Joseph R. Anticaglia, MD
Medical Advisory Board
The current pandemic has energized scientists around the globe to come up with targeted treatments and effective vaccines for COVID-19. Investigators in the U. S. are searching for off-label uses of pharmaceutical drugs.
A drug may be initially approved by the Food and Drug Administration (FDA), for example, to treat cancer. However, physician discovered that beyond the initial indication to fight cancer, it could be used to effectively and safely to treat other conditions or symptoms. This off-label use of drugs has also been called repurposing or repositioning of pharmaceutical medications.
Dr. Vivek Iyer, a Mayo Clinic physician, went off label to treat a rare, hereditary disease. He described the agony of a middle aged woman with severe nosebleeds. The patient talked about how the slightest touch to her nose could provoke a nosebleed that gushed as if “you would turn on the tap.”
She had been diagnosed as having hereditary hemorrhagic telangiectasia (HHT). It’s a mouthful to say but unfortunately she often had a mouthful of blood because of it.
This condition can cause anxiety, depression, iron deficiency anemia, shortness of breath and put you at greater risk to have a heart attack. People with HHT have abnormal connections between arteries and veins. These malformations (AVMs) are found in the nose but can be found in other parts including the brain, liver and lungs.
Dr Iver diagnosed her condition as HHT and thought outside the box. I imagine he might have asked himself: The malformed, connected vessels have a tendency to grow and rupture spontaneously. What if we used a medication that blocked the growth of her AVMs? Might we prevent a stroke, a pulmonary embolism and prevent, or at least, reduce the frequency of the nosebleeds? Can we improve the quality of her life and do it safely and effectively? Enter Avastin.
Avastin (bevacizumab) is a cancer drug that prevents tumors from growing new blood vessels. It’s approved — indicated to treat metastatic colorectal cancer. The repurposing and successful use of this drug in the treatment of Dr Iver’s patient has revitalized her life. As she puts it, “It’s given me my life back,”
A notice in Oxford News & Events on June 16, 2020 reported that researchers at Oxford University in the United Kingdom concluded the use of dexamethasone saves lives in hospitalized patients with severe respiratory complications of COVID-19 (SARS CoV-2).
The off-label indication of dexamethasone (Decadron) for severely ill COVID-19 patients has become the standard of care for such patients in the UK. The drug is a corticosteroid that has been around since the 1950s; it’s inexpensive, safe and is available in generic form. Dexamethasone is the first drug to reduce deaths in COVID-19 patients.
Drug repurposing or repositioning takes advantage of the facts that the medication has been tested and approved for use in humans This means the drug is considered safe, and therefore, eliminates the need for phase 1 clinical trials. As a result of bypassing phase 1 clinical trials, researchers save time and money. In addition, information is available about its chemical makeup, dosage and potential side effects.
Off-label drugs are commonly prescribed by physicians. The alternative use of drugs not approved by the FDA, has benefited many patients. Nevertheless, in the COVID-19 era, physicians must be attentive to the dosage, the age of individuals and their safety before prescribing off-label medications. Hydroxychloroquine is a sober reminder that efficacy and the patient’s safety come first and foremost.
- Sudeep Pushpakom1, Francesco Iorio; Drug repurposing: progress, challenges and recommendations; Nature reviews, October, 12, 2018
- Chiang, A. P. & Butte, A. J. Systematic evaluation of drug-disease relationships to identify leads for novel drug uses. Clin. Pharmacol. Ther. 86, 507–5 10, 2009
- Vivien Williams; Mayo Clinic goes off-label to treat severe bleeding disorder; April 26, 2019
- J. W. Austin … C. J. Hall; Drug Repositioning; Methods in Cell Biology; The Zebrafish, 2017
This article is intended solely as a learning experience. Please consult your physician for diagnostic and treatment options.