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Researchers Show Why Statins Can Be Used to Treat Medulloblastoma Without Side Effects

June 9, 2021

Zeng-jie Yang, MD, PhD, an associate professor in the Cancer Signaling and Epigenetics ProgramZeng-jie Yang, MD, PhD, an associate professor in the Cancer Signaling and Epigenetics Program

PHILADELPHIA (June 9, 2021)—There’s growing evidence that statins can offer an effective treatment for medulloblastoma, a childhood brain cancer, without the severe side effects of other chemotherapy drugs. Now a new study by researchers at Fox Chase Cancer Center explains why.

Specifically, the researchers demonstrated in mouse and cell models that statins successfully penetrate brain and plasma tissue, but do not penetrate bone. This explains why statins do not seem to cause the same bone deformities caused by other treatments.

“We’ve demonstrated that this exhibited very promising efficacy in treatment of medulloblastoma, but the question I kept getting from colleagues in the field is, ‘Why are there no side effects in bone development?’” said lead author Zeng-jie Yang, MD, PhD, an associate professor in the Cancer Signaling and Epigenetics Program.

“Now we’ve answered that question—it’s because it cannot penetrate into the bone,” added Yang, who conducted the study with colleagues at Fox Chase and at other centers.

Medulloblastoma is a rare cancer overall, affecting only around 500 children nationally each year, but it’s the most common form of childhood brain cancer.

Around 30% of medulloblastomas are caused by mutations in a cell signaling mechanism called the hedgehog pathway, which tells cells how to divide and grow. This pathway plays an important role in bone development, but when it becomes overactivated it can cause tumors to grow in the brain. Drugs that block the hedgehog pathway are an effective treatment, but they can lead to irreversible defects in bone growth in young children.

“The major challenge in medulloblastoma management right now is the toxicity of the treatment. Hedgehog pathway inhibitors such as vismodegib inhibit tumor growth but also severely disrupt bone development in patients with medulloblastoma,” Yang said.

Statins work by controlling cholesterol, an essential key to activating the hedgehog pathway.  While previous studies showed statins could be an effective treatment, it was not clear why they did not cause the same side effects in bone development.

For the new study, the researchers used mice that were genetically engineered to block cholesterol biosynthesis in the bone and observed that the animals’ bone development was disrupted. This confirmed that both the hedgehog pathway and cholesterol biosynthesis were important for bone development.

They then studied two kinds of treatment in young mice with tumors. Some were treated with statins, while others were treated with hedgehog pathway inhibitors. The inhibitor group developed bone deformities, but the statin-treated mice did not.

Finally, they examined the mice a half hour after treatment and found that the statins could be detected in the brain and plasma, but not in the bone.

“I think all this evidence provides a strong rationale to include statins in the clinical management of medulloblastoma,” Yang said. “People are excited, because we don’t need to worry about the safety of the drug.” Yang is preparing a clinical trial to follow up on this research.

The paper, “Statins Repress Hedgehog Signaling in Medulloblastoma With No Bone Toxicities,” was published in Oncogene.

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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