In the Lund Lab, we’re working every day to change the practice of medicine for boys with ALD. We have several active research projects aimed at finding safer, more effective therapies, validating biomarkers, understanding mechanisms, and personalizing care to meet the unique needs of each patient.
Our “Predicting the Future” project continues to advance. By leveraging the National ALD Registry and biorepository, we are studying ABCD1 mutations along with other clinical and biological factors to better understand why the disease presents so differently across patients. Our goal is to improve risk-stratification and enable more precise, personalized care, especially for boys in the monitoring stage. We are also exploring whether biomarkers, such as NfL and C26:0-lysoPC, could help predict progression and potentially reduce the need for frequent MRIs over time.
At the University of Minnesota Leukodystrophy Center, we are in a unique position to answer some of the most important questions in cerebral ALD treatment, given our extensive experience with transplant. Allogeneic hematopoietic cell transplant remains an effective option when disease is identified early, though it carries risks, including graft-versus-host disease. Gene therapy offers an alternative for patients without an HLA-matched donor, but important safety concerns remain, including reported cases of myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). Our team is actively comparing allogeneic HCT with gene therapy and other emerging approaches.
Every boy we care for fuels our determination to keep pushing forward. By joining our team, you’re standing with families affected by ALD and helping drive meaningful progress. Thank you for being part of this effort!
