 times a year in the UK; that is one every 3 minutes 27 seconds. Stroke is a major cause of disability and approximately half of all stroke survivors have long-term disability and impairment of the upper limb. Recently developed robotic therapy systems can improve arm movements in patients with weakness many years after stroke and may be cost-effective compared to ‘traditional’ one-to-one physiotherapy. It is believed that in order to maximise recovery that therapeutic interventions should be started as soon as possible after a stroke but there is no evidence yet for use of robotic therapy in the first weeks after stroke.

With the support of Chest Heart and Stroke Scotland, a team led by Dr Jesse Dawson at the Western Infirmary and Queen Elizabeth University Hospital in Glasgow, is testing robotic therapy systems, in the early post stroke period. The robots were developed by scientists at Massachusetts Institute of Technology (MIT). Patients were recruited within seven days of their stroke and had three one hour-long sessions per week, for four weeks. Tasks included circle drawing, reaching targets, and holding / moving against moderate resistance.Chest Heart and Stroke Scotland Patients’ limb function and wider functional capacity were assessed after one and three months. Final results are under analysis and the outcome will demonstrate whether robotic therapy is effective, and tolerated by patients, in the early stage after stroke. If successful, this will not only improve patients’ functional capacity, but should also prevent weaknesses becoming chronic, and more difficult to treat later on.

As a result of their position in the field and the experience and knowledge gained from the small scale trial funded by CHSS Dr Dawson’s team were chosen to be one of several countrywide centres collaborating on a larger randomised controlled trial of robotic therapy. The collaborative team was awarded further funding of over £3 million from the NIHR Health Technology Assessment programme for the RATULs trial. 720 participants will undergo standard NHS treatments, enhanced traditional therapy or robotic therapy to thoroughly assess whether there is any difference in outcomes between these forms of therapy. This will enable researchers to establish whether robotic therapy could be of widespread benefit to stroke patients all stages of their recovery. As these machines become more widely used, the costs are likely to reduce, increasing cost-effectiveness of the treatment.



Spinal Research

A promising treatment for spinal cord injury was shown to restore limb function in animal models and is being driven towards clinical trials by a Spinal Research funded consortium.

The spinal cord is the body’s ‘information superhighway’, enabling communication between the brain and all other regions of the body. If the spinal cord is injured the damaged neurons are no longer able to conduct signals to and from the brain leading to devastating life-long effects including paralysis, loss of sensory information and disruption of basic bodily functions including bladder control, breathing, and control of body temperature. 50,000 people in the UK are living with paralysis due to spinal cord injury (SCI). In most cases of SCI the spinal cord is not completely severed and many neurons do remain active initially. However over a short time scar tissue forms in the damaged region and this produces proteins that inhibit the regrowth of damaged neurons and also affects the survival of those that do remain.

Spinal ResearchFor many years a bacterial enzyme called chondroitinase has been suggested as a possible treatment option for patients with SCI. This enzyme is known to digest the toxic proteins produced by the scar tissue, protect the intact neurons and reactivate their ability to sprout new growths enabling them to make new connections and take over the functions of the damaged nerve cells. Spinal Research has funded studies on chondroitinase for many years and in 2012, having seen its great promise, it become one of the core research focuses of the charity to produce a therapeutic form of chondroitinase. To try and achieve this the charity established the CHASE-IT consortium, a collaboration of world leaders in the field.

The method of delivery of chondroitinase to the scar site is one of the major issues currently preventing chondroitinase being trialled as a treatment in humans. Members of the consortium are tackling this by developing and testing an engineered form of the chondroitinase gene that can be expressed by mammalian cells. The team have used engineered viruses to deliver this to the injury site in rat models of SCI and have shown it leads to efficient and lasting expression of the enzyme across the site of injury. Excitingly this was shown to lead to dramatic improvements in nerve function and long term recovery of limb function. This breakthrough offers real hope of a treatment for SCI and the consortium is now working on testing this therapy in larger animal models and proving its safety which, if successful, could lead directly to the first chondroitinase clinical trials in human SCI patients.

“We believe that a collaborative approach to research is imperative if we are to see successful clinical trials for spinal cord injury. Spinal Research has a great track record for supporting both collaboration and outcome-driven research and we feel privileged to have counted on their support towards our goal of developing a cure for spinal cord injury.” Researcher Perspective



Breast Cancer Now

Collaboration between UK and EU imaging projects is allowing researchers increased access to patients and increasing the likelihood that the teams can find new markers of disease progression that would allow more personalised treatment plans.

