Participant Profile: Findacure
UK-based charity Findacure is building the rare disease community to drive research and develop treatments.
The charity is bringing hope to patients and helping to give them a voice in their own treatment. One of Findacure’s key initiatives is the development of a drug repurposing programme that aims to find new ways to use old drugs to treat rare disease, hopefully saving lives. By quantifying the current cost to the UK’s National Health Service (NHS), Findacure are aiming to revealing potential savings through the use of repurposed drugs, and use this as an incentive to drive forward the key research that rare disease patients need.
Findacure and Elsevier have joined forces to focus efforts on the rare disease congenital hyperinsulinism (CHI), where newborns have permanently excessive levels of insulin in the blood, which can lead to brain injury or death if not caught quickly. Working together Findacure and Elsevier will analyze the disease’s biological pathways and structure, and look at published literature for further insights into the disease.
Meet the Findacure Team
Dr. Rick Thompson
Head of Research, Findacure
Dr Rick Thompson works primarily on drug repurposing, aiming to develop a programme to promote the identification of existing drugs that could be used to treat rare diseases.
CHI is a terrible disease that greatly affects its young patients – as an ultra-rare disease, treatment options are currently very limited. We are excited about the opportunity that working with Elsevier gives Findacure to advance our plans in repurposing research. The benefits of this collaboration are huge. By understanding the mechanisms of a disease and demonstrating that a generic drug could have a clear healthcare benefit to that patient population, we can more easily raise funding for crucial clinical trials and uncover new low cost treatments for rare disease patients. If successful, this approach could also be replicated for other rare diseases. Ultimately, by building a vibrant rare disease community, we aim to be able to offer new, safe treatments to CHI patients and their families.
Dr Nicolas Sireau
Co-founder and Chairman
Dr Nicolas Sireau has recently returned to the role of CEO of SolarAid, an international NGO bringing solar power to Africa – a charity that he helped to found before his work on rare diseases. 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 spent five years as the CEO of the AKU Society. He is the editor of ‘Rare Diseases: Challenges and Opportunities for Social Entrepreneurs’ (Greenleaf 2013).
Flóra Raffai was the first hire at Findacure; developing the charity’s projects, funding, and community. Now as Executive Director, Flóra organises Findacure’s patient group empowerment programmes, develops the charity strategy, oversees major funding applications, and manages other members of staff. Flóra is also a founding member and trustee of the Cambridge Rare Diseases Network, and a Chapter Co-Organiser of Good for Nothing Cambridge.
Prof. Khalid Hussain
GOSHCC Professor in Paediatric Metabolic Endocrinology
Honorary Consultant Paediatric Endocrinologist Genetics and Epigenetics in Health and Disease Genetics and Genomic Medicine Programme UCL Institute of Child Health Great Ormond Street Hospital for Children, UK. Prof Hussain leads the NHS specialist service for Hyperinsulinism at Great Ormond Street Hospital, as well as actively researching this disease.
Findacure on Pharma R&D Today
Using Text Mining to Find Treatments for Rare Diseases
Posted on May 5th, 2016 by Maria Shkrob in Pharma R&D
The Power of Collaboration: Elsevier and Findacure
Elsevier R&D Solutions will provide complimentary informatics expertise and advice, as well as access to published literature through its online tools for a period of three years, to help researchers understand the mechanisms of the disease. Working with Findacure and their collaborators, Elsevier will analyze and interpret data to identify existing generic pharmaceuticals with therapeutic potential for the treatment of CHI.
I was well aware of Elsevier’s role in publishing. But I didn’t know about the professional services side and all the data mining, analyses and research they could do. When we fully understood the kind of support being offered to our small charity, it blew us away. Dr Rick Thompson , Findacure
I’m particularly eager to expand on our work alongside dedicated life science researchers to make a demonstrable impact. Our team at Elsevier is energized by this collaboration, which clearly shows the power of data and the transformation from information to insights. Tim Hoctor, Findacure
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