Treatment of ALD:
Currently, there are only two treatment options to cure ALD. These are Lorenzo's oil and stem cell transplantation.
Lorenzo's Oil:
Currently, there are only two treatment options to cure ALD. These are Lorenzo's oil and stem cell transplantation.
Lorenzo's Oil:
- This is a combination of two fats extracted from olive oil and rapeseed oil. It was developed by Augusto and Michaela Odone to treat their son, Lorenzo, after he was diagnosed with ALD in 1982. The mixture of fatty acids in Lorenzo's oil works to reduce the levels of very long chain fatty acids, which are known to cause ALD.
- Parents with a child who may be at risk for ALD can consider enrolling in a clinical trial at the Kennedy Krieger Institute. If interested, please call 800-873-3377. That is presently the only way to obtain Lorenzo's oil in the U.S. because it is not approved by the Food and Drug Administration and is still considered an experimental drug. Not all insurance companies will provide coverage for the oil, which costs over $400 for a month's treatment.
Stem Cell Transplantation:
- There are currently two sources for transplanted stem cells: umbilical cord blood (UCBT) or bone marrow (BMT). In both cases, the goal is to provide the patient with healthy stem cells that produce a functioning ALD protein; the protein that is lacking in people with ALD.
- This treatment also has serious risks. Both types of transplantation require that the patient's immune system essentially be wiped out by high-dose chemotherapy and/or radiation, so that there bodies do not reject the foreign donor cells. This leaves patients vulnerable to almost any kind of infection, and these procedures have a mortality rate that is as high as 40%. It can also be difficult to find a well-matched donor, and sometimes the donor cell graft does not "take".