Due to the low patient population of each rare disease, living with one can be one of the most painful, devastating, and isolating experiences imaginable. We at Findacure want to change that. Findacure follows in the footsteps of William Bateson, the father of modern genetics, who reminded us we should “treasure our exceptions”. We are building a rare disease community that has the skills and knowledge to address patient needs, to drive research, and to develop treatments.
Findacure is a registered charity with the Charity Commission of England and Wales and a company limited by guarantee with Companies House.
Charity number: 1149646 Company number: 08174973
Our vision is a world in which all rare diseases have treatments – made together with patients, for patients.
We look to achieve our vision through focusing our work around two main aims:
To empower patient groups to build their patient communities, develop as a charity, and drive treatment development.
We believe that patient groups have a fundamental role to play in addressing patient needs and representing the patient voice in research. Any change for patient benefit must include patients in the process.
To promote collaboration between rare disease stakeholders to facilitate treatment development for all.
We truly believe collaboration between patients, clinicians, the pharmaceutical industry, medical professionals, and other interest groups, is necessary for the rare disease community to progress and confront its most pressing challenges. A unified rare disease community is certainly better than the sum of its parts.
Findacure was born out of the experiences of our founders, Nick Sireau and Tony Hall.
When Nick’s sons were born with the rare disease alkaptonuria (AKU), he was told there was no treatment available and he had no source of support to turn to. He quit his job to grow the AKU Society, a charity that reaches out to patients across the globe and is now in the late stages of a clinical trial for a promising treatment. But his journey was not easy and it is through his struggles in raising funds, setting up an international research consortium, and learning how to run a clinical trial that he realised the need for another organisation to help patient groups overcome these hurdles.
Nick teamed up with Tony Hall, a physician by training, who was establishing an organisation to support not-for-profit drug development for rare diseases. As an orphan drug development expert, Tony had seen first-hand the challenges pharmaceutical companies face developing treatments for small patient populations and the unsustainably high prices they had to charge to cover research costs. He realised the need for a non-profit approach to improve patient health while not breaking the NHS bank. Together they founded Findacure in 2012 in Cambridge, UK with the support of our board of trustees.
Findacure’s first employee, Flóra Raffai, was hired in late 2013 to develop the charity’s strategy and to run projects. Support for the charity has since been fantastic. We are now a team of four, running a diverse range of projects to meet the needs of our beneficiaries.
Dr Rick Thompson joined Findacure in 2015, after completing his PhD in Evolutionary Biology at the University of Cambridge. He oversees the charity’s strategy and development, leads on our drug repurposing social impact bond, oversees major funding applications, and manages other members of staff. He also works to encourage industry engagement with rare disease patient groups, promoting an open and collaborative approach to rare disease research.
Projects and Communications Officer
Libbie Read manages Findacure’s peer mentoring programme, training webinars, online resources portal and outgoing communications, including our website, social media, newsletter and press releases. Libbie also manages our volunteers and is responsible for outreach. She started at Findacure in 2015 after graduating from the University of Cambridge with a BA in Geography as the Fundraising and Communications Officer. She moved to her current role in September 2017.
Mary Rose oversees the project management of the charity’s events. She leads on the patient empowerment training workshops, our annual drug repurposing conference, and the rare disease showcase series. She is also responsible for securing corporate support for our collaboration events, and assists with the peer mentoring programme meet ups and community fundraising activities. Mary Rose has a BA (hons) degree in Politics from Keele University, and joined Findacure in mid-2016.
Katie is responsible for maximising Findacure’s revenue through a range of fundraising opportunities, including charitable trusts, corporate grants and sponsorship, and fundraising events. She works on building strong relationships with our supporters as well as developing Findacure’s community fundraising efforts. She graduated from Plymouth University with a BA (hons) in Illustration and has since worked for charities including Scope and British Heart Foundation.
Co-founder and Chairman
Dr Nicolas Sireau is the CEO of SolarAid, an international NGO bringing solar power to Africa. Nick is also Chairman of 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 the AKU Society. 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).
Co-founder and Director
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.
