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Researchers flip drug discovery script, unleashing thousands of medicines on cancer cells

February 13, 2020

Instead of starting with a disease and looking for a cure, Boston area researchers recently started with thousands of drugs and watched what they did to cancer.

The scientists found that a surprising number of the drugs – including some used for conditions like diabetes, inflammation, alcoholism, and even arthritis in dogs – actually did kill cancer cells in the lab.


The researchers from the Broad Institute of MIT and Harvard and Dana-Farber Cancer Institute tested 4,518 existing drugs against 578 cancer cell lines and found that “that non-oncology drugs have an unexpectedly high rate of anti-cancer activity,” the team reported in the journal Nature Cancer. Nearly 50 of the drug compounds show previously unrecognized anti-cancer activity, according to a Broad Institute report.

“We thought we’d be lucky if we found even a single compound with anti-cancer properties, but we were surprised to find so many,” said Todd Golub, chief scientific officer and director of the Cancer Program at the Broad, Charles A. Dana Investigator in Human Cancer Genetics at Dana-Farber, and professor of pediatrics at Harvard Medical School.

A new way to apply existing drugs

New uses for existing medicines are often stumbled upon, like the discovery of aspirin’s cardiovascular benefits. This study, however, employed Broad’s Drug Repurposing Hub, a collection that currently comprises more than 6,000 existing drugs and compounds that are either FDA-approved or have been proven safe in clinical trials (at the time of the study, the Hub contained 4,518 drugs).

“We created the repurposing hub to enable researchers to make these kinds of serendipitous discoveries in a more deliberate way,” said Dr. Steven Corsello, a Dana-Farber oncologist and lead author of the Nature Cancer paper.

Barcoding cell lines from human biospecimens

Using a molecular barcoding method known as PRISM, the researchers tagged each cell line with a DNA barcode, allowing them to pool several cell lines together in each dish and more quickly conduct a larger experiment, according to Broad. The team then exposed each pool of barcoded cells to a single compound from the repurposing library, and measured the survival rate of the cancer cells.

It’s too early to apply all the drugs in vivo. Although it’s “conceivable that some non-oncology drugs could be brought directly to clinical trials for testing in cancer patients,” the Nature Cancer paper reads, “results reported in this study also represent starting points for new drug development.”

Cell lines like those in this study are developed from human biospecimens, including tumor tissue samples such as those you can find on the iSpecimen Marketplace.

Learn about the iSpecimen Marketplace where you can browse millions of richly annotated, de-identified human tissue and biofluid biospecimens, in addition to hematopoietic and immune cell products. You can join for free and creating a login is easy. Request a quote or custom collection today.

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