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

Dr. David Fajgenbaum, having nearly died five times from Castleman disease, dedicated his life to drug repurposing, discovering a treatment for his own condition and establishing the nonprofit Every Cure to find new uses for existing drugs to treat thousands of diseases.

• Existing FDA-approved drugs hold untapped potential for treating thousands of diseases, often at low cost.

• Systematic drug repurposing, driven by AI and collaborative research, is essential to bridge critical treatment gaps.

• Dr. Fajgenbaum's personal battle with Castleman disease exemplifies the power of agency and drug repurposing in saving lives.

Dr. David Fajgenbaum, a physician-scientist, recounts his harrowing personal journey with Castleman disease, a rare and deadly autoimmune disorder, which prompted him to pioneer a novel approach to medicine: drug repurposing. He advocates for leveraging the 4,000 existing FDA-approved drugs to treat the 14,000 diseases that currently lack treatments, highlighting a critical blind spot in the medical system where effective, often generic, treatments remain undiscovered due to lack of incentive or systematic search. His nonprofit, Every Cure, utilizes AI and rigorous research to systematically identify and validate new applications for these medicines, aiming to proactively disseminate life-saving information to patients and physicians.

The Repurposing Mission

• 00:00:00 Faced with a terminal diagnosis for Castleman disease, Dr. David Fajgenbaum realized that while seven chemotherapies meant for other cancers had temporarily saved his life, the medical system hadn't explored all 4,000 existing drugs. He committed his life to finding new uses for approved medications for his condition and others, believing no one should suffer if a drug that could help them already exists for another purpose.

The "Blind Spot" in Medicine

• 00:02:51 The medical field has a significant 'blind spot' in that many effective treatments and potential cures for seemingly untreatable diseases already exist as approved drugs for other conditions. This issue stems from how medications are studied, patented, and categorized, leading even well-trained physicians to be unaware of these alternative uses. Dr. Fajgenbaum's mission is to educate medical professionals and the public about these overlooked facts.

Successful Drug Repurposing Examples

• 00:06:45 Numerous drugs have unexpected applications beyond their primary indications; for instance, aspirin, known for pain relief, also reduces the risk of colon cancer recurrence and heart attacks. Viagra, initially for heart disease, now treats erectile dysfunction and a rare pediatric lung disease. Lidocaine, a numbing agent, has shown a 29% reduction in mortality for localized breast cancer patients when injected before surgery, yet its adoption is slow due to lack of financial incentive for generic drugs.

Challenges in Drug Development

• 00:14:17 The pharmaceutical industry is heavily incentivized to repurpose existing drugs for new applications to extend patent life, rather than developing entirely new molecules, especially for generic drugs. This system means that once a drug becomes generic, research and development for new uses often ceases, leaving thousands of diseases without potential treatments even when effective, inexpensive drugs are readily available.

Personal Journey to Discovery

• 01:05:32 After five near-death experiences and being told there were no more options, Dr. Fajgenbaum, a medical student at the time, analyzed his own blood and lymph node samples. His research revealed that the mTor pathway in his immune system was in overdrive, leading him to try an mTor inhibitor, sirolimus (rapamycin), a drug approved for organ transplant rejection. This decision, against conventional medical advice, led to his current 11-and-three-quarters-year remission from Castleman disease.

Leveraging AI and Collaboration

• 02:30:35 To overcome the systemic challenges and randomness in medical discovery, Every Cure, Dr. Fajgenbaum's nonprofit, uses artificial intelligence and machine learning to map the world's biomedical knowledge. This approach quantifies the likelihood of every drug treating every disease, prioritizing promising matches for rigorous laboratory studies and clinical trials, ensuring that effective, repurposed drugs reach patients proactively and at scale, preventing them from reaching death's doorstep.