Salvador Soriano
Loma Linda University, CA·Neuroscience
Niemann-Pick disease type C (NPC) is a fatal brain-degenerating disease of children. It is caused by mutations in the NPC1 or NPC2 genes and has no approved disease-modifying treatment in the United States.
- Fields
Neuroscience
Pharmacology
Medicine & Clinical Research
- Topics
Niemann-Pick disease type C
ferroptosis
lipid peroxidation
- Grants
- 1 known grant, through Jan 2028
What they work on
- Characterize ferroptosis, iron-dependent cell death caused by oxidized lipids, along the front-to-back axis of cerebellar degeneration in a Niemann-Pick type C mouse model, focusing on Purkinje neurons.
- Test whether the anti-ferroptosis compounds J147 and CMS121 reduce NPC disease features in the brain of a mouse model of the disease.
- Determine whether J147 and CMS121 delay clinical symptoms and increase survival in NPC mice, assessing ferroptosis as a drug target for repurposing existing compounds.
More about the research
Affected cells show many problems: swollen lysosomes (the cell's recycling compartments), damaged mitochondria, imbalanced iron and calcium, oxidized fats and long-lasting inflammation. How these problems combine to kill neurons is unclear, and this gap slows drug development.
Why it matters
Children with Niemann-Pick disease type C have no approved disease-modifying therapy in the U.S. If ferroptosis proves to drive their neuron loss, it could become a treatment target. Because the anti-ferroptosis drugs J147 and CMS121 already have Investigational New Drug approval, they could move quickly toward clinical testing if they slow disease in mice.
Known grants
From NIH and NSF research grants. Labs often have other funding we can't see.
Closest labs
Jason D Huber
West Virginia University, WV·Pharmacology
Test whether NL-1, a compound that binds the mitochondrial protein mitoNEET, prevents ferroptosis (iron-dependent cell death) in a four-cell Transwell blood-brain barrier model after oxygen-glucose deprivation and reperfusion.
Jessalyn Ubellacker
Harvard University, MA·Biochemistry
Test whether melanoma cells depend on making N-acylglycines, a class of lipid molecules, to survive in lymph nodes, and whether blocking this pathway reduces their survival, using patient-derived xenograft and mouse melanoma models.
Peijian He
Georgia State University, GA·Medicine & Clinical Research
Test whether losing frataxin, a mitochondrial iron-handling protein, causes ferroptosis (iron-driven cell death) and liver injury in MASLD mouse models, and whether restoring frataxin prevents this damage.
The school
Loma Linda University: 2 labs on PI Pages, $24M a year on research, 138 research doctorates a year.
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