Funded Research

We fund research projects at all six ALD Connect Centers of Excellence, bringing together doctors and scientists who are working every day to better understand and treat this disease. Our focus is simple: move promising ideas forward faster and encourage collaboration so progress isn’t happening in isolation.

In Fall 2025, we committed over $1 million to a group of high-priority projects focused on preventing cerebral ALD, treating or preventing AMN, and improving our understanding of the disease mechanisms through biomarkers.

Pillar #1: New Treatments for Cerebral ALD

Cerebral ALD is the most aggressive form of the disease and can be fatal without intervention. We are funding research aimed at improving detection, expanding treatment options, and reducing treatment-related risks.

  • Testing FDA-approved cystic fibrosis drugs to restore ABCD1-related function and potentially prevent cerebral ALD progression.

  • Screening FDA-approved immune-modulating drugs in microglial models to identify potential preventatives for brain inflammation in CALD.

  • Systematic screening of FDA-approved compounds in human microglia to identify therapies that protect brain cells from VLCFA toxicity.

  • Evaluating nervonic acid in ALD mouse models to determine whether it can lower VLCFAs and reduce risk of cerebral progression

  • Investigating interferon signaling pathways in human and mouse brain tissue to uncover new therapeutic targets.

  • Studying cholesterol trafficking defects linked to ABCD1 dysfunction to identify new prevention strategies beyond VLCFA targeting.

  • Improving safety and delivery methods in lentiviral gene therapy to reduce long-term cancer risk and enhancing cell correction efficiency to make gene therapy more effective.

  • Developing RNA-based approaches to correct ABCD1 expression with potentially lower long-term risks than traditional gene therapy.

Pillar #2: New Treatments for AMN

AMN affects the majority of adults with ALD and currently has no disease-modifying treatment. Our portfolio includes projects focused on slowing progression and improving quality of life.

  • Testing whether blocking SARM1 can slow neurodegeneration in AMN mouse models, including studies in female models.

  • Using AI/ML to analyze MRI and sensorimotor data to identify measurable biomarkers and patient subtypes in AMN.

  • Testing small-molecule inhibitors of ELOVL1 to determine how much VLCFA reduction is needed for meaningful therapeutic effect.

Pillar #3: Biomarkers and Early Detection

Earlier detection and better biomarkers are essential to improving outcomes and accelerating therapeutic development. These projects focus on identifying measurable signals that can guide treatment targets and clinical trials.

  • Whole-genome sequencing of 100 ALD patients to identify genetic modifiers that influence disease severity and progression.

  • Developing a next-generation mouse model to better understand inflammatory triggers and uncover predictive biomarkers.