X-linked Adrenoleukodystrophy (ALD) affects 1 in 17,000 people, impacting the brain, spinal cord, or adrenal glands with varying severity. Young boys face the most devastating form—childhood cerebral ALD—which can be fatal without early intervention. While treatment options exist for some manifestations, there is no cure or preventative treatments. Since there is limited funding for rare disease research, funding for ALD research is heavily reliant on donations.

Cure ALD exclusively funds research aimed at curing ALD or preventing any symptom onset, and by empowering families as active contributors to the cause through both fundraising and direct action that advances treatment possibilities. Take a look at the incredible projects below.

The inspiration for Cure ALD comes from the Wiener family, who in 2024, discovered that their three-year-old son had ALD after the mother took a genetic carrier screening test during a subsequent pregnancy. Confronting this devastating diagnosis, they quickly realized two critical gaps: (1) insufficient funding directed toward research focused on curing ALD or preventing symptom onset; and (2) a lack of resources to help newly diagnosed families take meaningful action in the fight against this disease. Find out more about our story.

Cure ALD Research Network

Your gift, your choice! When you donate, you can direct your dollars to the general ALD research fund (split between both projects) or give to a specific project you're passionate about. We believe you should have a say in the impact you make.

Project 1: Assessing Nervonic Acid's Potential to Protect Adrenal Function in ALD

Project Lead: Keith Van Haren, MD — Stanford School of Medicine

This study will explore whether nervonic acid, a natural fatty acid, can prevent adrenal failure in a genetic mouse model of ALD. Dr. Kartha’s laboratory at the University of Minnesota has already shown that nervonic acid lowers the harmful fats (VLCFAs) that damage adrenal cells. Now the Van Haren Lab and Kartha Lab will work together to test whether giving nervonic acid to ALD mice can protect their adrenal glands before damage occurs.

The challenge with adrenal failure in ALD has always been that doctors can only treat it after it happens, with lifelong steroid medications. The Van Haren lab will measure whether nervonic acid can lower harmful fat buildup in adrenal tissue, prevent cellular damage, and reduce stress hormones. The intent for this is to help design clinical trials of nervonic acid as the first preventive treatment for adrenal insufficiency in ALD boys. Learn more about this project →

Project 2: Safer Gene Therapy for ALD

Project Lead: Alex LeNail, PhD — George Church Lab, Wyss Institute

Alex is exploring a new way to treat Adrenoleukodystrophy (ALD) using gene therapy. The goal is to safely deliver a working copy of the ABCD1 gene to the brain and nervous system—the areas most affected by ALD.

The challenge with treating ALD has always been getting therapeutic treatments into the brain, which is protected by something called the blood-brain barrier. He’s testing specially designed delivery vehicles that may be able to cross this barrier more safely and effectively. Learn more about this project →

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