From the Wires
HemoShear and Children's National Health System to Unveil New Paradigm for Accelerating Rare Disease Drug Discovery
~ Presentation Featured at Partnering for Cures Conference
By: PR Newswire
Nov. 17, 2014 07:00 AM
CHARLOTTESVILLE, Va., Nov. 17, 2014 /PRNewswire/ -- HemoShear and Children's National Health System (CNHS) together will present at the 6th annual Partnering for Cures conference to be held November 16-18, 2014 in New York City. Marshall Summar, MD, Division Chief of Genetics and Metabolism at CNHS, and Brian Wamhoff, PhD, Vice President of R&D and Co-founder of HemoShear, will introduce a new paradigm for enabling discovery of new rare disease treatments in less time and with higher success rates.
Drs. Summar and Wamhoff will describe how Children's knowledge of disease genetics and access to patients, in combination with HemoShear's translational rare disease tissue systems, have the potential to uncover the biology of liver-focused rare diseases and to predict patient responses to new drug candidates. This new paradigm is a major advancement over traditional drug R&D approaches, such as animal models, because they do not mimic human rare disease biology, leading to high failure rates and high costs for very small patient populations.
"At Children's National, we treat more patients with rare diseases than nearly anywhere else in the world," said Dr. Summar. "Because we have few or no available drug therapies for most of the 7,000 rare genetic conditions, we are forced to treat the patients symptomatically, day to day, without addressing the underlying problem. For our metabolic rare disease patients, there simply are few reliable drugs. For many, a liver transplant is often the last therapeutic alternative. Even then our patients' lifespans can be tragically short."
"Our ability to accurately recreate liver rare diseases will be a major step forward in accelerating discovery and development of new treatments," said Dr. Wamhoff. "With the HemoShear platform, we expect to be able to unmask meaningful mechanisms behind these diseases, identify new targets and new drug candidates, and accelerate translation of optimal candidates into the clinic for children in need."
"The presentations at Partnering for Cures will address some of the thorniest issues in medical research using models that can be scaled and translated across diseases," said FasterCures' Executive Director Margaret Anderson. "From re-imagining clinical trial infrastructure to improving and expanding data sharing, to creating the tools and resources needed to translate basic science into cures, they are accelerating the path from lab to market for novel – and needed – therapies."
Drs. Summar and Wamhoff will present the Rare Disease Drug Discovery Accelerator at the conference on Tuesday, November 18 at 9:00 am. Their presentation is one of only thirty innovative approaches selected from 120 submissions.
Partnering for Cures is convened by FasterCures and the Milken Institute. More than 1,000 healthcare leaders, policy makers, investors and medical researchers will come together at the conference to share creative approaches to speed scientific discoveries and new therapies to patients. For more information about the conference, go to http://www.partneringforcures.org.
About Children's National Health System
About the Division of Genetics and Metabolism at Children's National
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