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Can spatial proteomics find a TEN therapy?

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Deep visual proteomics of drug-reaction skin (>5,000 proteins/cell type) revealed interferon–STAT1 programs in TEN; JAK inhibitors improved disease in mouse models.

Source

Spatial proteomics identifies JAKi as treatment for a lethal skin disease

Nordmann TM, Anderton H, Hasegawa A, et al. · Nature · 2024

doi.org/10.1038/s41586-024-08061-0Read the full paper ↗101 citationscc by

What they did

Used deep visual proteomics on archived FFPE biopsies across CADR severities to quantify keratinocyte and immune-cell proteomes, then tested oral JAKis in two TEN mouse models.

What they found

TEN showed enriched type I/II interferon signatures and phosphorylated STAT1. Tofacitinib, baricitinib, abrocitinib, or upadacitinib ameliorated clinical/histological severity in mice. TEN involves >30% epidermal detachment and lacked effective therapy.

The limits

What it doesn't show

Mouse efficacy and proteomic nomination are not a completed large randomized TEN trial; dosing/safety in critically ill TEN patients need careful clinical evaluation.

Key terms

TEN
Toxic epidermal necrolysis: severe drug reaction with >30% epidermal detachment.
Deep visual proteomics (DVP)
Imaging + AI cell picking + ultrasensitive MS proteomics at cell-type resolution.
JAKi
JAK inhibitors (e.g., tofacitinib, baricitinib, abrocitinib, upadacitinib).
STAT1
Transcription factor phosphorylated downstream of interferon signalling in TEN.
CADR
Cutaneous adverse drug reaction spectrum from MPR to DRESS/SJS/TEN.
DRESS
Drug reaction with eosinophilia and systemic symptoms; comparator severe CADR.

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Proteins quantified were more than:

Common questions

How many proteins quantified?

More than 5,000 in keratinocytes and skin-infiltrating immune cells.

DVP cohort makeup?

n=21: 5 DRESS, 5 MPR, 5 healthy, 6 TEN.

Key pathway hit?

Type I/II interferon signatures with pSTAT1.

Drug class tested?

JAK inhibitors including tofacitinib/baricitinib/abrocitinib/upadacitinib.