United Front Again

Clinical and Basic Research Scientists Fighting Krabbe’s Disease

The Legacy of Angels Foundation sponsors The Krabbe Translational Research Network 3rd Annual Meeting • Biltmore Hotel, Coral Gables, Florida

For nearly 3 full days, the Krabbe’s Translational Research Network, a consortium of the leading scientists and clinicians who are dedicated to reducing the time it takes for research discoveries to become clinical therapies, met to fight the rare childhood disease, Krabbe’s. Along with related world-renowned experts and clinical practitioners, these members work together, integrating knowledge from clinical experience and basic science and developing unique studies to bring the latest research into the clinic where it can improve the lives of children with Krabbe’s Disease. The Goal of the latest meeting, which was held in the inspiring surroundings of the historic Biltmore Hotel in Coral Gables, Florida, was to share knowledge about Krabbe disease by increasing communication among researchers and identifying specific projects that have the potential to develop into new therapies for patients.

The Legacy of Angels Foundation, founded by Sue and Paul Rosenau, after their family experienced the tragic death of a grandchild due to Krabbe's, sponsored the program. The Rosenaus have stated, 

We wanted to focus our philanthropy on the education and awareness of this disease and ultimately help fund research for better treatments and improved health outcomes for those now and in the future with Krabbe’s disease.

Some topics that were addressed, included: Natural Progressions and Treatment Outcomes of Krabbe’s Disease, Unresolved Questions; Hematopoietic Stem Cell Transplantation for Krabbe’s Disease; Neuroimaging, What a Scan Shows and Does Not Show; Molecular Mechanisms of Demyelination and Gene Therapy in Peripheral Nerve Disease, Combining Cellular and Molecular Analysis with Unbiased Drug Discovery to Identify Potential New Treatments for Krabbe’s Disease; and Combination Therapy.

Maria L. Escolar, M.D. who directs The Program for the Study of Neurodevelopment in Rare Disorders (NDRD) at the University of Pittsburgh Medical Center and has created a Virtual Medical Home and Clinic for Children with Krabbe's Disease located on the web at: http://ndrdvirtualmed.com/index.shtml hosted the meeting. More than twenty-nine doctors, researchers, clinicians, and family members participated.

When asked to summarize the 2013 meeting Dr. Escolar noted,

We have now several laboratories that in the past worked independently, successfully collaborating and sharing knowledge towards improving therapy. The experiments and the challenges have been discussed, resulting in a more in depth understanding of Krabbe disease. Clinicians have helped basic scientist refocus their research to make their experiments more relevant to clinical challenges that will ultimately benefit patients.