Our vision is a world in which all rare diseases have treatments – made together with patients, for patients
Findacure is a UK charity that is building the rare disease community to drive research and develop treatments. We are here to transform the world’s understanding of rare diseases and the devastating impact they have on people’s lives.
How are we achieving our vision?
- We transform the lives of people affected by rare diseases by working directly with patient support groups, giving them the skills to professionalise their work and the confidence to recognise their own expertise in their rare conditions.
- We accelerate change in rare disease research and care by building a strong, supportive, and collaborative rare disease community that works for the benefit of all rare conditions.
What do we do?
Findacure’s projects are in high demand, helping patient groups to:
Build their charity
Raise awareness of their rare condition
Get research ready
Provide support to patients and families
Charity number: 1149646
Company number: 08174973
Findacure was founded in 2012 in Cambridge, UK. After hiring our first full-time employee in late 2013, we are now a vibrant team of four, running a diverse range of projects to meet the needs of our beneficiaries.
The need for our organisation was realised out of the separate experiences of our co-founders, Nick Sireau and Tony Hall:
Nick’s two sons were born with the rare disease alkaptonuria, an ultra-rare genetic condition that causes bones to turn black and their surface to corrode. At the time of his sons’ diagnoses, there was no treatment available. Nick quit his job to grow the AKU Society, a charity that now supports patients around the globe and is awaiting the results of a major clinical trial this coming September.
Later in his journey, Nick began to be contacted by other patients and parents wanting to repeat his achievements in their own rare conditions. He realised there was huge demand for a new organisation to share advice and knowledge between patient groups, saving each the time, resources, and stress of having to reinvent the wheel.
As an expert in orphan drug development, Tony had seen first-hand the financial and logistical challenges that pharmaceutical companies face when developing treatments for small patient populations. He realised the need for a non-profit approach to improve patient health while not breaking the NHS bank.
At the time when he met Nick, Tony was interested in establishing an organisation to work on sustainable models for not-for-profit drug development for rare diseases. The two teamed up to establish Findacure and work on his ideas.
Nick was delighted to share his story at a TEDxImperialCollege in 2012. Watch his talk here:
Dr. Rick Thompson
Dr Rick Thompson joined Findacure in 2015, after completing his PhD in Evolutionary Biology at the University of Cambridge. He began working as Findacure’s first Scientific Officer, helping to develop the charity’s proof of concept study for the innovative drug repurposing social impact bond. Having been promoted to CEO in May 2017, Rick now oversees the charity’s strategy and development, oversees major funding applications, and manages the team, while still maintaining Findacure’s scientific projects. Rick is always keen to speak about Findacure’s work, and aims to encourage industry engagement with rare disease patient groups, promoting an open and collaborative approach to rare disease research and patient support.
Mary Rose Roberts
Mary Rose is responsible for delivering and developing Findacure’s events. Primarily, she organises our annual Drug Repurposing for Rare Diseases Conference, the Rare Disease Showcase series and our patient training workshops. Mary Rose also oversees Findacure’s community fundraising events and acts as the charity’s Data Protection Officer. She is keen to encourage communication between industry and patients, and passionate about ensuring Findacure’s activities are engaging and accessible to our community. Mary Rose has a first class hons. degree in Politics from Keele University and joined Findacure in 2016.
Taryn is responsible for overseeing all of Findacure’s external communications. This includes increasing Findacure’s visibility within the UK healthcare system and overseeing our monthly newsletter, weekly blog, and website. She also manages our social media streams and press releases. Her goal is to streamline our communications strategies. Taryn has a first class hons. degree in English from the University of Cambridge and joined Findacure in 2019.
Our board of trustees
Dr. Nicolas Sireau
Co-founder and Chairman
Dr Nicolas Sireau is the Interim CEO and Chair of Trustees at the AKU Society, a patient group that helps people with AKU (short for alkaptonuria), a rare genetic disease affecting both his children. Previously, Nick was Patient Engagement Director in Oncology at AstraZeneca and beforehand, was the CEO of SolarAid. He is a fellow of the Ashoka Fellowship of Social Entrepreneurs and has a PhD in the social psychology of social movements. He is the editor of ‘Rare Diseases: Challenges and Opportunities for Social Entrepreneurs’ (Greenleaf 2013).
