Possible cure to a dangerous disease linked to cancer in children found

Possible cure to a dangerous disease linked to cancer in children found

12:28 09 May in Uncategorized

The list of cancers can be quite alarming, ranging from severe cases to more easily curable cases. Cancer is versatile and can ultimately target any demographics regardless of age, sex or ethnicity. The cancer squamous cell carcinoma, a skin cancer typically caused by sun exposure generally is one of the cancers that can be easily treated and cured. However, like many medical conditions, the effects of this disease have many factors, and unfortunately, in this case, age is a significant factor. Children with the severe skin disease, recessive dystrophic epidermolysis bullosa (RDEB) or otherwise known as the butterfly disease are at a much higher chance of developing squamous cell carcinoma. Luckily for these children, researchers from WorldCare Consortium® medical second opinion provider Jefferson Hospital at the Sidney Kimmel Cancer Center- Jefferson Health have possibly found a safe treatment for these children.


Children with RDEB often develop squamous cell carcinoma, skin cancer, by early adulthood. This is usually a curable type of skin cancer. However, for these patients, the cancer at stake is deadly and has a survival rate of near zero and with a life expectancy of five years.  Dr. Andrew South, PhD, an associate professor in the Department of Dermatology and Cutaneous Biology and researcher with the Sidney Kimmel Cancer Center- Jefferson Health and fellow colleagues found in previous research that the lack of protein development that helps to hold layers of skin together making even the smallest touch painful results in painful blistering. From these blisters, patients develop chronic skin spurs and inflammation leading to the development of life threatening cancer squamous cell carcinoma. Dr. South states “If we can reduce the cancer, or even reduce the spread of the cancer, that is going to improve patients’ quality of life and expand their lifespan.” Dr. South and his colleagues had previously found the enzyme in cell division called Polo-like Kinase (PLK1) could potentially be used as a drug to target squamous cell carcinoma. When researchers tested six versions of the anti-PLK1 compound, one dramatically stood out against the rest when attacking the RDEB patients’ cancer. This drug is referred to as Rigosertib.

Rigosertib is precisely what researchers look for in cancer treatment. When researchers isolated both cancerous cells and healthy cells from 10 patients treating them with Rigosertib this is what they found:

  • Rigosertib attacked the cancer cells while leaving the patients’ healthy cells intact
  • Healthy cells would only slow down if the dosage levels of the drug exceeded the dosage needed to kill the cancer cells
  • All 10 cases resulted in the cancer cells dying


This is astonishing, Dr. South states “sixty percent efficacy rate is something one might think about taking to the clinic, but 100 percent of the cells being responsive to the drug is something I have never seen before”.

Researchers were so pleased with these results they continued their research by testing the drug on mice. The results showed that the drug was effective in treating cancer in the pre-clinical mice models. It stopped the cancer’s growth and the tumor cells died. Clinical trials are on track to begin this spring or summer under the supervision of Bahar Dasgeb, MD, the principal investigator of the trial and an assistant professor of Dermatology, at Jefferson’s Adult EB clinic.  If clinical trials provide the same results as pre-clinical mouse trials, the drug will provide patients with a drug that will target their cancer.

If you have a child that has been diagnosed with RDEB and would like a medical second opinion contact us today.

Source: https://hospitals.jefferson.edu/news/2019/03/potential-treatment-for-cancer-in-butterfly-disease.html

Evan DeSimone