Drugs Discovered to Help Conditions Far Different from Their Original Indications: Off Label Drug Use

or Why Am I Taking a Seizure Medication for Foot Pain?

As the physician spouse of a wonderful woman living with ovarian epithelial cell cancer the recent article in Cancer documenting that women with type 2 diabetes who take metformin to have better survival rates than control patients with ovarian cancer who do not take metformin when controlled for confounding variables caught my attention. Metformin is by far the most commonly used drug used to treat type 2 diabetes mellitus, and is inexpensive and well tolerated. It also prompted me to think about other medications which were found to be effective therapy for conditions far different than their FDA approved original indications. Here are a few such drugs that have been uncovered in the years I’ve been in practice.
Drugs Found Useful for Indications Quite Different than Their Original Use:

  1. Methotrexate for Ectopic Pregnancy: For the early years I practiced medicine the discovery of a tubal, or other ectopic pregnancy was a surgical emergency. Diagnosis was often made much later that it is today due to advances in HCG testing and ultrasonography. Surgery to remove the fallopian tube was the standard of care, with “tube salvage surgery” sometimes possible. Now treatment with methotrexate can lead to the early ectopic pregnancy dying and being resorbed with little or no damage to the fallopian tube, and lower risk of infertility or recurrent ectopic pregnancy while avoiding the need for an invasive surgery.
  2. Beta-blockers for Congestive Heart Failure: The discovery that the use of certain beta-blocker drugs including carvedilol or metoprolol succinate, especially in conjunction with ACE inhibitors or ARB medications can improve cardiac function, reduce morbidity and mortality, and markedly improve the lives of patients with CHF has been a major breakthrough in modern cardiac care.
  3. Anticonvulsants for Neuropathic Pain: The use of gabapentin and Lyrica, drugs originally used for seizure control in epilepsy, for the treatment of post-herpetic neuralgia, diabetic peripheral neuropathy, trigeminal neuralgia and other neuropathic pain syndromes has become first line therapy for these exceptionally difficult pain conditions.
  4. Minoxidil and Finasteride for Hair Loss: Not a life-threatening condition, but an interesting use of drugs originally used for refractory hypertension (minoxidil) and benign prostatic hypertrophy (finasteride).
  5. Betamethasone for Lung Maturation in Preterm Labor: Betamethasone is a corticosteroid, typically used for systemic inflammatory conditions, that has been found to accelerate lung maturation if preterm infants at risk for pulmonary dysplasia from prematurity. Betamethasone is now routinely given in certain situations where a preterm birth is anticipated to reduce the chances of severe pulmonary disease in the baby.\SSRI drugs for Premature Ejaculation: Premature ejaculation is a common though often under-recognized condition, and SSRI antidepressant medications like sertraline have become a commonly used and often effective therapy.

Undoubtedly we will continue to see unanticipated benefits of medications discovered by observant patients and physicians from time to time.  I’m sure these are only a few examples and look forward to seeing comments to add to this list.

Leave a reply