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Research Highlight | August 31, 2020

Repurposing cholesterol drugs to treat multiple myeloma

Using a drug approved for one disease to treat another disease is often referred to as "drug repositioning." This is a common strategy in cancer treatment, and studies have revealed that drugs prescribed for ailments such as type 2 diabetes, high cholesterol and infections may be effective against certain cancers.

A new study TFRI-funded researchers at Princess Margaret Cancer Centre adds new insight into drug repurposing for cancer treatment. The team, led by Senior Scientist Dr. Linda Penn in collaboration with Clinician Scientist Dr. Suzanne Trudel, found that drugs prescribed to lower blood cholesterol, known as statins, may be effective for treating some types of multiple myeloma.

"Statins have been shown to induce cancer cell death in many tumour types, but it is difficult to predict which types of cancers will respond to statin treatment," says Dr. Penn.

Multiple myeloma is a cancer that originates in white blood cells called plasma cells. Some forms of the disease – those associated with worse prognoses –are characterized by a genetic change, referred to as t(4;14).

Using an experimental model of this cancer subtype, the researchers found a potential weakness: when exposed to statins, which block the production of cholesterol, t(4;14)-positive cancer cells died. Next, the research team found that statins, when combined with standard treatment, further improved the ability to kill cancer cells.

Preclinical and clinical studies are needed to test whether statins can help treat this aggressive subtype of cancer in patients.

"Because statins have already been shown to be safe for use in humans, future trials can be fast tracked," Dr. Penn says of these next steps. "By identifying statin sensitivity in this cancer subtype, our work could lead to effective and low-cost treatments."


The mevalonate pathway is an actionable vulnerability of t(4;14)-positive multiple myeloma


Joseph Longo, Petr Smirnov, Zhihua Li, Emily Branchard, Jenna E van Leeuwen, Jonathan Licht, Benjamin Haiba-Kains, David W Andrews, Jonathan J Keats, Trevor J Pugh, Suzanne Trudel, Linda Z Penn


This study was partially funded by a Terry Fox Program Project Grant to New era of precision medicine in triple-negative breast cancer