Van Haren Lab Research

Background and Mission

Dr Van Haren specializes in the diagnosis and treatment of children with neurodegenerative disorders. The VH Lab research team draws inspiration and insight from these remarkable families. We aim to alleviate human suffering through compassion, collaboration, and discovery.   

Approach

Neurogenetic and immunogenetic disorders serve as our starting point for understanding the functional relevance of individual human genes. We leverage clinical and laboratory insights from these disorders to unravel the molecular mechanisms of human neuroinflammation.

The VH Lab leads and collaborates on basic science, translational, and clinical endeavors worldwide. We utilize a variety of techniques to study the human brain and immune function in vitro (e.g. flow cytometry, histology, gene editing) and in vivo (e.g. MR spectroscopy, clinical assessments, clinical trials). 

Laboratory Research

X-linked Adrenoleukodystrophy & Multiple Sclerosis

X-linked adrenoleukodystrophy (X-ALD) is our lab’s prototypic disorder. X-ALD originates with a mutation in a gene involved in fatty acid beta-oxidation and manifests one of the most important genetic mimics of multiple sclerosis (MS). We leverage bidirectional insights from X-ALD and MS to power novel approaches in our understanding and treatment of both disorders. We are particularly interested in the metabolic dysfunction of myeloid cells in X-ALD and MS.

Single-gene immune disorders with neurologic phenotypes

Beyond X-ALD, an increasing number of genetic mutations have been linked to neurologic phenotypes (e.g. CTLA-4 mutation syndrome). Our lab collaborates with other investigators (e.g. Butte Lab) to identify the genetic origin of these disorders and characterize the resulting dysfunction within the brain and the immune system.

Clinical Research

Clinical Study Name Enrollment criteria Enrolling? ClinicalTrials.gov Identifier

Stanford IRB #

A pilot study of vitamin D in boys with X-ALD X-ALD boys age 3-12yo who have not yet developed cerebral ALD Yes NCT02595489 30805
Clinical outcomes after stem cell transplant in X-ALD X-ALD boys who are <2yrs post-transplant for cerebral ALD Yes NCT02204904 31193
Natural history study in X-ALD Men and women of any age with X-ALD Yes N/A

32670

Biobank for inflammatory brain disorders Individuals with suspected inflammatory brain disorders as well as healthy controls Yes N/A 23596