Julie Walters is an entrepreneur, who is now building her third business Raremark to help families affected by rare disease find credible information and connections. Julie also serves on the European regional council of the Entrepreneurs’ Organization, which helps leading entrepreneurs to learn and grow, and holds a BSc in molecular genetics from King’s College London.
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.
Professor Lakshminarayan Ranganath is a busy full time consultant at the Royal Liverpool University Hospital in the UK. There was no National Health Service available for rare disease Alkaptonuria in the UK when he developed an interest. At the time, systematic assessment of patients with this crippling disease was lacking. Exact numbers of AKU patients were unknown in UK and overseas. There was no effective treatment for AKU. To address these issues, he established an NHS Highly Specialised Services funded National Alkaptonuria Centre (NAC), employing off-label use of nitisinone. LRR is the inaugural Director of the NAC. Patients in the NAC are able to access 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.
Dr. Bruce Bloom is President and Chief Science Officer 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.
Cures Within Reach uses CureAccelerator™, the only global online repurposing research collaboration platform, to bring together clinicians, researchers, funders, and industry to create and conduct pilot clinical trials that drive more treatments to more patients more quickly.
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, the Findacure Fundamental Disease Charity, and Healx, LTD, 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.
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.
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.
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.
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 is a patient suffering from 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 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.
Claire Adams is a Specialist Severe Insulin Resistance & Diabetes Nurse and Senior Research Nurse based at Cambridge University Hospital NHS Foundation Trust. She has 9 years’ experience working with patients with rare diseases of metabolism and/or growth.
She has recently been awarded a grant to develop a programme of support designed for patients with lipodystrophy and will be working with the charity Changing Faces and the Foundation of Nursing Studies to do this. Claire has a particular interest in ensuring that healthcare professionals address the mental health and psychological needs of patients with rare disease.
David Bennett has worked in the Life Sciences sector for over 25 years, including time spent in the pharmaceutical industry and medical education agencies. His pharmaceutical career began at Beecham, later joining Kabi/Pharmacia at the launch of recombinant human growth hormone. Since leaving Pharmacia in 2012 he has worked on assignments for several orphan companies and specialise in this sector. Typical work during this time has centred around disease awareness programmes to aid identification and referral of patients with rare conditions.
An important part of his work has been to support the activities of patient support and advocacy groups. Access, in the broadest sense of the term, to the treatments and care required by patients with rare and difficult conditions has been a high priority.
He is very pleased to be an honorary member of the Turner Syndrome Support Society and a local volunteer representative for the Alzheimer’s society.
Deborah Mann is secretary of the rare disease patient group and charity; Organisation for anti-convulsant syndrome (OACS Charity). She is also the moderator the RareConnect Fetal Anti-Convulsant Syndrome page. She has experience in community politics, and retail management.
Deborah has been involved in the change of status of the drug sodium valproate from The Pharmacovigilance Risk Assessment Committee (PRAC) to the development of the valproate toolkit at the Medicines and Health Product Regulatory Agency (MHRA). This has led her to an involvement in the Patient Group Consultative Forum at the MHRA.
Jonathan is an experienced medical communications professional, specializing in rare diseases.
After graduating from Cardiff University in 2006 with a degree in Pharmacology and a PhD in Medical Biochemistry, Jonathan has spent his entire career working in the rare diseases field. Since 2012 he has held senior leadership positions within the medical communications field and has provided expert guidance to various stakeholders seeking to improve standards of care for patients with rare diseases.
Jonathan specializes in developing innovative, effective and cost-efficient communications strategies to identify and address the key issues that are unique to each rare condition. He has worked across more than 25 therapeutic areas and has supported the development of and access to some of the most innovative treatments for rare diseases in the recent years.
Alan Thomas is a patient engagement advocate for rare diseases in general and Ataxia in particular. 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.
Ataxia means ‘lack of order’.
People with ataxia have problems with movement, balance and speech. Alan is fighting to return order to the lack of order.
He was proud to receive a nomination from the Bevan Foundation in 2015 in the Health and Wellbeing category. Plus he participated in the Peer mentoring programme, run by Findacure, to progress Atacsia a Fi – Ataxia and Me©, an “on-line” resource for all affected by this with this “Life. Limiting” condition.
Findacure is a member of the following organisations: