Baltimore’s Kennedy Krieger Institute is getting an $8.17 million grant from the National Institutes of Health to establish and lead a first-of-its-kind network in the United States dedicated to advancing treatments for Batten diseases, a group of rare pediatric neurodegenerative disorders.
The new Kennedy Krieger’s Batten Disease Center of Excellence will serve as the headquarters and coordinating center for the 14-site Batten Disease Clinical Research Consortium (BDCRC). Dr. Erika Augustine will serve as director of the new center, which will be the scientific and administrative hub for the BDCRC network.
“The BDCRC represents a major opportunity to close critical gaps in knowledge about Batten diseases and readiness to design and launch clinical trials,” said Augustine, the Associate Chief Science Officer and Director of the Clinical Trials Unit at Kennedy Krieger Institute, in a statement. “By uniting leading academic centers and advocacy partners, we are laying the groundwork for meaningful advances.”
Batten diseases are also called neuronal ceroid lipofuscinoses. They are inherited neurogenetic disorders. These diseases often cause vision loss, motor and cognitive decline, seizures, and sometimes premature death. More than a dozen subtypes of Batten disease exist, most of which do not have effective treatments. The Centers for Disease Control says that in the U.S., 2-4 births per 100,000 are affected by Batten disease, though researchers suggest that estimate is low.
Kennedy Krieger will also join the Rare Diseases Clinical Research Network (RDCRN), and will work closely with Our Promise to Nicholas Foundation, and Batten Disease Support, Research, and Advocacy Foundation (BDSRA). This is to make sure patient perspectives and experiences are emphasized.
Amy Fenton Parker, president and CEO of BDRSA Foundation, feels this will help guide research priorities.
“This grant is extraordinary news for the Batten community,” Parker said in a statement. “The BDSRA Foundation is thrilled to collaborate with Kennedy Krieger and the BDCRC to help keep the Batten community’s research priorities front and center, while new ideas are pursued toward unmet needs for clinically relevant and effective treatments.”
This project is funded by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), the National Institute of Neurological Disorders and Stroke (NINDS), and the National Center for Advancing Translational Sciences (NCATS).
BDCRC sites include Boston Children’s Hospital, Children’s Hospital Colorado, Children’s Hospital of Orange County, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Nationwide Children’s Hospital, NYU Langone Health, Rush University, Sanford Research, University of Michigan, University of North Carolina at Chapel Hill, University of Rochester, and Washington University in St. Louis.
A national Batten disease biorepository will also be launched to provide a foundation for future advances in treatments. A biorepository is a facility that collects, catalogs, and stores samples of biological materials for lab research. The material can be blood, tissue, cells, DNA, RNA, and protein from humans, animals, or plants. If the sample is from a person, their medical information may also be stored there, as would written consent from them for their samples to be used in laboratory studies.
The Rare Diseases Clinical Research Network (RDCRN) will post updates on the project, with work expected to begin soon. Kennedy Krieger Institute’s website has information about additional Batten disease research already underway there.
