GLIA-CTN Program Officially Awarded - What it Means for the PMD Community
This month the National Institute of Health (NIH) awarded The Global Leukodystrophy Initiative - Clinical Trial Network (GLIA-CTN) a 5 year $5 Million dollar grant. Our Partners at Children's Hospital of Philadelphia (CHOP) lead by Program Director Dr. Adeline Vanderver are co-leading this initiative with Dr. Ali Fatemi Program Director at Kennedy Krieger Institute in Baltimore, MD along with Dr. Florian Eichler Program Director at Massachusetts General in Boston, MA.
What does this mean for PMD? This federally funded grant is the type of grant that will move the needle for all Leukodystrophy Research including PMD. This grant will fund a collaborative project that will allow researchers to work together sharing data and sharing resources to accelerate research in the field.
Dr. Vanderver states “This is the first time that leukodystrophies have been included in the Rare Diseases Clinical Research Network. 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. 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.”
This collaborative effort is monumental news for PMD as well as ALL of the leukodystrophies as we will be able to accomplish more together than we ever could separately as rare orphan diseases.
This initiative should accelerate research that would typically move at a snail's pace due to a lack of resources as well as a lack of communication that typically happens when collaboration like this is not planned at the onset.
This is an exciting time for PMD as there is much happening in research as we get closer to Clinical trials for potential treatments and therapies. This is why The PMD Natural History Study that we recently committed $180,000 to fund is so important. We have been so pleased with the response of The PMD community to enroll in the Natural History Study. If you have not yet done so please visit the PMD Natural History Study page to learn more and register. “This is a way for all PMD families to participate in helping to accelerate PMD Research.” said PMD Board Chair, Dave Manley. “Your participation does not require any travel. You just need to release your son’s medical records to our Natural History Study team to include their data in the study.”
If you have any questions about this news or how to participate in the PMD Natural History Study, please contact the PMD Foundation at firstname.lastname@example.org.