Breast cancer is the most common cancer in the UK with more than 50,000 women diagnosed each year. One in eight women in the UK will develop breast cancer at some point in their lifetime. More people than ever are surviving breast cancer thanks to better awareness, screening and treatments but nearly 1,000 UK women still die of breast cancer every month. Each patient is unique and will respond differently to the available treatments. Finding new ways to predict whether a certain treatment will benefit a patient would prevent patients undergoing unnecessary and unsuccessful treatments and give doctors more chance to focus on the treatments that could save lives.

Breast Cancer Now is funding a research team at the University of Dundee, led by Professor Andrew Evans, investigating a new imaging technique known as shear wave elastography. They have shown it is possible to predict how well patients will respond to chemotherapy given prior to surgery by studying changes in the stiffness of breast tissue using this technique.Breast Cancer Now During the course of the project, the team formed a collaboration with the EU funded VPH Prism project- a Virtual Physiological Human modelling system that allows the microstructure of the breast tissue to be modelled and which also takes into consideration the environmental, genetic and clinical factors known to influence breast cancer progression.

As a result of the collaboration, patients who are involved in the Breast Cancer Now-funded shear wave elastography studies had the opportunity to also be involved with the VPH Prism project which provided a more detailed image of their tumour and the surrounding breast. The collaboration meant both projects increased patient recruitment, increasing the chances of identifying useful new biomarkers from the collected data and images. Successful identification of such biomarkers would be a big step towards improved prediction of breast cancer progression and the ability to tailor patients’ treatment more effectively.

“The collaboration between members VPH-PRISM consortium and my group has led to a number of further research projects which are currently being under taken” Researcher Perspective



Arthritis Research UK

A global research consortium of both academic and industrial partners brought together by Arthritis research UK is investigating a potential new drug target that could help the 30% of rheumatoid arthritis sufferers who don’t respond to current treatments

Rheumatoid arthritis causes joint pain, swelling, stiffness and fatigue and affects approximately 460,000 people in the UK. Finding an effective treatment for rheumatoid arthritis is challenging but can have a huge impact on the daily lives of people who live with the condition, reducing the chance of disability or mobility issues. Since the 1990s, Arthritis Research UK has been a proud pioneer in developing anti-TNF therapy for rheumatoid arthritis, which has transformed lives of many people with the condition. However, up to 30% of patients do not respond to anti-TNF therapy, the cost of the treatment is very high and many do not qualify for it. Therefore, the organisation continues to work on finding the next generation of treatments.

Between 2012 and 2016 Arthritis Research UK helped establish a global research collaboration looking at new ways to treat rheumatoid arthritis. The research investigates the function of a particular enzyme called MT1-MMP (MT1- metalloproteinases) that causes damage to joints in rheumatoid arthritis by breaking down joint tissues.Arthritis Research UK Led by Dr. Yoshifumi Itoh at the University of Oxford, the research was supported by a global collaboration network that brought in different expertise and research skills, from both academic and industrial sectors. The three collaborators in the UK (based at the University of Cambridge, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Diseases and private company Bicycle Therapeutics) contributed to the design of inhibitors to supress the function of MT1-MMP and also develop the mouse and cell models for testing the function of the inhibitors. Another three partners in the US (based at Harvard University and private companies Kadmon corporation and Dyax) helped develop and test an inhibitory antibody for MT1-MMP. While two collaborators in the EU (based at Aarhus University and University of Pisa) helped prepare and test the inhibitors.

This study showed that targeting MT1-MMP provides a potential strategy for joint protection, and its combination with anti-TNF may be particularly beneficial in rheumatoid arthritis patients who do not respond well to anti-TNF therapy. Dr. Itoh plans to conduct further studies on MT1-MMP to evaluate its potential to become a therapeutic target for rheumatoid arthritis. This research is an excellent illustration of how by working together we can make changes that give fuller lives to the millions of people living with arthritis now and in the future.



Wellbeing of Women

A priority setting partnership is collating a list of the top ten research priorities for endometriosis to help researchers and funders ensure resources are focused on the work that matters most.

Endometriosis is a common condition that affects around 1.5 million women in the UK. In women with endometriosis small pieces of the womb lining (the endometrium) grow outside the womb, most commonly in the pelvis or on the ovaries. It is a chronic condition that is associated with debilitating painful or heavy periods, pain in the lower abdomen, pelvis or lower back. It may also lead to lack of energy, depression and fertility problems. There is no known cure for endometriosis and symptoms are currently managed with painkillers, hormone treatments or surgery.

Wellbeing of Women funded Professor Andrew Horne at the University of Edinburgh to investigate a group of genes that could pave the way to potential new treatments for endometriosis. The transforming growth factor-β (TGFβ) superfamily is a group of genes known to affect cell growth, cell survival and cell relationships, as well as on the development of new blood vessels. The team believes changes in the production and actions of the TGFβ-superfamily lead to some cells becoming abnormally sticky to the endometrium leading to endometriosis.Wellbeing of Women Drugs which change the production or action of these compounds might be able to block the development and progression of endometriosis. These drugs are already being developed for the treatment of other conditions giving hope that a new treatment will would greatly accelerate how quickly the drugs could be available if this approach was found to be beneficial for endometriosis.

