Summary
Overview
This episode explores the untapped potential of drug repurposing—using existing FDA-approved medications to treat diseases they weren't originally designed for. Through the story of physician-scientist David Fagenbaum, who saved his own life by repurposing an existing drug, and interviews with economists and FDA officials, the episode examines why so few resources go toward this high-impact, low-cost approach to saving lives, and what innovative funding mechanisms could change that.
The Brain-Eating Amoeba and an Unlikely Cure
The episode opens with the story of balamuthia, a rare brain-eating amoeba with a 90% fatality rate and no approved treatments. When a clinician in San Francisco tried treating a patient with nitroxeline—a European UTI drug that showed unexpected amoebicidal properties—the patient had a remarkable recovery. A few years later, FDA analyst Heather Stone helped secure emergency approval for the drug to treat a young girl named Elena, who also survived. This case demonstrates how existing drugs can save lives in unexpected ways, yet only a quarter of the 18,000 known human diseases have FDA-approved treatments.
- Balamuthia is a brain-eating amoeba with a 90% fatality rate, transmitted through soil exposure
- Nitroxeline, a European UTI drug approved for 50 years, showed off-the-chart amoebicidal activity
- Two patients with balamuthia survived after treatment with nitroxeline, a remarkable outcome for such a deadly disease
- Only about a quarter of 18,000 known human diseases have FDA-approved treatments
" You might not think that a UTI drug could treat a brain-eating amoeba, but biochemistry can surprise you. "
" How many more things are there that we could potentially uncover to save lives today? Because these drugs are already at the pharmacy. They're already manufactured. They're already available. "
David Fagenbaum's Journey from Quarterback to Doctor
David Fagenbaum spent his childhood dreaming of becoming a Division I quarterback, achieving that goal at Georgetown. However, when his mother was diagnosed with brain cancer during his freshman year, his life's focus shifted entirely. Watching doctors try to save his mother's life—and ultimately fail—inspired him to pursue medicine. This personal tragedy would later prepare him for an even more harrowing challenge: saving his own life from a mysterious, deadly disease.
- Fagenbaum was laser-focused on becoming a college quarterback from age 8-9, covering his walls with performance metrics
- He broke his collarbone senior year, but his orthopedic surgeon father helped him return to play within five weeks
- His mother's brain cancer diagnosis during college immediately shattered his focus on football
- Seeing his mother's illness and recognizing that some diseases like brain cancer have no treatments led him to medicine
" It shattered my belief in what was fair and right in the world. It also immediately shattered my focus on football. The moment that I learned my mom had brain cancer and I started seeing what she was going through and started seeing these doctors who were trying to save her life, I just said, I've got to do this. "
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