While COVID-19 vaccines present our greatest opportunity to turn the tide against this pandemic, hundreds of thousands of Americans are likely to die waiting for their vaccines. Our best hope of saving these lives is to find more effective treatments right now. But we don’t have to rely entirely on developing treatments from scratch; we must continue to look at repurposing existing medications.
This is particularly personal for me. I’ve nearly died five times from an illness, Castleman disease (CD), that ignites a deadly immune response in which the body attacks itself — a cytokine storm — just as COVID-19 often does. This also means that CD puts me at increased risk of dying from COVID-19. But I am alive today because of a repurposed treatment. My medication was approved by the Food and Drug Administration (FDA) for another condition two decades ago but had not been tried for CD. I dedicated my life to finding an existing drug that could save me. In 2014, I discovered it. Now, it is saving other patients’ lives too.
There are 2,500+ FDA-approved drugs but few incentives to repurpose them for other diseases. In March 2020, I redirected my Center for Cytokine Storm Treatment & Laboratory (CSTL) at the University of Pennsylvania to launch the CORONA Project to identify new or repurposed treatments for COVID-19. Now it’s the world’s largest database of COVID-19 treatments, including 350+ medications administered to 250,000+ patients. It has been accessed 20,000+ times by scientists at Google Health, FDA, and elsewhere.
So far, a medication called dexamethasone has been the most effective treatment. Traditionally, it has been used to combat a myriad of conditions – ranging from arthritis to breathing problems. It works by calming patients’ overactive immune systems and costs ~$1/day. Months before it was proven effective through a large trial, we identified it as a highly promising treatment approach. Unfortunately, it is only effective in a subset of patients.
Our continued search has revealed ten other promising drugs that we’re working with the FDA and others to move forward. These include drugs that boost the immune response like interferon (FDA-approved for leukemia), drugs that suppress the immune response like colchicine (FDA-approved for gout), and even psychiatric drugs like fluvoxamine (FDA-approved for obsessive-compulsive disorder) that work through an as-yet-determined mechanism. CORONA has also revealed significant inefficiencies: some clearly ineffective drugs have dozens of trials underway. Other drugs that have shown promise in small clinical trials have no large trials planned.
Fortunately, the new administration already released an executive order calling for expanded research into COVID-19 treatments. To succeed, we will need to systematically track all treatments in one central place, quickly identify promising approaches, and provide the resources to perform large, well-designed trials for these treatments.
As someone who is alive thanks to a repurposed drug, I feel compelled to advocate for this even beyond COVID-19. Wouldn’t a beautiful silver lining of this awful pandemic be the rapid discovery of new treatments for deadly conditions from the pool of already FDA-approved treatments? How many treatments are hiding in plain sight? The FDA only considers new disease approvals for existing drugs when drug makers apply for them. We need to create incentives and build a national effort to ensure that approved treatments are utilized for all diseases that they can help.
This month marks seven years that I’ve been disease-free on my current treatment. Now I desperately hope that our work and the work of researchers worldwide will enable many years of disease-free life for innumerable patients with COVID-19 and other diseases. We’re going to keep doing everything we can to turn our hope into action.