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JAK Inhibitors Achieve First-Ever Cure for Toxic Epidermal Necrolysis in Landmark Study

• Researchers have achieved a world-first cure for toxic epidermal necrolysis (TEN) by targeting the hyperactivation of the JAK/STAT pathway. • Spatial proteomics identified the JAK/STAT pathway's role in TEN, leading to the use of JAK inhibitors in preclinical studies and human trials. • Seven patients with TEN were successfully treated with JAK inhibitors, marking a significant breakthrough in the treatment of this life-threatening disease. • The findings pave the way for clinical trials aimed at regulatory approval of JAK inhibitors as a curative treatment for TEN.

A groundbreaking study has achieved the first-ever cure for toxic epidermal necrolysis (TEN), a life-threatening skin disease, by targeting the hyperactivation of the JAK/STAT pathway. The research, a collaboration with the Max Planck Institute of Biochemistry (Germany), utilized spatial proteomics to analyze skin samples from TEN patients, leading to the identification of the JAK/STAT pathway as a key driver of the disease. This breakthrough has resulted in the successful treatment of seven patients in Germany.

Deep Visual Proteomics Uncovers Key Mechanism

Researchers employed Deep Visual Proteomics, a cutting-edge approach, to examine individual cells in unprecedented detail. This allowed them to map the thousands of proteins driving TEN. Dr. Thierry Nordmann, clinician scientist at the Max Planck Institute of Biochemistry, explained, "By applying spatial proteomics to archived patient samples suffering from toxic epidermal necrolysis, we were able to precisely isolate and analyse individual cell types and understand what is actually occurring in the skin of these patients."

Identifying and Targeting the JAK/STAT Pathway

The analysis revealed a striking hyperactivation of the inflammatory JAK/STAT pathway. "We identified a striking hyperactivation of the inflammatory JAK/STAT pathway, revealing an opportunity to intervene in this deadly condition with JAK inhibitors," said Dr. Nordmann. This finding suggested that JAK inhibitors could be a potential therapeutic intervention for TEN.

Preclinical Validation and Clinical Success

The theory was tested in multiple preclinical studies, including a disease-approximating mouse model. The positive results from these studies allowed researchers to proceed directly to trialing the treatment in TEN patients. This research led to the groundbreaking result of seven patients in Germany being cured of the life-threatening disease.

Future Directions

Researchers are optimistic that these findings will pave the way for a clinical trial aimed at the regulatory approval of JAK inhibitors as a cure for TEN. The successful treatment of these patients represents a significant advancement in the management of this devastating condition.
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Reference News

[1]
World-first cure for lethal skin disease achieved in landmark study | WEHI
wehi.edu.au · Nov 10, 2024

Researchers used Deep Visual Proteomics to analyze skin samples from TEN patients, identifying hyperactivation of the JA...

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