Dr. Anthony Hall
Co-founder and Vice-chair
Dr Anthony Hall graduated from King’s College London with first class honours in physiology and pharmacology before going on to study medicine at the Royal Free Hospital. He joined the pharmaceutical industry in 1994 and has significant experience in the clinical and regulatory development of drugs for rare diseases. He speaks regularly at rare diseases conferences, is author of a number of articles on orphan drugs and is a member of the IRDiRC Working Group on Orphan drug-development and regulatory processes.
Professor Lakshminarayan R. Ranganath
Professor Lakshminarayan Ranganath is a busy full time consultant at the Royal Liverpool University Hospital in the UK. There was no National Health Service or treatment available for rare disease Alkaptonuria in the UK when he developed an interest. To address these issues, he established an NHS Highly Specialised Services funded National Alkaptonuria Centre (NAC), of which he is the inaugral Director. Patients in the NAC are able to access off-label nitisinone free of charge and access a multidisciplinary team of experts. LRR has carried out a national survey that identified 81 UK, 450 European and 1000 patients worldwide. He has pioneered an assessment of AKU patients. LRR is also co-ordinating DevelopAKUre, a European Union-funded international research programme, which involves 3 studies in AKU. This will bring advances in AKU to all patients with AKU worldwide.
Andy Milligan is a leading international brand and business consultant and a founding partner of The Caffeine Partnership, which provides ideas, energy and advice to senior managers on business growth and brands. His experience spans industries as diverse as airlines, ﬁnancial services, fast-moving consumer goods, telecommunications, sports and leisure and pharmaceuticals. Andy has published five bestselling business books.
Edna Kissmann is one of the owners and senior partners in Kissmann Langford, a strategic communications consultancy. Previously, she served as Assistant Press Secretary to the late Prime Minister Rabin of Israel and as Global Chair of Healthcare Practice and Chief Knowledge Officer at Burson-Marsteller, a major communications company. She has been a trustee of the Ashoka Fellowship of Social Entrepreneurs and is now a member of the Ashoka Support Network.
Our scientific advisory committee
Dr. Bruce Bloom
Dr. Bruce Bloom is Founder of Cures Within Reach, a non-profit saving lives by repurposing human approved drugs and devices to quickly deliver safe and affordable treatments and cures for diseases that have no currently effective therapy.
Dr. Bloom became an Ashoka Social Entrepreneur Fellow for his system-changing solutions to finding new treatments for unsolved diseases. He is the Patient Advisory Board Chair for the Institute for Translational Medicine, the Director of Scientific Affairs for Vision for Tomorrow, on the Science Advisory Boards of Rediscovery Life Sciences, the GARROD AKU Consortium, the Dr. Ralph and Marian Falk Medical Research Trust Awards Programs, and Healx, the Chair of the Governance Committee of the Kendall College Charitable Trust, and a member of the Board of Councilors of Midwestern University, and a member of the editorial board of ASSAY and Drug Development Technologies.
Dr. David Cavalla
David Cavalla has 30 years experience in various senior scientific and commercial roles within the pharmaceutical industry, and 15 years in drug repurposing; previously he was founder and CEO of Arachnova Ltd, and prior to that, at Glaxo Group Research Ltd and Napp Research Centre. He is also founder and principal of Numedicus Limited.
He is author of Off-Label Prescribing: Justifying Unapproved Medicine, and one of the first advocates of drug repurposing, using this strategy to create three first-in-class developmental products to have successfully passed human proof of concept clinical stages. He obtained a first degree and PhD at Cambridge University and spent two years as a visiting Fellow at the NIMH, Washington, DC. He is author/inventor of over 70 published papers and patents.
Dr. Mark Edwards
Mark is a medically-qualified doctor (a former NHS anaesthetist) with c.25 years’ pharmaceutical R&D experience. He was a Global Clinical R&D Director and then Director of Science and Medical Public Affairs at Pfizer. He has worked at all phases of clinical development across a wide variety of therapeutic areas. This included leading Pfizer’s global clinical development team to achieve US/EU regulatory approval of sildenafil as a first-in-class treatment for pulmonary arterial hypertension.
