Jada Bascom Foundation
All conditions

Inherited metabolic disorders

X-linked cerebral adrenoleukodystrophy

Also called: cALD · X-CALD · cerebral ALD · adrenoleukodystrophy · ALD · Lorenzo’s oil disease · Cerebral X-linked adrenoleukodystrophy · Childhood cerebral adrenoleukodystrophy

What a donor has to do with this

For some people with this condition, a transplant using blood stem cells from an unrelated donor is part of the treatment guidelines. When a transplant is the right route and no one in the family matches, that donor comes from a registry. Not everyone with this condition has a transplant, and many never need one.

This is our reading of published transplant guidelines for this condition, not a measurement of how many people need a donor. Where a source actually counted donors, the figure and the people it counted are shown further down. Where none did, we say so rather than estimate.

What the evidence says

Who it affects
Typical onset/diagnosis: cerebral disease affects boys and often begins in the first decade, although onset can occur later. Evidence: U.S. NIH GARD synthesis (updated 2026); no markedly enriched geographic population was identified.
Treatments other than a transplant
Skysona (elivaldogene autotemcel) — approved under accelerated approval — United States, 2022 — For boys aged 4-17 years with early active CALD and no available HLA-matched allogeneic donor; the donor restriction was added in the 2025 label update, which also strengthened hematologic-malignancy warnings and lifelong surveillance.
If a transplant is used, the cells come from
bone marrow: not separately reported in the opened disease-specific sources; mobilized peripheral blood stem cells: not separately reported in the opened disease-specific sources; umbilical cord blood: not separately reported in the opened disease-specific sources; dominance: no dominant graft source was reported in the opened disease-specific sources
How often the donor was unrelated
Not reported. No source we could read states this for this condition, so we do not give a number. An estimate here would be a guess dressed as evidence.

Where this gets complicated

Benefit is stage-dependent; advanced MRI burden or neurologic dysfunction can continue to progress despite treatment.; Skysona's hematologic-malignancy risk materially changes counseling and can favor allogeneic HCT when a suitable donor is available.

Written for transplant clinicians, not for patients. We quote it so you can see what the guidance actually says:
“who do not have an available human leukocyte antigen (HLA)-matched donor for allogeneic hematopoietic stem cell transplantation (HSCT)”

It describes what teams consider in general. It cannot say what applies to any one person. Read the source.

People with this condition need donors

Joining a registry is a cheek swab and a short health form. You are contacted only if you turn out to be a possible match for someone, and you can ask questions and decline before anything else happens.

Find your registry

Related conditions

Others in inherited metabolic disorders. They are genuinely different diseases with different treatments — the group name is not a diagnosis.

Where this came from

X-linked cerebral adrenoleukodystrophy — what it is and how it is treated | Jada Bascom Foundation