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A new study suggests rigosertib as a safe and effective treatment for skin cancer in RDEB patients

New scientific publication with EB House contribution

Squamous cell carcinoma (SCC) is a fatal complication that often arises in patients suffering from the severe genetic skin disease recessive dystrophic epidermolysis bullosa (RDEB). RDEB SCC is one of the most aggressive forms of this cutaneous tumor, and thus far, no approved or effective therapies are available for these patients.

In a recent study, published in Clinical Cancer Research, a team of researchers led by Prof. Andrew South in Philadelphia (USA) identified rigosertib as a potential drug to treat RDEB SCC.

Up until now, researchers have been unable to identify the genetic alterations that account for the highly aggressive nature of RDEB SCC when compared to similar tumor entities such as UV-induced skin SCC and head and neck SCC. However, in a previous study, the research team observed that cells derived from RDEB SCC produce more of an enzyme called polo-like kinase 1 (PLK1) than healthy skin cells. They reasoned that PLK1 could thus serve as a target to specifically kill these tumor cells.

In the current study, the scientists tested 6 different compounds, reported to inhibit PLK1 activity, on different patient-derived RDEB SCC cells, all of which showed increased PLK1 levels, and normal skin cells isolated from 10 different RDEB patients. The most efficient compound was rigosertib, which induced cell death in all RDEB SCC samples, while only slowing the growth of normal RDEB skin cells at much higher doses than needed to kill the cancer cells.

These data demonstrate a high sensitivity of RDEB SCC to rigosertib and provide the basis for the approval of a phase 1/2 clinical trial to evaluate the safety and efficiency of rigosertib in RDEB patients.

To access the article please click here

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