Statins Offer Hope for Children with the Most Common Brain Tumor Type

February 6, 2018

PHILADELPHIA (February 6, 2018) – Researchers at Fox Chase Cancer Center have found that cholesterol is required for medulloblastoma tumor growth, implying that statins may be an effective treatment option for children with the most common type of brain tumor.

“Approximately 30% of medulloblastomas arise from aberrant activation of the hedgehog pathway. We’ve found that cholesterol is necessary for this signaling to take place, and that inhibiting cholesterol biosynthesis with statins blocks hedgehog pathway activity in medulloblastoma cells,” said Renata Gordon, a PhD student at Fox Chase Cancer Center. Gordon co-authored the paper with Zeng-jie Yang, MD, PhD, an associate professor in the Cancer Biology Program at Fox Chase. It appears in the journal Clinical Cancer Research. The research was supported by a grant from the American Cancer Society.

There are two available drugs that have demonstrated efficacy against hedgehog pathway-associated medulloblastoma. Despite a dramatic response to these inhibitors in initial clinical trials, drug resistance and developmental toxicities arise. The paper notes that statins also synergize with vismodegib, one of the approved hedgehog pathway inhibitors.

“Given the excellent safety profile, low cost, and ready availability of cholesterol inhibitors, targeting cholesterol biosynthesis represents a promising strategy for treatment of medulloblastoma and potentially other malignancies caused by hedgehog pathway activation,” Yang said.

Medulloblasotma is the most common malignant brain tumor in children, accounting for about 20 percent of all brain tumors in children. The great majority of diagnoses occur before age 10. Despite significant progress in conventional strategies for tumor treatment, including surgery, chemotherapy, and radiation, more effective but less toxic chemotherapeutic drugs for medulloblastoma are still urgently needed.

The Hospital of Fox Chase Cancer Center and its affiliates (collectively “Fox Chase Cancer Center”), a member of the Temple University Health System, is one of the leading cancer research and treatment centers in the United States. Founded in 1904 in Philadelphia as one of the nation’s first cancer hospitals, Fox Chase was also among the first institutions to be designated a National Cancer Institute Comprehensive Cancer Center in 1974. Fox Chase researchers have won the highest awards in their fields, including two Nobel Prizes. Fox Chase physicians are also routinely recognized in national rankings, and the Center’s nursing program has received the Magnet recognition for excellence five consecutive times. Today, Fox Chase conducts a broad array of nationally competitive basic, translational, and clinical research, with special programs in cancer prevention, detection, survivorship and community outreach. It is the policy of Fox Chase Cancer Center that there shall be no exclusion from, or participation in, and no one denied the benefits of, the delivery of quality medical care on the basis of race, ethnicity, religion, sexual orientation, gender, gender identity/expression, disability, age, ancestry, color, national origin, physical ability, level of education, or source of payment.


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