COLUMBUS, OHIO (April 24, 2014) The Batten Disease Support and Research Association (BDSRA), in collaboration with international and family foundation partners, has announced grants totaling $408,000 to advance the development of treatments and cures for Batten disease.
Co-funders BDSRA-Australia, the Batten Disease Family Association of the United Kingdom, Noah’s Hope, and Hope 4 Bridget joined with BDSRA to support projects that span the United States, the United Kingdom, Australia, and Germany. Investigations to identify drug targets, drug discovery, and expand a patient registry have been funded through awards to six researchers: David Palmer, PhD, Lincoln University of New Zealand; Stephan Storch, PhD, University Medical Center Hamburg-Eppendorf, Germany; Angela Schulz, PhD, University Medical Center Hamburg-Eppendorf, Germany; Jonathan Cooper, PhD, King’s College, London; Jeffery Gerst, PhD, Weizmann Institute of Science, Israel; and Erika Augustine, MD, the University of Rochester.
The DEM-CHILD NCL Patient Database project led by Schulz is the first worldwide attempt to collect and analyze clinical data of all NCL forms through a consortium of NCL clinicians from 11 countries. Expanding a worldwide patient registry for Batten disease is a BDSRA strategic funding priority because of its necessity to understand the disease, identify novel biomarkers, advance clinical trials, and develop effective therapies.
With an emphasis on cutting-edge scientific inquiries to build on existing investigations in gene therapy, enzyme replacement, and drug discovery, BDSRA continues to identify and fund research with the highest potential to formulate treatments.
BDSRA and its co-funders select grant recipients through a scientific merit review process in which proposals are evaluated according to standards that screen significance of the project to advance NCL research, strategy to accomplish objectives, and potential for translation to treatments and cures.
As a rare, fatal, inherited disorder of the nervous system, Batten disease has no known treatment or cure. Batten disease is the most common, inherited neurodegenerative disorder found in children, and is also known as a group of disorders called Neuronal Ceroid Lipofuscinosis (NCL). There are multiple forms of the disease, which is most commonly diagnosed between infancy and school age.
BDSRA is the major support and research organization in North America for Batten disease. The first research grant from BDSRA was awarded in 1992, and since then more than $7.4 million has been allocated toward the search for treatments and cures. The organization is the largest provider globally for Batten disease patient and family support, education, research, and awareness programs.