Metabolic Storage Diseases

As expertise with blood and marrow transplants has increased, this treatment has been applied to a wider variety of disorders, including some which are not cancer-related or diseases that affect the bone marrow.

At the University of Minnesota , we have pioneered the use of blood and marrow transplants as treatment for patient with Adrenoleukodystrophy (ALD), Metachromatic leukodystrophy (MLD), or Globoid Cell leukodystrophy (GLD), also called Krabbe Disease.  In addition, transplants are used to treat metabolic “storage” diseases, such as Hurler syndrome and Maroteaux-Lamy syndrome.

While these disorders have their own characteristic challenges and complications, they are alike in their ability to cause severe, progressive neurologic deterioration and because they are inherited conditions genetically resulting in the deficiency of a single enzyme.

Blood or marrow transplant from a normal donor (an unaffected HLA-matched family, unrelated donor or unrelated cord blood) can provide replacement of the missing enzyme through the production of healthy white blood cells providing the missing enzyme.  In the presence of a source of enzyme the accumulated abnormal material can be cleansed, and the clinical features of the disease arrested or reversed.  The indications for transplant in each disease vary.