Unexpected Off-Label Drug Uses to Treat Various Health Conditions
"Off-label" drug use refers to the applications of medicinal formulations for purposes other than that for which they were initially developed.
Off-label uses for drugs are often discovered accidentally, sometimes years after the medicine has been in circulation. Arguably, the most attractive aspect of the drugs described in this article with various off-label uses described is that most of them are notably more affordable than the newer, more expensive (patented) therapeutic interventions pushed by pharmaceutical giants to treat the same conditions.
In this article, we'll explore many of the most interesting off-label uses of commonly-prescribed drugs and how they may help patients who do not respond favorably to conventional treatments.
Hydroxychloroquine to Treat COVID-19
Of all the off-label uses of drugs in recent history, none has been more controversial than hydroxychloroquine as a COVID-19 therapeutic.
Hydroxychloroquine has been a staple of hospitals throughout the world since its introduction in 1971 as an effective anti-malarial prophylactic. Until 2020, its therapeutic application for combatting pathogenic infections had gone entirely unchallenged.
In fact, hydroxychloroquine is so versatile and widely effective in treating pathogenic infections that is included on the World Health Organization (WHO) "List of Essential Medicines" that every hospital and clinic should have on hand.
The controversy surrounding hydroxychloroquine's off-label potential to treat COVID-19 patients kicked off in the early days of the pandemic when the medical journal Lancet published a study that found "no conclusive evidence of [hydroxychloroquine's] benefit."
Following a torrential outpouring of contradictory evidence from frontline medical providers and research groups that actually indicates the great potential of hydroxychloroquine (in combination with other therapies, particularly zinc and vitamin D) to effectively treat COVID-19 patients when administered in the early stages of infection, the Lancet retracted its rushed (and apparently politically-motivated) study purporting to demonstrate the drug's cardiovascular dangers.
Minocycline and Other Antibiotics to Treat Major Depression
Depression remains a major public health challenge in the United States and across the West. Several recent studies, in fact, have suggested that the problem is getting worse.
Western nations consistently break records regarding the numbers of SSRIs (serotonin reuptake inhibitors) that are prescribed to patients with major depression annually, seemingly with little to no effect in reducing the overall burden of depression (and corresponding skyrocketing suicide rates across demographics).
While SSRIs and other conventional treatments for major depression work for many patients, they conversely prove ineffective for many and also carry substantial side effects.
Enter minocycline, a common antibiotic that has been routinely used since the 1970s in hospitals throughout the world. Only recently, though, have researchers taken an active interest in minocycline's potential to effectively treat depression.
The preliminary research indicates that minocycline owes its anti-depressive properties to its ability to tamp down systemic inflammation, which is often found to be elevated in patients with depression.
According to Dr Olivia Dean, a leading Australian medical researcher, "We believe that minocycline is targeting more recently understood biological factors [that trigger depression], including inflammation. Specifically, minocycline reduces brain inflammation in cell models."
Propranolol (Inderal) for Performance Anxiety
Propranolol is a common beta-blocker pharmaceutical therapy, often sold as Inderal in the United States.
Beta-blockers are traditionally used to treat high blood pressure, heart failure, and angina (chest pain). They achieve improvements in these symptoms by calming the heart rate and reducing blood pressure in the patients who take it.
Interestingly, propranolol has been found to prevent performance anxiety by these same mechanisms. These drugs alleviate the symptoms associated with performance anxiety (i.e., "stage fright" or test anxiety) by calming the cardiovascular system.
All that is needed to reduce anxiety performance for most patients is a relatively low dosage (10-20 mg) administered about 30 minutes before the anxiety-producing event occurs.
Clomiphene (Clomid) for Testosterone Deficiency and Male Infertility
Clomiphene, marketed, as Clomid to US patients, was originally designed and approved by the FDA to treat female infertility. However, clomiphene has also shown clinically impressive potential for treating male fertility as well when prescribed off-label for this purpose.
In men affected by hypogonadism (testosterone deficiencies caused by under-performing testes), clomid both boosts testosterone levels and increases sperm counts in these men.
