Double whammy. Researchers have turned up a pill against heartburn that could be used to treat lung cancer and an antiepileptic pill that might treat Crohn's disease.
Credit: Marina Sirota
Posted on 08/17/2011 9:31:27 PM PDT by neverdem
What if a cheap medicine sold over the counter turned out to be a cure against cancer or another deadly disease? Scientists have devised a new way of predicting such unexpected benefits of existing drugs, and they have confirmed two potential new therapies just to prove the point. "This promises new uses for drugs that have already been tested for their safety and offers a faster and cheaper way to new medicine," says Atul Butte, an expert in bioinformatics at Stanford University School of Medicine in California, who conducted the study.
There are plenty of examples of drugs originally developed to treat one disease that turned out to help another: Acetylsalicylic acid (aspirin) is not just a pain killer but is also used to reduce the risk of heart attack. And when a blood pressure drug called sildenafil was discovered to have an unexpected side effect, it went on to become the erectile dysfunction blockbuster now known as Viagra. Such crossovers can save drug developers a lot of time and money. Developing a single new drug on average takes more than a decade and costs about $800 million. Existing drugs have known safety profiles and are approved for human use, so they can be rapidly evaluated for new indications.
"But most repurposing of drugs is still due to chance observations or educated guesses," Butte says. In today's issue of Science Translational Medicine, he and his colleagues present a more efficient way of finding such new uses for old drugs: by bringing together data on how diseases and drugs affect the activity of the roughly 30,000 genes in a human cell. Researchers have collected information on which genes are activated or silenced in certain diseases and by certain drugs for many years. "Our hypothesis was, if a disease is characterized by certain changes in gene expression and if a drug causes the reverse changes, then that drug could have a therapeutic effect on the disease," he says.
To find such opposing pairs, Butte and colleagues used public databases and compared the data for 100 diseases with that for 164 drug molecules. They found candidate therapeutics for 53 of the diseases. Many matches had already been discovered and turned into therapies, but others were completely unexpected. For example, the analysis predicted that an epilepsy drug called topiramate would be active against inflammatory bowel diseases such as Crohn's disease. And the over-the-counter drug cimetidine, which inhibits acid production in the stomach and is used to treat heartburn, matched a certain type of lung cancer.
To confirm this latter link, the researchers investigated the compound in a mouse model of lung cancer. They showed that it slowed the growth of human lung cancer cells but not kidney cancer cells in these mice. Similarly, giving topiramate to rats with colitis reduced swelling and ulceration in the animals.
John Overington, a computational chemical biologist at the European Bioinformatics Institute in Hinxton, U.K., is not convinced that these two particular drugs will get very far. "Topiramate hits quite a lot of targets and has complex side effects, while the doses needed for functional effects for cimetidine seemed high," he cautions. But he praises the paper's main idea. "This is a really important concept; it is almost like they are looking for an antidote to a disease." Stefan Schreiber, an expert on the genetics of inflammatory bowel diseases at University of Kiel in Germany, agrees that the idea behind the papers is more important than the two drug candidates. "This is a proof of principle," he says. "The main point is that someone has taken all of this available genomic data and shown what you can do with it."
The opportunities are growing rapidly, Butte says. "When we started the project 5 years ago, we had data for a hundred diseases and 164 compounds, he says. "Today it would be about 1400 diseases and 300 molecules." Butte hopes that scientists and pharmaceutical companies will continue to make data publicly available. The unknown benefits of many more drugs are just waiting to be discovered, he says.
“it is a violation of federal law to use this product in any manner not intended by the manufacturer”.
You see this warning on everything.
It also applies to drugs, it’s illegal to use a drug for another purpose with it first being approved for that purpose again, after new clinical trials.
No it's not, or physicians would be getting fined or prosecuted left and right for off label indications. It happens all the time. Eventually, they get FDA approval to put a new indication on the label after FDA testing.
How would it be discovered if it was illegal? The pharmacist doesn't know why the drug is being prescribed. If the drug works, the patient gets better.
