In what researchers are calling a ”breakthrough hiding in plain sight,” scientists have redesigned a 40-year-old deworming drug into a potent cancer-fighting compound, one that could one day be cheap, safe and even protective against some of the deadliest cancers.
The drug is mebendazole, familiar to parents, pet owners and doctors as a routine treatment for parasitic worms. But a team at Johns Hopkins University has discovered that one particular version of the drug, a crystal form known as polymorph C, behaves in the body in a completely different way, slipping past biological barriers that stop most cancer drugs in their tracks.
And that simple tweak could transform a humble worm medicine into a powerful cancer therapy.
Mebendazole comes in three natural “shapes,” or polymorphs. For decades, they were treated as interchangeable. The Hopkins team discovered they aren’t.
Polymorph C, the least studied of the three, appears to penetrate tumors far better including brain tumors, which are notoriously difficult to reach because of the blood-brain barrier.
According to the patent the researchers secured in September 2021, an oral pill made of at least 90% polymorph C reaches tumor sites at far higher concentrations than standard mebendazole. That makes it potentially much more lethal to cancer cells.
“Our goal was to see if we could take an already safe drug and make it truly effective against cancers where options are limited,” the inventors wrote.
The list of cancers polymorph C could target includes brain tumors such as gliomas and medulloblastomas; breast, colon, lung and pancreatic cancers; thyroid, ovarian, prostate cancers, and melanoma and various sarcomas.
The researchers even floated a bold possibility: that polymorph C could help prevent cancer in high-risk people when paired with anti-inflammatory drugs like celecoxib. Since chronic inflammation fuels cancer development, the combination could act as a chemopreventive shield.
In early tests on mice, animals that received polymorph C showed stronger tumor suppression than those given ordinary mebendazole, with the drug hitting tumors at “effective, therapeutic levels.” Toxicity remained “acceptable,” meaning the treated mice handled the drug well.
Researchers pushed the concept further by pairing polymorph C with elacridar, a drug that blocks cancer cells from pumping treatments back out. The combo kept the drug inside tumors longer — and extended survival in mice with aggressive brain cancers.
But it came with a warning: at higher doses and longer use, the combined treatment caused weight loss and even deaths in mice, underscoring the need for careful dosing in any human trials.
One of the most exciting aspects of the discovery is its simplicity. Mebendazole has been prescribed safely for decades. Doctors know its risks, its interactions, and its limitations.
That means scientists don’t have to start from scratch. Clinical trials could begin far sooner than with most experimental cancer drugs that require years of safety testing.
And because mebendazole is inexpensive and available globally, a cancer-killing version could become one of the rare treatments accessible far beyond elite hospitals.
Experts stress a familiar caveat: mice aren’t humans. A drug that works in the lab still has to survive the rigorous, expensive, and often unpredictable journey through clinical trials.
Researchers are still refining how to formulate the pill — whether granulated, coated, or micronized — to boost absorption. And they must determine how the human body handles polymorph C and whether it plays well with other medications.
Still, the evidence so far is promising enough that some scientists believe polymorph C could rewrite the fate of patients with the toughest cancers, especially those of the brain.
For decades, mebendazole was just a quiet, trustworthy drug sitting on pharmacy shelves. Now, thanks to a bit of chemical re-engineering, it is emerging as one of the most intriguing cancer candidates in years.
If clinical trials confirm what the early research suggests, the next big advance in cancer treatment may not come from a cutting-edge molecule — but from a familiar little tablet designed to kill worms.
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