While testosterone replacement therapy (TRT) using synthetic testosterone is still considered the "gold standard" for correcting relevant hormonal deficiencies, clomiphene offers an unexpected and effective alternative treatment modality.
Many endocrinologists utilize clomiphene concurrently with synthetic testosterone to achieve improved hormonal profiles in their male patients.
Modafinil (Provigil) for Cognitive Enhancement
Modafinil, sold as Provigil to US patients, is a commonly prescribed therapeutic for promoting wakefulness in patients affected by narcolepsy, a neurological condition in which individuals with the condition fall asleep throughout the day due to an impaired sleep-wake cycle (circadian rhythm).
Due to modafinil's interactions with the nervous system, it also carries recently discovered potential to improve working memory and overall cognitive function in individuals who take it for these off-label purposes.
As such, modafinil has become increasingly popular in the "nootropics" community. The term "nootropics" refers to a loosely defined category of supplements that are intended to promote improved cognitive function. Nootropics are alternately referred to as "smart drugs."
Metformin for Cancer Prevention, Improved Cardiovascular Function, and More
Metformin was originally introduced to the US market as a diabetes treatment because it inhibits the synthesis of blood sugar by the liver, thereby reducing overall blood sugar levels, a critical therapeutic goal in the treatment of patients with diabetes and other metabolic disorders.
Due to its ideal safety profile and reliable efficacy, metformin is frequently the very first medication prescribed to diabetic patients following their diagnosis.
Since widespread use of metformin began in the US in 1995, many off-label uses for the drug have been discovered. They include:
- Improved insulin sensitivity (critical for preventing diabetes before it develops).
- Reduced cancer risk. Metformin has been seen to activate the AMPK pathway, a key factor in tumor suppression as well as anti-aging.
- Cardiovascular protection. Heart disease remains the #1 killer of Americans, so any unintended off-label aid from pharmaceuticals in this department is welcomed by doctors treating affected patients. Metformin is believed to protect heart function primarily by preventing oxidative stress and resulting damage to heart cells.
- Lowered levels of harmful cholesterol. Metformin has been shown to decrease unhealthy low-density lipoprotein (LDL) levels without affecting beneficial high-density lipoprotein (HDL) levels.
These promising off-label applications of metformin represent only a handful of the many therapeutic potential uses of the drug, ranging from weight loss to anti-aging.
Rapamycin for Cognitive Enhancement
As an effective immunosuppressive agent, rapamycin was originally intended for use in organ transplants to prevent rejection by the host and is particularly effective for this purpose in kidney transplants.
However, rapamycin has gained off-label popularity for its ability to prevent the cognitive decline associated with Alzheimer's disease and other neurodegenerative conditions, a major public health challenge that has largely gone unanswered as more and more Americans are diagnosed with these types of as-yet poorly treated debilitating diseases each year.
In addition to metformin, the nootropics community has also become interested in rapamycin's potential cognition-enhancing properties, with many including the drug in their nootropic "stacks," the colloquial term for nootropics supplementary regimens.
Trazadone is a common (and very cheap) drug that was originally marketed to treat anxiety, depression, and insomnia. The mechanism by which it works for these purposes is its function as a serotonin and adrenergic receptor antagonist as well as a weak serotonin reuptake inhibitor (SSRI).
However, many healthcare professionals also administer trazadone off-label to their patients to treat:
- Obsessive-compulsive disorder (OCD).
- Alcohol and benzodiazepine withdrawal syndromes.
- Erectile dysfunction.
- Schizophrenia (in combination with other therapies).
- Chronic pain.
The Final Word on Off-Label Pharmaceutical Use
Although the drugs explored in here were originally designed and approved by the FDA to treat very specific conditions, other unanticipated clinical applications are frequently discovered through trial and error.
The drugs described in this article, while carrying significant science-backed potential as off-label treatment modalities to conventional therapies, should only be taken for these alternative purposes under the careful supervision of a medical professional.