He has also established major R&D projects at the interfaces between academia, the NHS and the biopharmaceutical industry e.g. MRC/industry stratified medicine consortia in COPD and RA, and the NIHR’s Translational Research Partnership Programme.
Now he mainly provides strategic and applied clinical, scientific and policy advice to small and medium-sized biopharmaceutical companies. Mark works with a diverse range of other life sciences organisations at Board/Committee level, or as a business mentor. These include the ME Trust and Dyskeratosis Congenita Action.
Dr. Kathryn Johnson
Kathryn is a neonatal paediatrician working at the Leeds Teaching Hospitals NHS Trust where she holds positions as a Consultant Neonatologist and Research Lead for Leeds Neonatal Service. The Neonatal Service in Leeds is one of the busiest in the country with 55 beds including 15 intensive care cots. The service provides care to infants with surgical, cardiac, neurosurgical, renal and hepatic conditions including many with rare congenital abnormalities or diseases.
Kathryn has a long standing interest in neonatal/paediatric rare disease and is committed to supporting research into such conditions with the ultimate aim of improving the lives of babies, children and families. She has a strong research delivery record within the neonatal service and as an executive committee member for the British Paediatric Surveillance Unit has a unique experience and insight into surveillance for paediatric rare disease. Kathryn’s is also Scientific Advisor for the charity Action for Ataxia Telangectasia, where her role is to monitor the progress and outcomes of the research projects the charity funds.
Margaret Ogden lives with Wegener’s Granulomotosis, a rare auto-immune disease. She has been a volunteer in patient and public involvement activities over the past decade. She is a Patient Safety Champion and has worked with medical students at Teesside University. Her main areas of interest centre around research and she attended the International Research Summer School in 2011. Her specialisms include diagnostics, developing guidance and quality standards, the community pharmacy and medication reviews, oral health, respiratory disease, equality and diversity and patient empowerment.
She has found living with a chronic physical illness difficult and challenging. While tolerating the treatment well, the side effects have been overwhelming. She hopes that this is an area where she can give a substantial contribution to the work of the Committee. Repurposing of drugs will be a new field and she is looking forward to meeting the challenges of this important area of work.
Dr. Edmund Jessop
Edmund Jessop has been practising public health for the NHS in England since 1981 in various jobs at district, regional and national levels. Since 2002, he has been medical adviser to the team which plans, funds and monitors services for patients with very rare disease. This has included decision making, and more recently close liaison with NICE, on the so-called “ultra-orphan” drugs as well as other highly specialised technologies. Edmund is UK representative to the EU Expert Group on Rare Disease.
He was Vice President of the UK Faculty of Public Health (FPH) from 2011 to 2014 and Editor of the Journal of Public Health 1998 – 2007. He has taught courses on public health including health economics and health technology assessment for over 15 years.
Dr. Kamlesh Sheth
Dr Kamlesh Sheth is a UK-based licensed pharmaceutical physician with over 25 years experience in the healthcare and life-science industry. He is director of PharmacoMedics, Aegleio and Glockal. Kamlesh has significant experience of working with patient groups including mentoring. He also has drug development, clinical research and regulatory experience of over ten years in rare diseases. Kamlesh has a keen interest in and experience of drug re-purposing in the pharmaceutical industry. He is a Fellow of the Faculty of Pharmaceutical Medicine (FPM) with additional responsibility as a chairman of the Commercial, Industrial and Medical Affairs Special Interest Network of the FPM and also a member of its Rare Disease Network. Kamlesh is an operational committee member of British Association of Pharmaceutical Physicians (BrAPP), medical appraiser for pharmaceutical physicians and is on the Board of Examiners of FPM.
Our Patient Engagement Committee
Ola holds a BSc in Nutrition and Dietics as well as a PgDip in Psychology, and established a career in research. She was diagnosed with a Desmoid Tumour in 2011, and since then has conducted extensive research about the condition which she has shared with others with this condition, as well as medical professionals. Her experience with supporting the Desmoid community led her to a change of career last year, having obtained a qualification in wellness coaching from the Institute of Leadership Management.
Ola took part in the first, and so far only, research on quality of life amongst Desmoid patients in the UK. She is interested in supporting older groups (40+), particularly from BME groups, as many struggle to deal with a diagnosis of a rare condition alongside caring for family and work. She believes contributing her extensive experience, knowledge, skills and training, as well as supplying a BME voice (groups often not represented) will help the committee reach and support a wider and diverse audience.
