NIH Grant to Support a Multi-Center Initiative to Advance Research, Treatment for Leukodystrophies

Published on in CHOP News

Hand holding dish under microscope Children’s Hospital of Philadelphia (CHOP) is co-leading an initiative to establish a multi-institutional program to advance research and treatment in leukodystrophy, a group of heritable movement motor function disorders caused by deficiencies in myelin, the protective layer surrounding nerve fibers. A newly announced five-year grant from the National Center for Advancing Translational Sciences (NCATS), part of the National Institutes of Health, supports the Global Leukodystrophy InitiAtive-Clinical Trial Network (GLIA-CTN).

“This Initiative brings together leading programs at a pivotal moment in leukodystrophy research, as we enter an era of new clinical trials for a set of related diseases that have previously been largely untreatable,” said Adeline Vanderver, MD, Program Director of the Leukodystrophy Center of Excellence at CHOP, and the principal investigator of the grant, issued under the auspices of the NCATS Rare Diseases Clinical Research Network.

Vanderver is collaborating with co-principal investigators from two other leading leukodystrophy centers: Ali Fatemi, MD, who directs the Moser Center for Leukodystrophy at the Kennedy Krieger Institute in Baltimore, and Florian S. Eichler, MD, director of the Leukodystrophy Service at Harvard Partners/Massachusetts General Hospital in Boston. In addition, other leukodystrophy programs and patient advocacy groups throughout the U.S. are involved in GLIA-CTN.

“This is the first time that leukodystrophies have been included in the Rare Diseases Clinical Research Network,” said Vanderver. “What is particularly exciting is that this our new consortium spans more than 30 different types of leukodystrophies, which have different genetic causes but largely overlap in clinical symptoms. Consolidating infrastructure and research in a large network provides greater numbers of patients for stronger statistical power, and avoids fragmentation and duplication of effort.”

Some leukodystrophies are more common in infants, while others are prevalent in young adults. The Network’s initial areas of research will compile natural history data, clinical outcome assessment measures, biomarkers, and long-term patient follow-up, to provide key information for innovative studies and clinical trials. CHOP, which will house the Data Integration Core for the Network, is currently conducting three clinical trials in leukodystrophy, and the Network’s data-gathering and research into disease mechanisms will help inform future clinical trials at other sites. “This collaborative Initiative offers the opportunity to increase public awareness of leukodystrophies,” said Vanderver, who added, “Ultimately, we aim to fundamentally transform treatment for this group of diseases.”