Alongside their research work on new treatments Professor Horne’s group have initiated a priority setting partnership (PSP) in collaboration with the James Lind Alliance. PSP’s are a structured way to allow the research community, patient population and healthcare staff interested in a condition to highlight the next questions they feel researchers should address. These have been successfully run in many other health areas over the last few years. The result of the partnership is the generation of a top ten list of research priorities taking into account all the gathered views. This is a huge help to both funders and researchers who can make sure they are focusing time and resources in the most important topics. It also means research becomes highly focused on the areas that will really make a big difference to women living with endometriosis.

“A PSP brings patients, carers and clinicians together to identify and prioritise uncertainties, or ‘unanswered questions’, about the effects of treatments that they agree are the most important. PSPs are important to help ensure that those who fund health research are aware of what really matters to both patients and clinicians” Researcher Perspective



British Heart Foundation

Long term support from the BHF has allowed Dr Nicola Smart to become a world leading scientist in the field of regenerative medicine.

The British Heart Foundation’s (BHF) vision is a world in which people do not die prematurely or suffer from heart disease. Over half a million people are living with heart failure in the UK, a condition for which there is no cure. The BHF’s Mending Broken Hearts initiative seeks to develop ways of regenerating the heart following a heart attack to stop the progression of heart failure.

Dr Nicola Smart’s research is at the forefront of the Mending Broken Hearts initiative. Nicola is the BHF Ian Fleming Senior Basic Science Research Fellow at the University of Oxford (The Ian Fleming fellowship is named in honour of the James Bond author, Ian Fleming, in recognition of his family’s support of the BHF). Her research focuses on finding ways to repair adult heart tissue and blood vessels by looking for clues in the embryo. She believes that if we know more about the cells and pathways that instruct the heart and blood vessels to form in the embryo we can find ways to reactivate these dormant ‘construction blueprints’ in the adult heart, boosting repair processes.

Early in her postdoctoral career, Nicola showed that the epicardium, a dormant cell layer that covers the surface of the heart, can be triggered to help repair damaged heart muscle. The BHF has supported Nicola since this initial discovery through the BHF Ian Fleming Intermediate Basic Science Research Fellowship and now as the BHF Ian Fleming Senior Research Fellow.British Heart Foundation	She continues to focus on finding ways to boost the reparative power of the epicardium, and on defining the mechanisms behind this regenerative ability. As a leader in the field of regenerative medicine, Nicola has also successfully applied for BHF project grant support allowing her to grow her lab with two postdoctoral scientists.

The BHF is committed to supporting the most talented researchers at all career stages as outlined in our recent Research Strategy to 2020. Our fellowship schemes allow flexible working and we have recently changed the duration of our schemes to ensure we fully support the brightest scientists in the fight against heart disease.



Northern Ireland Chest Heart and Stroke

Charity supported researchers play a vital role in the global peer review process required to ensure only the very best science is funded.

Worldwide public health campaigns mean we all know that increasing fruit and vegetable intake would be a healthy change to most of our diets. However why exactly fruit and vegetables lead to the health benefits they do is something scientists are still studying. Increased fruit and vegetable intake is associated with a reduced risk of cardiovascular disease (CVD), yet the mechanism by which this dietary change acts to reduce risk remains to be established. Northern Ireland Chest Heart and Stroke funded a project by Professor Jayne Woodside at Queen’s University of Belfast to try and unravel this mystery. Her results demonstrated that the dietary changes altered specific DNA methylation and gene expression levels in a dose dependent manner meaning the number of portions of fruit and vegetables we eat has a direct effect on which of our genes we express. The team feels these findings further strengthen public health messages to increase fruit and vegetable intake, and could even form a new basis for public health messages.

As a result of her excellent international reputation and her body of work in the field Professor Woodside was invited to be a grant panel member on an EU Joint Programming Initiative “A healthy diet for a healthy life” Biomarkers in Nutrition and Health call.Northern Ireland Chest Heart and Stroke	The vision of the JPI A healthy diet for a healthy life is that by 2030 all citizens will have the motivation, ability and opportunity to consume a healthy diet from a variety of foods, have healthy levels of physical activity and that the incidence of diet-related diseases will have decreased significantly. The specific call had a 5million euro budget and aims to further increase our understanding of how diet changes biomarkers like the DNA methylation and gene expression changes seen in Professor Woodside’s study.

