November 30, 2024, 3:01 pm | Read time: 3 minutes
Toxic epidermal necrolysis (TEN) is a hypersensitivity reaction of the skin – with life-threatening consequences. Around 15 to 48 percent of those affected die from this skin disease. However, researchers have now found a way to treat the disease successfully.
Toxic epidermal necrolysis is characterized by extreme redness on the skin, can blister, and cause the skin to peel off – similar to severe burns.1 Fever can also be a possible symptom. This skin disease is often caused by taking medication such as antiepileptic drugs and antibiotics. Once it has broken out, the disease takes a severe course. The medication that triggered it must be discontinued and intensive treatment provided. Around half of those affected do not survive toxic epidermal necrolysis – but researchers now claim to have found a cure.
Overview of the disease
Creation of a molecular map
Researchers from the Max Planck Institute of Biochemistry and the Walter and Eliza Hall Institute of Medical Research (WEHI) collaborated to study toxic epidermal necrolysis. They used a technical method called deep visual proteomics to create a detailed molecular map.2 They used skin biopsies of lesions (tissue damage) from patients with drug-induced skin reactions as a basis. This manifested itself in mild and severe skin irritation; samples from healthy people were used for the control group.
With the help of artificial intelligence, the scientists separated the cell types, extracted their proteins, and analyzed them. This allowed them to identify around 5000 proteins in each cell type for each participant. “By applying spatial proteomics to archived patient samples suffering from toxic epidermal necrolysis, we were able to precisely isolate and analyze individual cell types and understand what is actually happening in the skin of these patients,” summarizes Dr. Thierry Nordmann, the first author of the study, in a press release.3
Samples from mice
Upon further analysis of the cells, the researchers identified a so-called JAK/STAT signaling pathway in patients with toxic epidermal necrolysis (TEN). Based on this observation, they used drugs that block this signaling pathway. These are JAK inhibitors, which are already approved for the treatment of inflammatory diseases such as rheumatoid arthritis. They first used the drugs in the laboratory and then in mice. The result: toxic epidermal necrolysis was halted.
Treatment of seven patients
To find out whether the drugs also work in humans, they administered the JAK inhibitors to seven patients with toxic epidermal necrolysis. Each of the test subjects experienced a rapid improvement in symptoms and went through to full recovery with no significant side effects.
“Finding a cure for deadly diseases like this is the holy grail of medical research. I am extremely proud of this incredible research collaboration that has already helped save the lives of many patients,” said Dr. Holly Anderton, second author of the study.
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Classification of the study
This study is a significant milestone in the development of a cure for toxic epidermal necrolysis. However, further clinical trials still need to be carried out, as the JAK inhibitors were only tested on seven patients. There is a risk that the results may no longer apply to the wider population. Once the drug has been tested on more patients, regulatory approvals for the treatment of toxic epidermal necrolysis must be obtained before it can be used in acute therapy.
Furthermore, the long-term safety and efficacy of JAK inhibitors must be further researched.