Cure ALD empowers families and engages researchers to make meaningful progress toward preventative treatments, and ultimately, a cure for ALD. Find out more about our story.

Project Details

Purpose

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.

Aligned with Cure ALD’s Mission

This project directly supports Cure ALD's mission to find treatments that can be given to all individuals with an ABCD1 mutation before they become sick.

Currently, newborn screening identifies babies with ALD, but there is nothing that can be offered to these families except to "watch and wait." We are hopeful nervonic acid may be able to change that. It is a natural compound that can be taken by mouth, and Dr. Kartha’s early research suggests it effectively lowers the harmful fats that cause damage in ALD.

By focusing on adrenal protection, this study addresses one of the earliest and most common problems in ALD. If nervonic acid can protect the adrenal glands, it provides hope that it might also protect the brain and spinal cord, and it gives us concrete evidence to move toward human trials.

Approach

Dr. Kartha’s lab at the University of Minnesota has shown that nervonic acid can lower the harmful fats that build up in ALD and cause damage. Now they must work together to take the next step: Can nervonic acid actually protect organs from damage?

They’re going to test this by looking at the adrenal glands in ALD mice. Research from colleagues studying ALD show alterations in hormone levels that suggest the adrenal glands in ALD mice are struggling to function, just like in boys with ALD. They’ll feed some of these mice nervonic acid for three months and see if it protects the adrenal glands.

Why this Matters for ALD Families

When a baby is diagnosed with ALD through newborn screening, there's currently no medicine to prevent problems from developing, families can only watch and wait. Parents must live with constant anxiety, waiting to see if their child's adrenal glands will fail or if brain disease will appear on the next MRI. This research could fundamentally change that experience.

If nervonic acid protects adrenal glands in mice, it would be the first treatment that works before damage occurs. What makes this especially promising is that nervonic acid is a natural supplement that can be taken by mouth, no hospital visits, transplants, or injections required. Children diagnosed with ALD could start taking it right after diagnosis to help protect their bodies while researchers continue working toward a cure. Success in this pilot study would help us plan for human trials, offering hope that families might one day have a simple, safe intervention to give their children from the very beginning—transforming ALD from a disease of anxious waiting into one where early action is possible.

"There is so much uncertainty with ALD, so preemptively protecting adrenal function could prove a massive relief to so many in our community. One less thing to watch and wait for. One more thing to be hopeful about." - Savannah Merrill, ALD Parent

About Keith Van Haren

Dr. Keith Van Haren is a pediatric neurologist at Stanford School of Medicine dedicated to finding better treatments for X-linked adrenoleukodystrophy (ALD). As a parent of a child affected by a rare disease, Dr. Van Haren has earned the trust of ALD families not only because of his scientific expertise, but because he understands what it means to partner with families in the fight against devastating childhood disorders.

At Stanford School of Medicine, Keith leads a team focused on cerebral ALD, the most severe form of the disease. His team conducts cutting edge research in the lab and in the clinic. His team recently developed the first mouse model to mimic human cerebral ALD and has previously led clinical trials studying vitamin D supplements in ALD. His current NIH-funded studies are studying the effects of a cutting-edge immune therapy in the mouse model of cerebral ALD.

Keith co-founded ALD Connect and helped establish the first care guidelines for leukodystrophies, ensuring families have access to the best possible supportive care while researchers work toward cures. He co-founded the annual ALD Family Weekend, creating a community where families can connect with each other and with researchers who are working to help their children.

Recognized with the Moser Award for Science & Humanism from ALD Connect and multiple awards from Stanford School of Medicine, Keith combines scientific rigor with compassion and a sense of urgency. His research aims to accelerate the discovery of treatments that could save children's lives and preserve their futures.

Visit our website to learn more about Cure ALD

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

By Cure ALD

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

driven by Dr. Keith Van Haren Lab at Stanford School of Medicine

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