1. Treatment of skin cancer in patients with recessive dystrophic epidermolysis bullosa.
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EPIDERMOLYSIS bullosa , *SKIN cancer , *CLINICAL drug trials , *CANCER cell growth , *CANCER treatment , *SQUAMOUS cell carcinoma - Abstract
Recessive dystrophic epidermolysis bullosa (RDEB) is a rare disorder that leads to painful blisters and large open wounds of the skin that do not heal. Individuals with RDEB often develop skin tumours called cutaneous squamous cell carcinoma (cSCC), which are difficult to treat and rapidly get worse. The overall risk of death in patients with RDEB and cSCC can be up to 90% by the age of 55 years due to disease complications. We and others have found that patients with RDEB and related cSCC have increased levels of a protein called transforming growth factor‐beta (TGF‐β). This protein controls processes (signals) within cells that can affect cell growth and development. TGF‐β signalling is known to have a dual role in tumour development by either increasing or blocking cancer cell growth. The aim of this study, carried out by researchers from the UK, the USA and Mexico, was to clarify whether increased levels of TGF‐β promote or inhibit growth of cSCC in patients with RDEB. This is important to assess for these patients because it could improve treatment options, which are significantly lacking for these patients To assess whether targeting TGF‐β signalling is a possible treatment option, we used cells extracted from patients with RDEB and cSCC and showed that drugs that block TGF‐β in cancer cells could help the majority of these patients, specifically those where TGF‐β is contributing to tumour cell growth. However, we also found that in a small number of patients, TGF‐β slows down tumour growth, and therefore using drugs to block TGF‐β might be harmful for this patient group. Based on our findings, studies such as clinical trials using drugs targeting TGF‐β should be done cautiously due to the possibility of these drugs actually causing harm in some patients with RDEB and cSCC. This research highlights the different roles of TGF‐β in RDEB skin cancer, and therefore the need to identify those patients who might benefit from using drugs targeting TGF‐β signalling. Linked Article: Dayal et al. Br J Dermatol 2021; 184:697–708. Linked Article: Dayal et al. Br J Dermatol 2021; 184:697–708. [ABSTRACT FROM AUTHOR]
- Published
- 2021
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