How A Doctor Cured His Own Terminal Disease | Dr. David Fajgenbaum

Andrew HubermanAndrew Huberman
Science & Technology3 min read119 min video
Nov 3, 2025|76,823 views|2,052|217
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Key Moments

TL;DR

Doctor turned patient finds cures for diseases using repurposed drugs.

Key Insights

1

Many FDA-approved drugs can treat diseases other than their intended purpose, but the system lacks incentives to discover and utilize these off-label uses.

2

Dr. Fajgenbaum survived a terminal illness by discovering a treatment using a repurposed drug, highlighting the potential of existing medications.

3

Information asymmetry and the patent system create barriers, leaving patients and even doctors unaware of potentially life-saving treatments.

4

Patient advocacy, connecting with disease-specific groups, and identifying world experts are crucial for navigating complex health challenges.

5

Artificial intelligence can play a significant role in identifying potential drug-disease matches by analyzing vast amounts of biomedical knowledge.

6

The journey from scientific discovery to patient treatment requires rigorous validation, clinical trials, and effective communication to overcome systemic hurdles.

THE POTENTIAL OF REPURPOSED MEDICATIONS

Dr. David Fajgenbaum, a physician diagnosed with a terminal illness, discovered that many of the 4,000 FDA-approved drugs have the potential to treat a multitude of diseases beyond their original indications. While drugs are typically approved for one or two pathways, they can affect many others. However, the current system, driven by patents and profit motives, offers little incentive to explore these additional uses, particularly for generic medications.

A DOCTOR'S PERSONAL JOURNEY TO DISCOVERY

Fajgenbaum's own life-threatening experience with Castleman's disease, a rare autoimmune disorder, propelled him to investigate alternative treatments. When conventional options were exhausted, he found a life-saving drug by exploring off-label uses of existing medications, experiencing 'overtime' in his life. This personal victory ignited his mission to systematically uncover and validate these hidden therapeutic potentials.

SYSTEMIC BARRIERS TO DRUG REPURPOSING

A significant challenge lies in the economic model of drug development. Pharmaceutical companies are incentivized to patent new uses for existing drugs to extend market exclusivity, rather than finding new applications for generic drugs where profit margins are minimal. This leads to a 'giant blind spot' in medicine, where effective treatments may exist but remain inaccessible due to lack of funding for research and physician awareness.

EMPOWERING PATIENTS AND ADVOCATING FOR CHANGE

Fajgenbaum emphasizes the importance of patient agency and self-advocacy. He advises patients to connect with disease-specific advocacy groups, identify leading experts, and diligently ask questions about potential treatments. He highlights examples like aspirin's use in cancer prevention and Viagra's repurposing for pediatric lung disease, illustrating how existing drugs can offer profound benefits if their full potential is explored.

EVERY CURE: A SYSTEMATIC APPROACH TO DISCOVERY

To address these systemic issues, Fajgenbaum co-founded 'Every Cure,' a non-profit organization that systematically scans global biomedical knowledge, utilizing AI and machine learning to identify potential drug-disease matches. The goal is to validate these opportunities through rigorous research and clinical trials, ensuring that people benefit from drugs already approved for other conditions, thereby filling the gap where no treatments currently exist.

SCIENTIFIC RIGOR AND ACCELERATING ACCESS

The organization prioritizes finding drugs with strong pre-clinical or early clinical evidence and moves them through systematic validation processes. By connecting disparate scientific findings, Every Cure aims to break down silos and accelerate the translation of promising discoveries, like lidocaine's potential use in breast cancer or DFMO for rare pediatric syndromes, into standard clinical practice, ultimately making life-saving treatments widely available.

Common Questions

Physicians typically stay updated through random, piecemeal methods such as encountering relevant papers for specific patients or attending conferences. There isn't a systematic way for them to review millions of papers or hundreds relevant to their practice, especially for generic drugs where there's no incentive for pharmaceutical companies to promote new uses.

Topics

Mentioned in this video

personJoe Parvizi

A neurosurgeon at Stanford who discovered the anterior midcingulate cortex's role in tenacity and the will to live through stimulating awake patients' brains.

drugColchicine

An ancient drug primarily used for gout, now repurposed and FDA approved for reducing heart attack risk in individuals with prior heart attacks, especially those with diabetes, due to its anti-inflammatory properties.

organizationEvery Cure

A nonprofit organization founded by Dr. David Fajgenbaum that scans global biomedical knowledge using AI and machine learning to find new uses for existing drugs to treat rare and common diseases.

drugGLP-1s

A class of pharmaceutical products being explored for potential preventative concepts, with emerging evidence for improvement in Parkinson's symptoms, and reduction in the risk of Alzheimer's and breast cancer.

drugTafasitamab

A drug originally developed in Japan for Castleman's disease, but later repurposed and approved in the US for rheumatoid arthritis and other autoimmune diseases.

personDavid Fajgenbaum

Professor of Translational Medicine and Human Genetics at the University of Pennsylvania, guest on the podcast, and author of 'Chasing My Cure'. He cured his own terminal Castleman's disease through drug repurposing.

organizationJournal of Clinical Oncology

A great journal that published the major study on lidocaine and breast cancer, yet the findings have seen limited uptake.

organizationMichael J. Fox Foundation

A disease organization dedicated to Parkinson's disease research and support.

drugAdalimumab

A TNF inhibitor successfully used to treat a Castleman's patient named Al in Vancouver, leading to a two-year remission and publication in the New England Journal of Medicine.

productDavid Protein Bars

Protein bars with 28g of protein, 150 calories, and 0g sugar, designed to help meet protein goals efficiently.

bookChasing My Cure

Dr. David Fajgenbaum's book detailing his personal journey with Castleman's disease and his quest to find a cure.

organizationEnvironmental Working Group
supplementLidocaine
supplementAspirin

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