Research teams around the world continue their efforts to help those affected by Krabbe and Leukodystrophies. Therefore, we continue to fund important and promising extramural research projects.
Our greatest desire is for a cure for these horrible disorders, and our hope is that researchers throughout the world continue to work together to accomplish this.
Research Projects Currently Funded
Steven Gray, PhD
University of North Carolina Gene Therapy Center
Several encouraging laboratory studies have been published by a number of groups that demonstrate the potential of gene therapy to treat Krabbe Disease. However, these studies were done in ways that would be difficult to translate directly to humans.
The laboratory at the UNC Gene Therapy Center has been investigating ways to globally deliver a gene to the nervous system in a way that can scale to humans. This approach will be implemented in a planned human clinical trial for Giant Axonal Neuropathy and will use a modified virus called AAV, which has been engineered to carry a therapeutic gene instead of virus genes, essentially serving as a molecular delivery truck termed a “vector.” This vector is administered by an intrathecal injection, similar to a spinal tap, which is a routine outpatient procedure with minimal risk.
The objective of this study is to bridge the gap between earlier encouraging laboratory studies and a possible human treatment, modeling this AAV intrathecal approach in younger and older mice with Krabbe Disease.
In-Utero Transplants in Krabbe Patients
Joanne Kurtzberg, MD
Duke Medical Hospital
Dr. Joanne Kurtzberg is conducting cord blood transplants in Krabbe patients before birth. The benefits of this research include a faster time to transplant as compared to those administered after newborn screening, and avoidance of chemotherapy.
The purpose of this Phase I Clinical Trial is to determine if it is safe to administer unrelated umbilical cord blood to pregnant mothers during their first trimester. This study includes Krabbe Disease as well as other Lysosomal Storage Disorders including MLD, Tay Sachs, Sandhoff, and PMD.
It is anticipated that these results will shed light on the importance of timely diagnosis and providing treatment before the onset of symptoms.
To learn more please visit: clinicaltrials.gov
For a list of past research projects funded by Hunter’s Hope Foundation, please visit our Historical Research page. For more information on extramural research projects and funding, please email firstname.lastname@example.org.