Findacure is building the fundamental diseases community to drive research and develop treatments. Findacure follows in the footsteps of William Bateson, the father of modern genetics, who reminded us that we should “treasure our exceptions”.
A world in which a vibrant fundamental disease community is collaborating towards treatments and cures.
1. To empower patient groups to become effective campaigners for change.
2. To facilitate the development of treatments for fundamental diseases.
3. To campaign for a receptive research environment that recognises the pivotal importance of fundamental diseases.
The idea for Findacure stemmed from Nick Sireau’s experience trying to find a cure for his children’s rare genetic disease alkaptonuria (AKU). As CEO of the AKU Society, faced with the uphill struggle of trying to raise funds, setting up an international research consortium, learning how to run clinical trials and support patients, Nick realised there was a huge need for practical, hands-on advice for patient groups to achieve their goals.
Nick teamed up with Tony Hall, a physician by training, who was looking to set up an organisation to support not-for-profit drug development for rare diseases. Together, they founded Findacure in 2012 in Cambridge, UK. Through fundraising and an a pledge of matched funding from the Myrovlytis Trust, Findacure’s first employee Flóra Raffai was hired in late 2013 to begin running training for patient groups.
Flóra Raffai oversees the charity’s strategy and development, leads on our patient group empowerment programmes, oversees major funding applications, and manages other members of staff. Flóra graduated from the London School of Economics with a BSc (hons) in International Relations. She is also a founding member and trustee of the Cambridge Rare Diseases Network, and a Chapter Co-Organiser of Good for Nothing Cambridge.
Dr Rick Thompson works primarily on our drug repurposing program, aiming to develop a program to promote the identification of existing drugs that could be used to treat rare diseases. Rick holds a PhD in Evolutionary Biology from Cambridge University, and has experience in palaeontology, genetics and evolutionary theory. He is working to apply his scientific experience to the treatment of rare diseases, and help communicate the science behind these illnesses to both the rare disease community and the general public.
Fundraising & Communications Officer
Libbie Read is responsible for maximising Findacure’s revenue through a range of fundraising opportunities, including charitable trusts, corporate sponsorship and fundraising events. She also spreads word about Findacure through print media, online communications and event-based marketing, and helps to update the website. She graduated from the University of Cambridge with a BA in Geography. Libbie has previously volunteered with Innovate South Africa and has ongoing voluntary commitments with Campus Children’s Holidays.
Mary Rose Roberts is responsible for the project management of the charity’s events, including our annual conference, workshops, peer mentoring scheme, networking events and fundraising activities. Mary Rose graduated from Keele University with a BA (Hons) degree in Politics and has previously been involved in event managing and fundraising as part of her University Trampoline Club.
Co-founder and Chairman
Dr Nicolas Sireau is Patient Engagement Director in Oncology at AstraZeneca. 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 CEO of the AKU Society and, beforehand, was the CEO of SolarAid, an international NGO bringing solar power to Africa. 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.
Dr John Solly is Director of the Myrovlytis Trust, a charity founded in 2007 to promote research into rare genetic disorders and advance education of the public in medical and molecular genetics. Previously, John worked at the management consultancy ZS Associates, advising clients in the biotech and pharmaceutical industries. He has a PhD in genetics from the Gurdon Institute at Cambridge University.
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.
Rob Forsyth is a Senior Lecturer and Consultant in Paediatric Neurology at Newcastle University UK. He trained in Cambridge, Oxford, Nottingham, Newcastle upon Tyne and Vancouver. His clinical interests include acute paediatric neurology, neurointensive care, and neurorehabilitation. His research interests centre on transitional research in the field of paediatric acquired brain injury and epilepsy. He was UK national training advisor in paediatric neurology 2003-9 and lead editor of the Oxford Specialist Handbook in Paediatric Neurology (2007; second edition 2012). He was on the editorial board of Developmental Medicine and Child Neurology 2005-2011 and is a Trustee of the UK Children’s Neurological Research Campaign (www.ukcnrc.org.uk). He is also a contributor to the Simulconsult paediatric neurology decision support project (www.simulconsult.com).
