Using Existing Drugs in New Ways to Treat & Cure Diseases of Brain & Body | Dr. David Fajgenbaum

David Fajgenbaum describes the moment when his doctors said that every available option had been exhausted. Seven chemotherapies had repeatedly saved his life, but the relapses made clear that this was not a durable solution. He concluded that the relevant question was not whether a treatment had been designed for his disease, but whether any of the thousands of existing drugs might affect a useful cell type or signaling pathway. That experience became a commitment to finding new uses for approved medicines rather than accepting the boundaries implied by their original labels.

The conversation frames drug repurposing as both a scientific and an information problem. Fajgenbaum says that many approved drugs affect dozens of proteins and pathways even though regulatory approval usually covers only one or two uses. Examples include aspirin's possible role in reducing colon-cancer recurrence for some patients with mTOR-pathway mutations, sildenafil's later use in a rare pediatric lung disease, and the unexpected range of effects associated with small molecules. The point is not that people should self-medicate, but that patients and clinicians need better ways to identify credible possibilities and then evaluate them.

Lidocaine illustrates the gap between a promising result and adoption. Fajgenbaum cites an Indian trial of 1,600 patients in which injecting lidocaine around a localized breast tumor eight to ten minutes before surgery was associated with a 29 percent reduction in five-year mortality. Lidocaine is inexpensive, already used during surgery, and the study appeared in the Journal of Clinical Oncology, yet the practice has seen little worldwide uptake. He argues that a neutral organization must identify such opportunities, examine the mechanism and the full evidence, and do the implementation work that generic-drug economics do not reward. The episode presents this as an institutional failure of translation, not as evidence that every preliminary result is ready for routine care.

Fajgenbaum argues that promising medical findings need an organization willing to carry them from evidence to patients. The problem is not only discovery: once a result seems credible, someone must examine the mechanism, test the evidence and push for implementation. The episode's sponsor messages sit alongside this discussion but are separate from the medical argument.