Elsevier Supports Findacure to Address Rare Disease Challenge

Working with UK charity to offer informatics expertise in drug repurposing programme for rare diseases

New York February 29, 2016

Elsevier, a world-leading provider of scientific, technical and medical information products and services, today announced it is working with Findacure to identify drug repurposing candidates for the rare disease Congenital Hyperinsulinism (CHI).

Elsevier R&D Solutions will provide pro bono 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.

Findacure is a UK-based charity whose mission is to build the rare disease community to drive research and develop treatments. Findacure and Elsevier will start their collaboration by focusing on CHI, an ultra-rare disease where new-borns have a permanently excessive level of insulin in the blood, which can lead to brain injury or death if not caught quickly. For the most severe cases of CHI, the only current viable treatment is the removal of the pancreas (near total pancreatectomy), consigning the patient to a lifetime of diabetes. Elsevier will support Findacure in battling CHI by analyzing the disease’s biological pathways and structure, as well as by looking at already published literature for further insights into the disease. Moreover, Elsevier will help Findacure understand the current research landscape and affiliations for CHI, helping Findacure build its network with authors and institutions.

This analysis will be conducted using tools such as Pathway Studio, which enables the study and visualization of disease mechanisms, gene expression, and proteomics and metabolomics data, to assess CHI’s biological make-up in depth. Elsevier will then shortlist the most promising potential treatments that could be repurposed safely and effectively, supporting Findacure to create a strong evidence base for existing generic drugs that can be progressed to proof-of-concept phase II clinical trials, with the ultimate aim of repurposing those drugs in the treatment of CHI.

“CHI is a terrible disease that greatly affects its young patients – as an ultra-rare disease, treatment options are currently very limited,” said Dr. Rick Thompson, Scientific Officer at Findacure. “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.”

“For the pharmaceutical industry as a whole, addressing the challenge of rare diseases is high on the agenda. But for the patients and their families, rare diseases can be very isolating and it can often feel like the potential for new treatments is low,” said Tim Hoctor, Vice President, Life Science Solutions Services, Elsevier R&D Solutions. “When Elsevier was first introduced to Findacure and its mission, we were very pleased to help this talented group with their project. Further, working on this kind of difficult project helps us become even more attuned to the needs of the research community and increases our scope through which we can service research into rare and orphan diseases. In the future, this kind of collaboration between different partners involved in the research, development and treatment of rare diseases, is the best way of addressing the industry-wide decline in pharmaceutical R&D productivity. Our goal, is that through this project, we can work together with Findacure to offer hope of a future treatment for CHI suffers and their families.”


About Findacure
Findacure is a UK-based charity building the rare disease community to drive research and develop treatments. Findacure works to empower patient groups to meet the challenges of their conditions and to promote collaboration between rare disease stakeholders to facilitate treatment development for all. The charity is setting up a social impact bond for rare disease drug repurposing to provide a platform to fund promising projects identified by patient groups, clinicians, and researchers. The bond will fund ten rare disease drug repurposing clinical trials, with the aim of delivering new treatments to the patients that need them. Congenital Hyperinsulinism (CHI) is the first condition being considered for this programme.

About Elsevier
Elsevier is a world-leading provider of information solutions that enhance the performance of science, health, and technology professionals, empowering them to make better decisions, deliver better care, and sometimes make groundbreaking discoveries that advance the boundaries of knowledge and human progress. Elsevier provides web-based, digital solutions — among them ScienceDirect, Scopus, Research Intelligence and ClinicalKey— and publishes over 2,500 journals, including The Lancet and Cell, and more than 35,000 book titles, including a number of iconic reference works. Elsevier is part of RELX Group, a world-leading provider of information and analytics for professional and business customers across industries. www.elsevier.com

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