It most certainly IS illegal, just not usually prosecuted at the moment.
Another of those wonderful federal laws that is selectively enforced.
A change in Fed. regulatory agency personnel, focus, definition of the crime, a determination to levy fines for revenue, all sorts of minor changes could result in the law suddenly being enforced.
Here we are in the 21st century, and here you are advocating trial and error to replace science.
No thanks.
Physicians and drug manufacturers get prosecuted left and right for ON LABEL INDICATIONS !
Off label indication abuse gets them disbarred and sent to jail.
MDs can go off label as they wish.
I found this in Wikipedia, which supports my position on off-label drug use:
"The FDA approves a drug for prescription use, and will continue to regulate the pharmaceutical industry through the work of the Division for Drug Marketing, Advertisement and Communication (DDMAC).[3] The FDA does not have the legal authority to regulate the practice of the medicine, and the physician may prescribe a drug off label. Contrary to popular notion, it is legal in the United States and in many other countries to use drugs off label, including controlled substances such as opiates. Actiq, for example, is commonly prescribed off label even though it is a Schedule II controlled substance. While it would be legal for a physician to independently decide to prescribe a drug such as Actiq off-label, it is illegal for the company to promote off-label uses to prescribers. In fact, Cephalon, the maker of Actiq, was fined for illegal promotion of the drug in September 2008.[4] Under the Food, Drug, and Cosmetic Act (FDAC) at U.S.C. 21 §§301-97, manufacturers are prohibited from directly marketing a drug for a use other than the FDA approved indication. The Food and Drug Administration Modernization Act of 1997 created an exception to the prohibition of off-label marketing. Manufacturers are now able to provide medical practitioners with off-label information in response to an unsolicited request. 21 U.S.C. §360aaa-6.
So, since Wikipedia is not invariably correct, possibly you could discussed a prosecution of off label prosecution? Or of a doctor being "disbarred" and sent to jail for it? Any incident?
Correction of my previous post:
So, since Wikipedia is not invariably correct, possibly you could discuss a sample prosecution of off label prescription? Or of a doctor being “disbarred” and sent to jail for it? Any incident?
Nope. Wrong. It’s legal for a doctor to prescribe a drug for off label use, it is NOT legal for the drug manufacturer to advertise the drug for off label use, although it may provide the physician with information about off label use under certain circumstances.
I am neither a doctor or lawyer, but I am very familiar with selective prosecution.
What may be being “Allowed” is not necessarily the same as “Completely Legal”.
As I said, a change in the enforcement winds may have drastic effects on this practice.
Try getting any Fed. agency to bless it in writing.
This statement of yours:
“...it’s illegal to use a drug for another purpose with it first being approved for that purpose again, after new clinical trials.”
is wrong.
Well, I take that as fact from numerous prior articles on the subject which also stated so.
The contention that new clinical trials are required before being prescribed for new uses has been made many times, by medical writers.
So your contention is that they are also ALL wrong.
Not a major issue for me, I don’t write prescriptions.
I believe that the intent of the article (study) is to encourage drug manufactures not doctors to explore new uses of existing approved drugs to fight diseases that those drugs are not currently approved to treat.
The drugs then could be more quickly approved for the new uses because of preexisting data on their side effects and safety.
Since you don’t write prescriptions, I would not expect you to have any kind of accurate knowledge about off label use.
I work for a Big Pharma company. All I can say is you couldn't be more wrong if you tried.
No thanks.
onona made that comment, not me.
Clinical studies are trial and error because species are different. It's smarter to check drugs in lower forms of life before human clinical trials. These two studies reported the results of computational bio-informatics on genetic/genomic signalng pathways. It was hardly trial and error.
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FReepmail me if you want on or off my health and science ping list.
Thanks.
I like this. These older drugs should be relatively inexpensive and they have a track record.
http://www.firstwordpharma.com/node/881520
http://www.firstwordplus.com/msl_kol_engagement_ensuring_compliance.do
Hope the links work for you !
Big difference.
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