Press Release (ePRNews.com) - SKOKIE, Ill. - Mar 20, 2017 - Cures Within Reach, the leading global nonprofit focused on repurposing research as a fast track to saving patient lives, today announced the recipients of its 5th Annual Global Health Repurposing Awards. The 2017 Global Health Repurposing Awards’ (GHRA) recipients are: Timothy P. Walbert, Chairman, President and Chief Executive Officer of Horizon Pharma plc; Berish Rubin, PhD and Sylvia Anderson, PhD, Head and Director, Laboratory for Familial Dysautonomia Research, Fordham University; Dr. Michael Tranfaglia, Medical Director and Co-Founder of FRAXA Research Foundation; and Dr. Lieven Lagae, Director of the Childhood Epilepsy Program at the KUL University Hospitals.
Award recipients will be recognized at the Global Health Repurposing Awards on June 27, 2017 at the Radisson Blu Aqua Hotel in Chicago, IL. Awardees are also invited to attend and to present at the 6th Annual Global Health Drug Repositioning and Repurposing Conference, held in conjunction with the Global Health Repurposing Awards.
The Awards honor leaders in business, science, medicine and philanthropy whose achievements have made a difference in patients’ lives through repurposing research that finds new uses for existing drugs, devices and nutraceuticals. This year the GHRAs have a rare disease focus, to commemorate Cures Within Reach’s first repurposing success, which found that the drug sirolimus created a life-saving treatment for a rare disease called autoimmune lymphoproliferative syndrome.
There are four awards being bestowed at the 2017 GHRA: the Cures Within Reach Patient Impact Legacy Award (business); the Janet Davison Rowley Patient Impact Research Award (science), the Golan Christie Taglia Patient Impact Philanthropy Award (philanthropy) and the Cures Within Reach Patient Impact Clinical Award (medicine).
“Cures Within Reach is excited to recognize these leaders who continue to advance repurposing research for rare diseases,” said Dr. Bruce Bloom, President and Chief Science Officer at Cures Within Reach. “There are over 7,000 unsolved rare diseases, and 350 million people worldwide suffer from some type of rare disease. Our mission is to work with the business, scientific, medical and philanthropic communities to positively impact rare disease patients through repurposing.”
The Cures Within Reach Industry Patient Impact Research Award is being presented to Timothy P. Walbert, Chairman, President and Chief Executive Officer of Horizon Pharma plc, for his commitment to pursuing treatments for rare diseases, and his belief that repurposing is part of the solution. Tim has guided Horizon from an idea to a successful biopharmaceutical company in a short period of time. Moreover, he and his team at Horizon are laser focused on the rare disease community.
The Janet Davison Rowley Patient Impact Research Award is being conferred upon Drs. Berish Rubin and Sylvia Anderson, who jointly direct the Laboratory for Familial Dysautonomia (FD) Research at Fordham University. Drs. Rubin and Anderson and their team discovered the gene mutation for Familial Dysautonomia (FD), a rare disorder that impacts the development and function of the autonomic nervous system in affected individuals because of the loss of functional IKAP protein. They were then able to restore IKAP function through repurposing 1) two bioactive agents from soy; 2) derivatives of Vitamin A and E; and 3) EGCG, a bioactive agent found in green tea. Their repurposing research has been published in the numerous journals, including Molecular Nutrition & Food Research.
The Golan Christie Taglia Patient Impact Philanthropy Award is awarded to the FRAXA Research Foundation, a nonprofit committed to finding a cure for Fragile X syndrome. Fragile X is a rare disease that is the most common known cause of autism worldwide. Dr. Michael Tranfaglia, FRAXA’s co-founder, will be accepting this award on his organization’s behalf. FRAXA has invested significantly in repurposing research, and Dr. Tranfaglia’s son, who has Fragile X, is treated with a repurposed therapy.
This year, Dr. Lieven Lagae of KUL University Hospitals, a pediatric neurologist, will receive a new award, the Cures Within Reach Industry Patient Impact Clinical Award, for his research on Dravet Syndrome, a severe seizure disorder. Dr. Lagae is a pioneer in research repurposing the drug fenfluramine for Dravet. This drug was part of the Phen-Fen diet drug that was taken off the market, and fenfluramine, which was helping Dravet patients, was no longer available. Dr. Lagae has doggedly fought to bring Dravet patients access to this drug, and is leading a worldwide effort to secure regulatory approval for fenfluramine so that it will help stop these debilitating seizures.
To attend the 2017 GHRA and honor these heroes, visit www.cureswithinreach.org/ghra-2017.
ABOUT CURES WITHIN REACH
Cures Within Reach (http://www.cureswithinreach.org) works to catalyze repurposing research to quickly and affordably improve patient lives. We accomplish this by connecting funders with researchers to jumpstart repurposing research clinical trials, by providing collaboration tools so repurposing stakeholders can work together more easily, and by pioneering alternative finance engines and incentives for repurposing research. Cures Within Reach’s repurposing research projects have generated over a dozen “new” treatments making patient impact through off-label use in clinical practice or through a commercialization track. Cures Within Reach currently has 24 repurposing research projects either funded or approved for funding.
Cures Within Reach built CureAccelerator (http://www.cureaccelerator.org) to provide a global collaboration space and marketplace for repurposing research that can deliver effective solutions to unsolved diseases. The CureAccelerator platform was built with a grant from the Robert Wood Johnson Foundation, to provide a platform in which drugs, devices and nutriceuticals approved for one or more human diseases can be repurposed to create “new” treatments in other diseases. CureAccelerator has over 1,000 users who have proposed 120 projects, of which more than 20 have received funding and are moving towards patient impact.
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