Krabbe Translational Research Network

The Krabbe Translational Research Network (KTRN) is a consortium of scientists and clinicians who are dedicated to reducing the time it takes for research discoveries to become clinical therapies.To achieve this goal, members work together, integrating knowledge from clinical experience and basic science and developing unique studies.

The second KTRN meeting was held March 12–15, 2012 in Pittsburgh, Pa. The goal of the meeting 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.

Among the research discussed were:

• The latest findings on genetic mutations in patients who screen positive for Krabbe disease and those who present with symptoms
• Researchers shared what is currently known about the genetic mutations that cause the disease and how these mutations may influence enzyme activity and cause disease in the nervous system.
• There was active discussion about the structure of GALC enzyme, genetic modifiers, gene therapy advances, and medications that could potentially ameliorate symptoms.
• Physicians described improvements to hematopoietic stem cell therapy, which is currently the best available treatment for Krabbe disease.

Advanced imaging tools are being tested that may reveal in more detail the changes that occur in the nervous system of children with this disease. This will help identify which children are in need of immediate treatment. In addition, researchers described new methods to identify and test drugs that might be useful in treating Krabbe disease. Promising new therapies are already being tested in large animals.

Sponsored by The Legacy of Angels