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Rigosertib Provides Hope in Patients With Butterfly Disease

For many patients with the genetic disease recessive dystrophic epidermolysis bullosa (RDEB), also known as butterfly disease, developing squamous cell carcinoma (SCC) by early adulthood is inevitable. Researchers teamed together and found a potential targeted treatment for patients with RDEB and SCC.

"We hope that the drug will be a cure for the cancer," remarked one of the authors of the study, Andrew South, PhD, Associate Professor in the Department of Dermatology and Cutaneous Biology at Thomas Jefferson University. "If we can reduce the cancer or even reduce the spread of the cancer, that is going to improve patients' quality of life and extend their lifespan."

People with RDEB lack a crucial protein responsible for holding layers of skin together, making their skin fragile and prone to damage and blisters. Dr. South and colleagues conducted previous research in which they discovered that because patients with RDEB have chronic inflammation and scarring, these patients are more susceptible to developing SCC.

In addition, the investigators tested six drugs and determined that a drug called rigosertib killed cancer cells most effectively in mouse models of REDB-associated SCC by targeting Polo-like Kinase 1 (PLK1), an enzyme involved in cell division.

When the scientists sampled cancerous and healthy cells from 10 patients with REDB-associated SCC and dosed the cells with rigosertib, all of the cancer cells were destroyed in all 10 cases. Rigosertib was able to demolish cancer cells without harming normal cells; it was only able to slow the growth of normal cells at doses much higher than those needed to kill the cancer cells. In mouse models, rigosertib stopped proliferation of cancer cells, and the malignancies died.

A clinical trial testing the efficacy of rigosertib in patients with RDEB-associated SCC is set to occur soon.

For More Information

Atanasova VS, Pourreyron C, Farshcian M, et al (2019). Identification of rigosertib for the treatment of recessive dystrophic epidermolysis bullosa-associated squamous cell carcinoma. Clin Cancer Res. [Epub ahead of print] DOI:10.1158/1078-0432.CCR-18-2661

Image courtesy of Derek Ramsey

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