Anil Mehta is a clinical academic with a 50% split between teaching and research into the children’s disease cystic fibrosis, and working as a consultant paediatrician in neonatal medicine and paediatric cardiology. He focuses on patient care, health outcomes, the efficiency of engagement with orphan drug development in rare diseases and better informed policy making at an EU level. He has worked to improve the welfare of the most disadvantaged patients across Europe who suffer because their diseases are very rare in a given hospital setting. Anil implemented the first comprehensive UK-wide CF patient registry of ~ 7000 patients across 60 NHS CF clinics between 1995 and 2006 and recently developed the first 30,000 patient, 35-country pan-European demographic CF registry, with EU FP6 funding (2006-2009). He is now amalgamating registry data with genomic data.
Dr. Khan has pioneered the ‘drug repositioning approach’ namely the use of old drugs for other indications. These includes: a drug for Alzheimer’s disease; a drug for malaria; and he is developing novel treatments for ‘incurable’ rare genetic diseases such Alkaptonuria, giving hope to suffers of this debilitating disease. Dr. Khan has been awarded several Technology Strategy Board (TSB) grants totalling over £1M funding for technological innovation. The TSB is the UK government’s foremost agency which supports innovation and enterprise. Dr. Khan’s funding includes the use of new fluorescent proteins to diagnose cancer, the development of new temperature labels for drugs and the use of smartphones for diagnosing diseases. In 2013, Dr. Khan was shortlisted by BioNow as one of the most promising technologists in the North of England. He has been interviewed by The Guardian and by Bloomberg and holds a research position at Peterhouse College, University of Cambridge.
Tom Blundell is Director of Research and Professor Emeritus in the Department of Biochemistry, University of Cambridge. He has previously held teaching and research positions in the Universities of London, Sussex and Oxford.
In his academic lab in Cambridge Tom researches on molecular and structural biology of receptor activation, signal transduction and DNA repair. He has written extensive software for structural bioinformatics and developed new approaches to structure-guided and fragment-based drug discovery.
He is focusing on new ways of designing antimicrobials for tuberculosis and targeting protein-protein interactions for cancer therapeutics. He is also modelling mutations that occur in proteins in cancer, infectious diseases and fundamental diseases.
In 1999 Tom co-founded Astex Therapeutics, an oncology company that has eight drugs in clinical trials and was sold as Astex Pharma to Otsuka in 2013. He was founding CEO, BBSRC (1991-1996); Chair, Royal Commission on Environment (1998-2005); Deputy Chair, Institute of Cancer Research (2008-) and President, UK Science Council (2011-).
Professor Jim Gallagher holds the Derby Chair of Anatomy at the University of Liverpool. Jim obtained his PhD from Cambridge on vitamin D metabolism and worked in Bern on bisphosphonates. In Sheffield, he and Jon Beresford developed the first techniques to culture human osteoblasts. In 1984 he moved to UCL and in 1986 to Liverpool. Jim has published over 120 full peer-reviewed publications. He has an outstanding record in academic leadership and has supervised 25 PhD students, 9 of whom hold academic positions in UK universities. His group pioneered research on P2 receptors bone. In 2006 Jim started working with Lakshminarayan Ranganath and Jonathan Jarvis on the pathogenesis of ochronosis in alkaptonuria (AKU), supported by the AKU Society UK. They developed in vitro, ex vivo and mouse models of ochronosis, which helped elucidate the mechanism of joint destruction in AKU and showed that nitisinone is effective in preventing joint disease. This work has recently culminated in a EUFP7 grant to undertake a clinical trial on nitisinone with “DevelopAKUre”, a consortium of European researchers and patient groups co-ordinated from Liverpool.
Bruce Bloom is President and Chief Science Officer of Cures Within Reach, a 501c3 that has been saving lives since 2005 by facilitating the repurposing of human approved drugs and devices to deliver more than a dozen fast, safe and affordable treatments and cures for a wide variety of diseases that had no effective therapy. He holds a JD from Chicago Kent College of Law, a DDS from University of Illinois Medical Center, and a BS in Biology from University of Illinois Urbana.
