A highly effective new drug, known as OTS964, could be the miracle cure for certain kinds of cancer that we've all been waiting for. The medication, which can be taken via a simple pill or through injection, has been shown to completely eradicate cancers that have been transplanted into mice, reports MedicalXpress. Human trials are set to begin as early as next year.
The drug works by inhibiting a protein called TOPK that several types of cancer, such as lung and breast cancer, require to properly divide. Since the protein is rarely expressed in healthy, non-cancerous cells, the treatment targets cancer cells specifically, so there are very few — if any — negative side effects.
"We identified the molecular target for this drug ten years ago, but it took us nearly a decade to find an effective way to inhibit it," said study author Yusuke Nakamura, MD, PhD. "We initially screened 300,000 compounds and then synthesized more than 1,000 of them, and found a few that were likely to work in humans. We focused on the most effective. We think we now have something very promising."
So far the drug has only been used in mice, but the mice were harboring human cancer cells. After being administered OTS964 intravenously twice a week for three weeks, the tumors disappeared completely for 5 of 6 mice within a month of their first treatment. The drug was even more effective when taken in pill form, curing the cancer in all of the mice. Mice that took the drug via mouth showed a reduced white blood cell count at the end of the trial period, but all of them recovered fully within two weeks.
Seeing these results was a "quite an exciting moment," said Nakamura. "It is rare to see complete regression of tumors in a mouse model. Many drugs can repress the growth, but it is uncommon to see them eradicated."
The study looked primarily at lung cancers, but there is good reason to believe the drug will also be effective on other cancers that also rely on TOPK to divide properly, such as breast, brain, liver and bladder tumors. It may even work against certain types of leukemia. But the drug's effect on these other cancers is still speculative, pending further research.
Though this initial study was small, its success means phase-1 clinical trials can begin as soon as the fall of 2015.
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