New Breast Cancer Drug Kills Mouse Tumors In Single Dose

The typical treatment for estrogen receptor positive (ER+) breast cancer involves surgery and years of hormone therapy that can come with an array of unpleasant side effects. However, a new drug candidate could hold the potential to eliminate the need for such a grueling process, after a study in mice found that it wiped out a range of breast tumors in a single dose.

Breast cancer is one of the most common types of cancer, and though more people than ever are surviving it, that doesn’t mean that treatments are perfect. For people diagnosed with ER+ breast cancer – meaning the tumor has receptors for the hormone estrogen, which triggers the cells to grow – the usual course of hormone therapy can have an impact on the entire body.

Side effects can include pain, sexual dysfunction, and fatigue, while treatment is also associated with an increased risk of bones becoming weak and brittle or developing endometrial cancer. Even if a person is able to tolerate all of that, there’s still a reasonably high chance of the cancer coming back within five years. So, what’s the solution? 

Researchers have long been attempting to find an answer in the form of a more precise and effective treatment that targets cancer cells with minimal side effects. Part of that effort includes Dr Paul Hergenrother and colleagues, who’ve been working on a small molecule drug called ErSO.

ErSO works by causing tumor cells to swell and die via a process called necrosis. Previous research in mice had found it to be pretty effective at this – but it still had some unwanted side effects in its initial form. Hergenrother and colleagues have now developed a derivative of the molecule, ErSO-TFPy, that appears to eliminate that problem while remaining effective.

In three different mouse models of ER+ breast cancers, the researchers found that a single dose of ErSO-TFPy was enough to either nearly or completely eradicate tumors across a whole range of sizes. It was also found to have minimal side effects, something that was demonstrated in rats and dogs in addition to mice.

It’s undeniably an exciting prospect – a single dose of a super-effective treatment could minimize the chance of side effects and prevent breast cancer patients from having to endure years of therapy.

“An anticancer regimen that consists of a single dose, or a handful of doses, could change the face of breast cancer treatment,” the study authors write.

However, it’s important to note here that these results aren’t guaranteed to translate to humans. Even though the team also found that the drug was able to effectively kill cultured human ER+ breast cancer cell lines, a model animal or cells in a dish isn’t the same thing as a fully-fledged human being with all of the complex physiology and biochemistry that entails. 

As such, there’s a long road of further research ahead before ErSO-TFPy can make it from the lab to the clinic, which the study authors acknowledge.

Nevertheless, they remain hopeful.

“It is very rare for a compound to shrink tumors in mouse models of breast cancer, let alone completely eradicate those tumors with a single dose, so we are eager for ErSO-TFPy to advance for treatment of breast cancer,” Hergenrother said in a statement.

The study is published in the journal ACS Central Science.

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