Dr. Bloom was elected an International Ashoka Social Entrepreneur Fellow in 2010 and currently services as a Science Advisor to the Marian Falk Trust, Chair of the Science Advisory Board of Rediscovery Life Sciences, Trustee of the Kendall College Charitable Trust, member of the Board of Councilors of Midwestern University, Board member of the Judy Hirsch Foundation, and Client Advisor to Northwestern Mutual Financial Network. Dr. Bloom is a host of the Clinician’s Roundtable heard on ReachMD.com and on XM 161, and is a facilitator for Pathways to Successful Living.
Professor Ranganath specialises in Chemical Pathology & Metabolic Medicine, with extensive experience acquired both in India and UK. He has research interests in the field of Gastro-intestinal tract hormones having obtained his PhD from the University of Surrey while researching gut hormones in relation to obesity. Professor Ranganath founded the Alkaptonuria (AKU) Society in 2003 and established Liverpool as the International Centre for AKU. Along with a team of like-minded individuals he has helped create a National Centre funded by the Department of Health for AKU as well as co-ordinating clinical trials in AKU in the EC-funded project DevelopAKUre. He is a busy Clinician in a teaching hospital in the Department of Clinical Biochemistry Metabolic Medicine and also has responsibility for leading the Mass Spec section of the laboratory. He is also the Training Programme Director for Chemical Pathology and Metabolic Medicine for the Mersey Deanery.
Nathalie Kayadjanian, Ph.D is the founder and director of TransBioMed solutions LLC, an international consulting company whose mission is to help organizations accelerate the development of innovative therapies for patients with rare diseases. Prior to founding TransBioMed solutions, Dr. Kayadjanian was associate science director of the French Association against myopathies (AFM). She was also associate director of biomedical research of the Amyotrophic Lateral Sclerosis Association (ALSA) in the US. Dr. Kayadjanian received her Ph.D in Neuroscience from the University Pierre and Marie Curie in Paris. She has extensive R&D experience in academia, biotech, and pharmaceutical industry in Europe and in the USA.
Andrew Tee is a Senior Lecturer at Cardiff University. His main research strength is with uncovering new cell signalling mechanisms linked to genetic disorders at the protein level. Such fundamental work is necessary to better understand human disease, and he emplosy genetic models to do this, such as Tuberous Sclerosis Complex (TSC) and Birt-Hogg-Dubé (BHD). Recently, Andrew’s findings were translated into the clinical setting for the treatment of TSC patients with the use of the mammalian target of rapamycin (mTOR) inhibitor, and rapamycin. He has also become very interested in BHD, as the features observed are similar to that seen in TSC patients, but the tumour suppressor function of BHD is unknown. His team believe that BHD is necessary for maintaining cell homeostasis, where loss of function of BHD leads to tumour progression, and that BHD is involved in many sporadic cancers.By understanding these fundamental genetic diseases, his research team hopes to uncover new therapeutic strategies that will impact health care for both TSC and BHD, but where these strategies will also be transferable for other human diseases.
Jonathan Jarvis studied Physics and Physiology at the University of London, and completed his doctorate on activation of muscle in Duchenne muscular dystrophy at Imperial College. He was then Reader in Human Anatomy and Cell Biology in the School of Biomedical Sciences at the University of Liverpool and led the development of the AKU mouse model project. He is now Professor in Sport and Exercise Science at Liverpool John Moores University.
Oliver Timmis is the Head of Projects for the AKU Society. He graduated from Cambridge University with a BA(hons) in Natural Science (Physiology, Development and Neuroscience) and has experience in science communication, including through BioNews, a news website focusing on healthcare and genetics. Oliver represents the AKU society on several groups, including the Drug Information task force at EURORDIS, the Specialised Healthcare Alliance, the UK Genetic Testing Network and Rare Disease UK.