Taking part in the peer review process is a fundamental part of every researcher’s career. All AMRC member charities have rigorous peer review procedures that ensure they are making the most effective use of their resources through supporting the best science, and the best scientists, and similar procedures are undertaken by all large funding bodies across the world. This requires a constant stream of peer review of every application, by the leaders in the field like Professor Woodside, the vast majority of whom will have been funded by a charity at some point in their career. Whilst time consuming this global process ensures we focus on what really matters and maximise the potential benefits of the billions of pounds we invest in research. Without charity funding, which accounts for over a third of all publically funded medical research in the UK, the pool of researchers available to take part in this vital process would be greatly diminished.



Alzheimer’s Society and Alzheimer’s Research UK

A nationwide network of brain banks jointly funded by the Alzheimer’s Society and Alzheimer’s Research UK are providing vital tissue samples to researchers working across the dementia spectrum to drive research forward.

There are over 850,000 people with dementia in the UK today, and this figure is set to double in the next 30 years. The impact of this growing number of people with dementia is one of the most urgent health and social care challenges currently facing the UK. Current treatment options are limited and there is no cure highlighting the urgent need for research into the range of diseases that cause dementia.

In 2007 Alzheimer’s Research UK and the Alzheimer’s Society recognised this need and worked together to establish Brains for Dementia Research (BDR) - a network of brain bank facilities across England and Wales. Understanding what is happening in the brains of people with dementia compared to those who are not affected is a vital step towards treatment development. Brain tissue is not covered in standard organ donation schemes so BDR coordinates the collection and storage of donated brain tissue in six leading centres across the UK, with support from the Medical Research Council (MRC).Alzheimer’s Society and Alzheimer’s Research UK There are more than 3,000 people currently registered with BDR, two-thirds of whom do not have dementia. Brains from the 500 people who have died are being used in research projects. The assessment information being collected by BDR will also become part of the Dementias Platform UK where it can contribute to very large studies looking at the onset of dementia. All brains from BDR are also listed on the MRC UK Brain Banks Network database to increase the discoverability of the cohort.

BDR provides full support to potential donors and their families to help them understand the need for tissue donation and how their tissue will be used. Each potential donor has regular assessments to check their memory, thinking and behaviour.Alzheimer’s Society and Alzheimer’s Research UK This unique approach to brain donation provides researchers with a complete medical history to accompany the donated tissue, allowing them to conduct much more powerful research that can link information on changes seen in the brain structure directly with the symptoms experienced.

Almost 200 research projects looking into all forms of dementia have requested tissue samples from BDR. Each application is peer reviewed by both leading scientists in the field and people with a direct personal connection to dementia to ensure the tissue is only used for research of the highest calibre and relevance to people with dementia. In our dataset, 21 different projects funded by five of our members reported using samples provided by BDR. This shows that not only do our members fund the high quality research required to access the samples provided by BDR, but also that collaboration between member charities is providing a vital resource used by a wide array of the dementia research community in the UK.



Rosetrees Trust

Rosetrees Trust research strategy allows researchers to develop innovative projects and obtain the data needed for larger scale funding applications.

Rosetrees Trust funds research across the spectrum of human health. Funded by the founding family, the Trust looks to secure the best possible outcome for money awarded to research. Initially, Rosetrees funded a large number of small grants but as its expertise has grown, it has expanded into the provision of major grants whilst at the same time offering seed corn funding.

The idea behind the seed corn funding is to enable researchers to obtain data in order to apply for major grants from larger funders. This type of funding allows researchers in all health fields to test new and innovative approaches and investigate ideas that larger funders may not yet be willing to support. Without charities playing this vital role in the funding ecosystem, it can be hard for researchers to obtain funding for the types of cutting edge high-risk high-reward research that drives healthcare forwards.

Rosetrees TrustOver the past few years the charity has made multiple small awards to support a spectrum of innovative research projects in the lab of Professor Molly Stevens at Imperial College London. Professor Stevens has a large multidisciplinary research group that comprises biologists, bioengineers, chemists, material scientists and surgeons. One of the main focuses of the group is regenerative medicine. Put simply, regenerative medicine includes research into the process of engineering, replacing, or regenerating human cells, tissues and organs to restore or establish normal function.

Rosetrees has supported regenerative medicine projects in the Stevens group in areas including repair of the heart after a heart attack, developing scaffolds to help engineer kidneys for transplant, and regeneration of cartilage and bone and many more. This breadth and quality of work in the group led to them being awarded the title of Research Group of the Year at the 2014 European Life Science Awards. This recognition would not have been possible without charities like Rosetrees Trust being willing to support the early stages of new work across the group’s cutting edge research portfolio. Many AMRC members have similar funding schemes and these make up an essential component of the UK research funding landscape, driving innovation that may otherwise not be funded.