ALD Treatment Options
Navigating adrenoleukodystrophy (ALD) treatment options doesn’t have to be overwhelming or confusing. With the right care plan, your child can have the best chance at treatment outcomes. Early detection of cerebral ALD can help introduce treatment to prevent progression.
While there is currently no cure for ALD, your care team will work with you to relieve your child’s symptoms and slow disease progression.
ALD treatment options may include:
- Adrenal insufficiency treatment: Your doctor can treat adrenal insufficiency with medicines that replace the hormones that the body isn’t making (hormone replacement therapy).
- Exercise and physical therapy: If there are mobility issues, a physical therapist may provide exercises that can help strengthen the muscles and relieve spasms or stiffness. They may also recommend wheelchairs and other mobility devices if needed.
- Lorenzo’s oil: Lorenzo's oil is an experimental drug and is not approved by the US Food and Drug Administration (FDA). It is a combination of two fats and works to reduce the levels of very long-chain fatty acids. Van geel et al. (1999) has reported that Lorenzo’s oil is not an effective treatment option to slow progression to cerebral ALD, or to improve endocrine function. There are no ongoing studies on Lorenzo’s oil at this time.
- Medications: A doctor may prescribe your child medications to help relieve a number of different symptoms, including muscle stiffness and seizures.
- Bone Marrow Transplant (BMT) or Hematopoietic Stem Cell Transplantation (HSCT): A BMT or HSCT is a treatment option that can stop ALD from causing more damage by replacing the cells with dysfunctional ALDP protein with healthy ones. Transplants can come with serious risks and considerations.
If you want to learn more about this option, it’s important to talk to your doctor about the benefits and potentially severe risks involved. This may be an option to slow or halt the progression of cerebral ALD in children if diagnosed and managed early. You may want to discuss your options with a transplant specialist so that you are prepared if your child develops cerebral ALD in the future. This will help you take action quickly.
If your child does progress to cerebral ALD, you will want to meet with a transplant specialist as soon as possible. Treatment for cerebral ALD at early stages is associated with better results. Your transplant specialist will help you to determine which treatment is appropriate for your child.