Sue is the chair of Pitt Hopkins UK, a charity she helped found in 2016 after her middle son was diagnosed in 2007 with Pitt-Hopkins syndrome (PTHS) as part of a European research project. She is also a moderator of the international and UK Google groups, a co-moderator of an international Facebook group and of the PTHS RareConnect group. She has been the International Representative for the Dutch Pitt-Hopkins syndroom charity since 2009, when it was founded, and is also a patient advocate for the ERN ITHACA.
Laurence is founder and director of On The Pulse – an independent consultancy delivering specialist insight, strategy and value proposition to support the holistic and integrated approach of treatment and care provision for people affected by the rarebleeding disorder, haemophilia.
Laurence has extensive patient advocacy experience, having advised on and implemented a broad scope of community awareness, research and educational initiatives for commercial partners and third-sector organisations. His particular focus and expertise with On The Pulse
is youth engagement and empowerment to both accelerate and make more effective the transition process and support life-affirming self-management.
Laurence is highly driven by his own journey and challenges of living with severe haemophilia, and the impact on the family dynamic, to campaign for and effect real change in improvements to quality of life for his peers.
Alan is a patient engagement advocate for rare diseases in general and Ataxia in particular. Ataxia means “lack of order” and people with ataxia have problems with movement, balance and speech. Alan is fighting to return order to the lack of order. He is known as the “rare disease warrior” and, as a patient with a life-limiting rare disease, he can convey the patient engagement message from his point of view in all issues regarding the wellbeing of patients.
Alan has established a patient-driven and focused group and campaigns at many levels, including patient forums, local and regional health boards, Welsh/UK government organisations, as well as taking part in many global conferences, in person or via the internet.
He was proud to receive a nomination from the Bevan Foundation in 2015 in the Health and Wellbeing category. He also participated in the Peer mentoring programme, run by Findacure, to progress Atacsia a Fi – Ataxia and Me©, an online resource for all affected by this life-limiting condition, as well as incorporating associated rare diseases and movement disorders.
Sandra’s career started as a nephrology and urology nurse, which she did for 12 years. She moved into the pharmaceutical arena when she had her family and worked in pharmacovigilance roles in phase 2-3 trials. Within these roles she was also responsible for training the study teams on protocols related to capturing safety data, event driven data and development of protocols. After her contracts ended she decided to return back to nursing as a research sister to understand studies from this perspective and set up a clinical trials unit for stroke studies.
She moved into a nurse practitioner role for a few years specialising in renal and urology, then returned to the pharmaceutical industry as an in-house Clinical Research Associate (CRA) and progressed to a Senior CRA. She is currently working for patientprimary as Director of Patient Services where she has been developing the service in collaboration with patients, families, caregivers, advocacy groups and charitable organisations.
Sally is the Young People’s Project Officer for Metabolic Support UK, a leading umbrella patient organisation which deals with around 500 rare disorders. She also lives with a rare inherited bone disorder named XLH (X-linked Hypophosphatemia) and is a volunteer with XLH UK, the first
organisation of its kind for patients in the UK, as well as using Instagram to blog about her life with the condition and raise awareness.
Prior to joining Metabolic Support UK Sally’s background was in continuous improvement, training, and process analysis. Through their ‘NextGen’ project she now uses these skills and her experiences as a patient to develop support services for young people with inherited metabolic disorders, dealing
with topics such as education, employment, mental health, and independence.
Sally is passionate about ways in which the rare disease community can work together to understand and promote shared interests and experiences particularly living with physical
differences, breaking down perceptions and barriers in wider society, and connecting the patient
Wendy studied psychology at Bangor University, Wales and has a PhD in Cognitive Neuroscience. She has worked in the field of special educational needs, both in local government and directly with children and their families. She is passionate about research and development, and has been involved in public consultations to enable service improvement.
Wendy is currently a full-time mum. She has two children, one of whom has a clinical diagnosis of Dyskeratosis Congenita(DC). DC is a complex multi-system disorder that causes premature ageing to cells and organs, affecting approximately 1 in a million people. Wendy is a parent advisor for the patient organisation and charity, DC Action.