Dr. Bruce Bloom, September 5, 2013

Drug and device repurposing in medicine and medical research is everywhere, but unless we look closely, we don’t see it.  Several recent studies show that 20% of all prescriptions are written off-label and for mental health drugs over 30%Almost 2/3 of all pediatricians say they prescribe drugs off-label to their patients  Off-label drug use is common in obstetrics since during the last five decades of activity the FDA had approved only two drugs for obstetrical indications.  Some drugs are used more frequently off-label than for their original, FDA-approved indications.  While this is drug repurposing in its most basic, much of this off-label prescribing is done without the benefit of scientific validation

Cures Within Reach supports the off-label use of drugs, especially in diseases for which there is no approved drug, and our mission is to find and support these off-label uses through scientifically validate them in small pilot clinical trials.  Often these ideas come to us from researchers at academic medical centers.  Other times they come to use from practicing physicians.  A 1996 study from MIT found that over 50% of the validated off-label uses for recently approved drugs came from practicing physicians.

There are new repurposing ideas in the news every day.  Recent studies indicate that a drug first used almost 100 years ago to treat African Sleeping Sickness (and still used for that purpose!) might work for kidney disease, liver disease and even autism.   It has some serious side effects in some of the people who use it, but it might be better than the alternative in life-threatening diseases for which there is no effective therapy.  And further research might lead to the creation of a brand new drug that works as well without the side effects.  That’s what happened over the last 15 years as Thalidomide what repurposed for the deadly blood cancer multiple myeloma, and then the pharmaceutical company Celegene created Revlimid, and is now working on further analogs of Thalidomide.

Just about every drug ever fit for human use has the ability to be repurposed for other disease indications, and many of them, like metformin, propranolol, sirolimus might eventually find their way into multiple treatment regimes.  Every time we ask for new repurposing proposals we get numerous grant applications repurposing these drugs, often in combination with each other, or with other drugs.  The opportunities to repurpose drugs to help patients is almost unlimited.  It’s the first thing I’d look for if I was a patient with a currently untreatable disease-it’s faster, safer and less expensive than finding a brand new drug.   If only repurposing could turn a profit, everyone would be doing it!  As for now, there’s Cures Within Reach, our researchers and clinicians, and our philanthropic funding partners.

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