Kennedy Krieger Institute Joins Multi-Center Initiative to Advance Research, Treatment for Leukodystrophies

tags: Center for Leukodystrophies

NIH grant to fund Global Leukodystrophy InitiAtive-Clinical Trial Network

BALTIMORE, OCTOBER 9, 2019Kennedy Krieger Institute’s Chief Medical Officer and Director of the Institute’s Moser Center for Leukodystrophies, S. Ali Fatemi, MD, MBA, is co-leading an initiative to establish the Global Leukodystrophy InitiAtive-Clinical Trial Network (GLIA-CTN). A newly-announced five-year grant from the National Center for Advancing Translational Sciences (NCATS), part of the National Institutes of Health, will support the network, which will work 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.

Fatemi is partnering with principal investigators, Adeline Vanderver, MD, program director of the Leukodystrophy Center at the Children’s Hospital of Philadelphia (CHOP), and Florian S. Eichler, MD, director of the Leukodystrophy Service at Harvard Partners/Massachusetts General Hospital in Boston, as well as other leukodystrophy programs and patient advocacy groups in the U.S., to launch the Network, which will span more than 30 different types of leukodystrophies and serve as a consolidated infrastructure for leukodystrophy research.

“This consortium will dramatically enhance the collaboration between existing leukodystrophy centers nationally by allowing us to work together to utilize innovative tools and share data for critical research and clinical trials,” said Fatemi. “This network will transform the way we gather knowledge about this group of diseases and ultimately impact the way we treat patients.”

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. The increased network of resources, research and patients will provide more accurate statistical power and avoid duplication and fragmentation.

For more than 30 years, the Institute has been globally recognized as an authority on the study and care for patients diagnosed with a wide range of leukodystrophies. The Moser Center provides comprehensive care to patients with leukodystrophies through an interdisciplinary approach, bringing together the fields of neurogenetics, genetic counseling, neurorehabilitation, endocrinology, and urology, along with physical, occupational, speech and aquatic therapy. The Center also works collaboratively with other leukodystrophy programs across the country and around the world to ensure that every patient receives the best possible care.

About Kennedy Krieger Institute:  

Internationally recognized for improving the lives of children and adolescents with disorders and injuries of the brain, spinal cord and musculoskeletal system, Kennedy Krieger Institute in the greater Baltimore/Washington, D.C. region serves 25,000 individuals a year through inpatient and outpatient clinics, home and community services, and school-based programs. Kennedy Krieger provides a wide range of services for children with neurological issues, from mild to severe, and is home to a team of investigators who are contributing to the understanding of how disorders develop, while at the same time pioneering new interventions and methods of early diagnosis.