CureDuchenne, a global leader in research, patient care, and innovation in improving and extending the lives of those living with Duchenne muscular dystrophy (Duchenne), announced today it has initiated the development of a biobank for Duchenne. The CureDuchenne Biobank is dedicated to empowering Duchenne research and the search for a cure for Duchenne that is independent and accessible to all researchers.
In partnership with Dr. Tahseen Mozaffar of the University of California, Irvine (UCI) for strategic development and implementation, and with RUDER Infinite Biologics (RUCDR), the worlds largest university-based biorepository, for biobanking services, the CureDuchenne Biobank plans to begin sample collecting immediately at CureDuchenne Cares events around the country.
CureDuchenne is committed to supporting projects that could lead to a cure for Duchenne. But with limited resources to conduct research, scientists at academic institutions, biotechnology and pharmaceutical companies are not able to conduct the work necessary to find a cure for every child with Duchenne, said Debra Miller, founder and CEO, CureDuchenne. To fill that gap, its important to provide researchers with free and unrestricted access to the cell lines they need for Duchenne research, Miller added.
Advances in precision medicine rely on open access biobanks with human biological specimens for analysis with the latest technologies long after the time of their collection, said Dr. Andrew Brooks, chief operating officer, RUDCR Infinite Biologics. We are thrilled to add Duchenne to the growing list of biobanks to help cure genetic diseases like Duchenne, Dr. Brooks added.
Minimally invasive blood and skin tissue samples will be requested on a voluntary basis from patients and, if possible, from their unaffected immediate blood relatives (parents, siblings or adult children) who may be interested in supporting scientific studies. To help ensure that Duchenne patients from all population groups can potentially benefit from the research, Duchenne patients from diverse age ranges, ambulatory status, genetic mutations and racial, cultural and socioeconomic groups are strongly encouraged to participate in this initiative.
Only 5% of rare diseases have a treatment approved by the FDA, mostly due to the lack of high quality biospecimens for research, said Dr. Mozaffar, professor and interim chair, Department of Neurology, UCI School of Medicine. Human specimens have long been used for translational purposes in cancer research, to investigate disease pathogenesis, to test scientific hypotheses, and to assess biomarkers identified in experimental studies, Dr. Mozaffar added.
By donating specimen to the dedicated CureDuchenne Biobank, families will: enable the development of patient cell lines specialized to their childs gene mutation that can be used by any researcher to develop customized treatments; contribute to genetic studies to understand how any of the mutations in the body affect disease progression and response to therapies; and contribute to research investigating how to harness the immune response to allow for more effective treatment.
The proposed CureDuchenne Biobank board will consist of experts in biorepositories and registries, technology transfer, genetics, neuromuscular disease, immunology and ethical, legal and social issues. Dr. Tahseen Mozaffar and Dr. Armando Villalta, assistant professor, Department of Physiology & Biophysics, UCI School of Medicine are leading the CureDuchenne Biobank board. Together with CureDuchenne executives, the governing board will determine access to the CureDuchenne Biobank collection. Deidentified data and samples will be released to approved researchers in Duchenne.
Having access to patient cells is the first step in pursuing precision medicine initiatives that will enable us to facilitate families getting involved and engaged in research, and ensure all stakeholders are able to conduct research for all individuals living with Duchenne, said Romina Foster-Bonds, director of programs, CureDuchenne and lead for the CureDuchenne Biobank.
CureDuchenne is the nations leading nonprofit organization dedicated to finding a cure for Duchenne, the most common and lethal form of muscular dystrophy. As the leading genetic killer of young boys, Duchenne affects more than 300,000 boys living today. CureDuchenne has garnered international attention for its efforts to raise funds and awareness for Duchenne through venture philanthropy. For more information on how to help raise awareness and funds needed for research, please visit www.cureduchenne.org.
CureDuchenne Cares workshops around the country will utilize the UCI clinical team to collect samples from patients all over the country. Patients interested in learning more or participating, please contact Romina Foster-Bonds, director of programs at: [email protected]
About RUCDR Infinite Biologics
RUCDR offers a complete and integrated selection of biological sample processing, analysis and biorepository services to government agencies, academic institutions, foundations and biotechnology and pharmaceutical companies within the global scientific community. RUCDR provides DNA, RNA and cell lines with clinical data to hundreds of research laboratories for studies on mental health and developmental disorders, drug and alcohol abuse, diabetes and digestive, liver and kidney diseases. RUCDR completed an $11.8 million expansion and renovation of its facilities in May 2013. Read more at www.rucdr.org.
About the University of California, Irvine:
Founded in 1965, UCI is the youngest member of the prestigious Association of American Universities. The campus has produced three Nobel laureates and is known for its academic achievement, premier research, innovation and anteater mascot. Led by Chancellor Howard Gillman, UCI has more than 36,000 students and offers 222 degree programs. Its located in one of the worlds safest and most economically vibrant communities and is Orange Countys second-largest employer, contributing $5 billion annually to the local economy. For more on UCI, visit www.uci.edu.