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Deworming Pill Could Treat Liver Cancer


It doesn’t happen all that often, but sometimes the medical community will discover that a drug or medication that was intended to be used for one thing, can in reality be used to also treat something potentially unrelated.

Viagra, for example, is approved for use as a treatment for erectile dysfunction, and is also marketed under the name Revatio to treat pulmonary hypertension, a buildup of pressure in blood vessels leading to the lungs.

Avodart is approved for the treatment of enlarged prostate, Avodart, like Propecia, has been tested for prevention of prostate cancer as well.

Efudex is a skin cream that has been used for years to combat the early stages of skin cancer, may one day have a second use as a wrinkle-buster. We could keep going, but we think you understand the point.

Now in a recent discovering, it has been found that a popular deworming drug may show promise in the treatment of hepatocellular carcinoma (HCC), a cancer associated with underlying liver disease and cirrhosis that often only becomes symptomatic when it is very advanced, and is also the second leading cause of cancer deaths around the world. At the present, there is no effective treatment.

As with other conditions without treatments, the data that scientists need to understand and treat the disease may be sitting in plain view in databases that have barely been analyzed, says Atul Butte, MD, PhD, director of the Institute for Computational Health Sciences at UC San Francisco.

Bin Chen, PhD, a former postdoctoral scholar in Butte’s lab and now a faculty member in Pediatrics in the Institute for Computational Health Sciences, recently published a paper in Gastroenterology about using data-mining computational tools to identify a treatment for HCC. Here is how it played out.

Taking advantage of publicly available gene expression data, he first derived a molecular disease signature for HCC – looking at 274 genes that are either up or down regulated in cancerous liver tissues, but not in normal liver tissues. Then, he looked for drugs that were known to target those genes and found, to his surprise, that a close cousin of a deworming pill, when used in combination with the standard care drug, was highly effective at killing cancerous liver tissue that had been engrafted into experimental mice.

“We found these disease genes were reversed after six weeks of treatment in a patient-derived tissue in mouse model,” Chen said, adding that the advantage of the approach he developed is that it targets a host of genes at the same time, rather than simply targeting a single mutation.

“In this study, patients had similar gene expression profiles, but not identical,” Chen said. “In most cases, cancers have many mutations, and patients will relapse. Our approach might be used to control the disease, to make it a chronic condition rather than a lethal one.”

“Because our method is literally virtual, we can evaluate hundreds of drug candidates very quickly,” Chen said. “We looked at more than 1,000 drugs before discovering that the deworming pills were effective. This is a very efficient way to do drug discovery.”

While it may be a fair bit off before these drugs are used as an effective treatment, the process alone has opened the door and shown great potential in cross referencing thousands of medications for their benefits without relying on invasive human testing.

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