Who Knew? New Off-Label Uses for Well-Known Drugs, Part II

Recall in Part I that, although Pfizer’s angina treatment did little for the chest pain, it changed multiple thousands of lives in the bedroom when the scientists reincarnated it as Viagra. And date rape drug ketamine earned itself some points from ‘the good side’ when one study author found it to be “the biggest breakthrough in depression in half a century.”

What more could there be, what hidden potential is just lying behind drugs we thought we knew?

Look at well-known cancer drug Avastin.  It has been used to treat late-stage colon, breast, and long cancers–and it’s recently been found to treat age-related macular degeneration. Not as glamorous as erectile disfunction or date rape drugs, but important nonetheless.

In 2006 Lucentis was approved for age-related macular degeneration, with claims that it could stop the disease’s progression–and maybe even reverse it. But recently doctors have argued that Avastin is successful in producing the same results–at a fraction of the cost. With Lucentis at around $2,0000 per treatment, and Avastin $150 per treatment, its advantage is clear. However, Avastin is not FDA-approved for AMD, and Genentech, the company that markets both drugs, has stopped sales of Avastin to the pharmacies that cut them into the proper doses for AMD.  Its future as a treatment remains to be seen.

But Avastin doesn’t ist around waiting. Just this past year, a team of researchers at Massachusetts General decided to try Avastin on neurofibromatosis type 2, a rare or orphan disease typified by the growth of noncancerous tumors in the nervous system. Found in about 1 in 25,000 people, the tumors can often impact the auditory nerve,  resulting in deafness. Traditionally, treatment has consisted of surgery or radiation, but both have negative effects. Operations on those with tumors the size of small plums almost always yield deafness, and radiation can actually lead to cancer.  Of the 10 patients in the  study, the tumors of six shrank by 20% or more;  of the seven patients who had hearing loss before the treatment, the hearing of six improved or remained the same, rather than deteriorating further as would commonly occur in the disease.

We’re up to 5 new uses here as well.

Every year in the U.S. 100,000 people are diagnosed with kidney failure, the final stage of chronic kidney disease (CKD).  Diabetes is the most common cause of kidney failure, making up 44% of all cases.  At the end stages of the illness, people will have to undergo dialysis or transplantation. But, in a surprise finding, ACE inhibitors which treat high blood pressure and weak heart muscles turn out to also slow the progress of kidney disease significantly.


Other illnesses are managed by surprising medications, as well. For example, enuresis, or persistent bed-wetting past the age of 5, found in 15% of 5-year-olds, is treated by. . . .tricyclic antidepressants and desmopressin nasal spray. Scout’s honor.

And then just this year, in a surprise to those familiar with heart disease, a study of chelation therapy, which involves infusing agents that remove metals from the blood stream, found that it reduced the rate of death and heart-related problems in a clinical trial sponsored by the National Institutes of Health.

In the very old wine in new bottles category, take acetylsalicylic acid, better known as aspirin, used for ages to alleviate pain.  The U.S. took the formula from the German Bayer as spoils of World War I.  And it was a wise little gift we handed ourselves, the gift that keeps on giving. Besides anti-platelet action,  75 mg a day of aspirin a day reduces incidence of colorectal cancer, providing cardiovascular protection at the same time. Aspirin is in the rare category of drugs that cost little and can effectively replace others that are expensive.

Given that it takes approximately $1 billion to develop a new drug, companies are thrilled when they discover one of their medications can treat an apparently unrelated disorder. The research studies can bypass the first few steps–finding the molecule, preparing it for medical use  and testing for toxicity.

Just to leave you with a few more: Astemizole,used to treat allergic rhinitis, has been found to treat malaria; Evista for osteoporosis has now been found helpful in breast cancer, and Rheumatatrex and methotrexate, both used to treat cancer, help treat rheumatoid arthritis and other immune disorders. Provigil originally treated narcolepsy, and is now used to manage chronic depression; Inderal for high blood pressure blood pressure also manages migraines.

Let’s hope researchers continue this lucky streak. The autoimmune-cancer link has produced a number of cross-overs. Surprise uses for old drug save lives and save millions, if not billions, of dollars.  It’s really recycling at its best.

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