(RxWiki News) Orphan drugs are those that have been abandoned because they were proven ineffective against the disease they were designed to treat. Thousands of these medications are sitting on shelves in labs around the country. And one may be a powerful cancer drug.
The drug was going to be used to treat sleep disorders.
Now it appears this so-called orphan drug has powerful cancer fighting properties that could be used against a number of different malignancies.
"Ask your pharmacist to explain your medications."
Researchers at the Fred Hutchinson Cancer Research Center used advanced genome analyzing technology that could speed the development of better cancer drugs.
The team of investigators, led by corresponding author, Carla Grandori, MD, PhD, an investigator in the Hutchinson Center’s Human Biology Division, used robotic technology to reveal the fatal flaws of cancer cells driven by an oncogene called Myc.
Myc is found at excessively high levels in a number of cancers including malignancies of the brain, breast, lung, ovary and liver.
Dr. Grandori says that "Myc-driven cancer cells have an Achilles heel." It uses other genes to repair the damage Myc causes in the cancer cells. Going after those genes, more than 100 of which the team identified, "can ripple the cancer’s ability to grow," she explains,
A drug originally designed to treat sleep problems attacks and blocks a particularly important enzyme (CSNK 1 epsilon) that's especially good at killing Myc-driven cancer cells, while sparing normal cells.
Researchers used the compound to treat mice that had a nervous system cancer known as neuroblastoma. The mice that received the orphan drug thrived and the tumors dissolved, while the untreated mice quickly died.
“This is one in a series of recently published studies that highlight the power of high-throughput screening technologies to identify novel therapeutic targets in cancer," said Donald P. McDonnell, PhD, the Glaxo-Wellcome Professor of Molecular Cancer Biology and Chairman of the Department of Pharmacology and Cancer Biology at Duke University School of Medicine.
"What makes this study so exciting is that it has resulted in the identification of a drug that hits a pathway whose function is absolutely required for cancer cell proliferation but which has generally been considered undruggable," McDonnell told dailyRx.
"The fact that one of the drugs identified has already been in clinical development for another indication could mean that it could be available for clinical testing in cancer in the not too distant future,” said McDonnell, who also serves as the Associate Director for Basic Research for the breast, ovarian and prostate programs within the Duke Cancer Institute.
“We’ve barely scratched the surface,” Dr. Grandori said. “These techniques are incredibly powerful, but they’re new and not widely known. There are thousands of researchers who could apply this approach to their work. In the right hands, it could speed up the development of new cancer therapies a thousand-fold,” she concludes.
This research was published in the May 21, 2012 issue of the Proceedings of the National Academy of Sciences.
Funding support came from Ben Towne Endowment and the Cancer Consortium. No conflicts of interest were reported.