The University of Minnesota Pediatric Blood and Marrow Transplant Program has more experience treating inherited metabolic storage diseases, including adrenoleukodystrophy (ALD), than any other center in the world. To date, we have treated hundreds of ALD patients with a blood or marrow transplant (BMT). The first ALD patient came through our program in 1991, and in the more than 20 years since, we have continued to improve our transplant protocols, providing better outcomes with less side effects for the patients we treat.
ALD is a rare genetic disorder that affects males. It’s characterized by the inability of the body to break down "very long chain fatty acids" (VLCFA). The resulting build-up of VLCFA can cause dysfunction of the adrenal glands, testes and the central nervous system by breaking down the myelin sheaths that protect nerves. Normally, nerve endings are covered with a myelin sheath which helps speed the communication of nerve impulses. If myelin is lost, the underlying nerve cell will become dysfunctional. When this breakdown occurs in the brain, it’s known as cerebral ALD (cALD), which often causes rapidly progressive neurologic deterioration and eventual death.
Parents of children with cALD typically begin to notice behavioral changes, such as hyperactivity, poor performance in school, and short attention span.
Nearly 40 percent of ALD cases are the cerebral form of ALD which begins during childhood, usually between the ages of 4 and 8. The only effective treatment for cALD is a blood and marrow transplant (BMT), which can halt progression of myelin loss.
ALD affects about one in 21,000 males.
For patients with a diagnosis of ALD, we can monitor patients with regularly scheduled MRI scans to watch for the first sign of cerebral involvement. Many boys with ALD will never develop the cerebral form.
For patients who come to us with a diagnosis of cALD, we will perform a comprehensive assessment to determine how far the disorder has progressed. Based on this assessment, BMT may be an option to stop further deterioration. Unfortunately, transplant is not an effective treatment for patients with very advanced cALD.
Blood and Marrow Transplantation
Patients who are candidates for BMT will begin preparations for transplant. Patients and their families meet with many members of our transplant team, along with other specialists within the areas of endocrinology, genetics, neurology, neuropsychology and physical and occupational therapy. Patients with cALD may have a number of medical problems which could require specialized treatments. Our specialists are prepared to address all of these needs.
We have the most experience treating patients with cALD, and continue to modify and improve the transplant process, providing better outcomes and better prognosis for the young boys in our care.
Learn more about what to expect during the transplant process.
While our entire BMT team is involved in the care of our patients, the following providers lead the care and treatment plans for patients with cALD:
We continue to explore novel ways to effectively treat and improve quality of life for patients with ALD. We also collaborate with other researchers throughout the world, keeping our program at the forefront of ALD care worldwide. Learn more about current research projects here.
ALD transplant patients may be eligible for clinical trials, taking advantage of the latest breakthroughs in therapies and treatments. View a list of open trials.