Stem cell therapy might seem promising for children with adrenoleukodystrophy (ALD). However, not all of these children would benefit from the therapy, which comes with certain risks.
Information about stem cell therapy and ongoing clinical trials is available on our “Stem Cell Therapy” page. Here, we answer a few questions if you’re considering stem cell therapy for your child.
How beneficial is stem cell therapy?
Stem cell therapy is risky, and the benefits largely depend on three main factors:
- Whether ALD has been diagnosed early.
- The availability of a suitable donor.
- The reaction of the patient’s immune system to the transplant.
Of note, stem cell therapy cannot reverse neuronal damage that has already occurred.
Is my child a candidate?
An MRI can reveal the extent of ALD’s damage to the brain. The damage is graded using a Loes score on a severity scale of zero to 34. Children with a Loes score lower than 8 or 9 are considered eligible for stem cell transplantation.
What are the risks?
Stem cell therapy requires the child’s immune system to be shut down using chemotherapy or radiotherapy so that the donor stem cells are not rejected. This shutdown puts the child at high risk for infections. There is also a possibility that the donor cells are not “taken” by the recipient, despite good compatibility.
Even if the transplant is successful, the disease might progress for another six to 18 months before positive outcomes can be seen. Hence, stem cell therapy is best performed as early as possible once ALD diagnosis is confirmed and the eligibility criteria are met.
Last updated: September 05, 2019
Adrenoleukodystrophy News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.