Breakthrough Single-Dose Drug Wipes Out Breast Tumors in Mice Without Side Effects

Credit: Angiola Harry

In the search for better and better treatments for breast cancer, medical researchers at the Univ. of Illinois have synthesized a remarkable substance.

In small tumors, a single dose of ErSO-TFPy eliminated them entirely, while the same dose significantly shrank large tumors. In both mice and rats, no deleterious side effects were observed.

Limited to estrogen receptor-positive breast cancer (ER+) the compound addresses many common concerns with existing treatment that involves choking off the supply of estrogen on which the tumors rely.

“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,” said Paul Hergenrother Ph.D., lead author on the paper describing the treatment, and decorated biochemist from the University of Illinois.

ER+ breast cancer is the most common form of the most common cancer in the United States. At current rates, 13.1% of women will develop breast cancer in their lives, accounting for 15% of all cancer diagnoses in the US.

ER+ breast cancer treatment will typically consist of surgery to remove the tumors, followed by hormone therapy to starve any remainder. Effective treatment like these has raised the 5-year relative survival rate in breast cancer patients in the US to well above 90%, but several undesirable side-effects tend to follow.

The reduction in estrogen frequently results in blood clots, sexual dysfunction, and osteoporosis. Additionally, hormone therapy does nothing to prevent recurrence or resistance in tumors, leading cancer researchers like Hergenrother to seek additional options.

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New research published on Wednesday in the journal ACS Science demonstrates how ErSO-TFPy, a spinoff of the previously developed treatment dose ErSO, achieved complete regression of smaller tumors in mice without any of these side effects.

The effect was robust and independent of tumor size with the eradication of even very large tumors (500−1500 mm3) being observed in the paper. Mechanistically, the authors write, these tumor regressions are a consequence of rapid induction of necrotic cell death in the tumor and even more positively, are immune cell independent.

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The study also involved dosing transplanted human breast cancer tumors in mice, during which the same regression rate and degree were observed, suggesting the medication will work in humans as well.

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