Dr. David Fajgenbaum - Doctor Finds a Cure for His Own Castleman’s Disease | SRS #240

Dr. David Fajgenbaum, who nearly died five times from Castleman disease, discovered his own cure by repurposing an existing drug and now leads a nonprofit, Every Cure, using AI to find new uses for FDA-approved medications to treat untreatable conditions.

• Dr. David Fajgenbaum found a cure for his own Castleman disease by repurposing an existing drug, sirolimus.

• The nonprofit Every Cure uses AI to identify new uses for 4,000 existing drugs to treat 18,000 diseases, including many currently untreatable.

• Repurposing generic drugs faces profitability challenges, necessitating philanthropic and governmental support to unlock 'hidden cures' for patients worldwide.

Dr. David Fajgenbaum, a physician-scientist, overcame idiopathic multicentric Castleman disease by identifying a hyperactive immune pathway and repurposing an FDA-approved immunosuppressant, sirolimus. His personal battle inspired him to found Every Cure, a nonprofit that leverages artificial intelligence to systematically discover and advance new uses for existing generic drugs to address 14,000 diseases that currently lack treatment options, aiming to save lives and improve patient outcomes globally.

Dr. Fajgenbaum's Background

• 00:08:31 David Fajgenbaum grew up in Raleigh, North Carolina, and pursued his dream of playing Division I college football at Georgetown. His life changed when his mother was diagnosed with an aggressive brain cancer shortly after he started college. Witnessing her 15-month battle and ultimate passing instilled in him a promise to dedicate his life to finding treatments for patients, leading him to pursue a career in medicine.

Battle with Castleman Disease

• 00:17:15 During his third year of medical school, David experienced extreme fatigue, swollen lymph nodes, and organ failure, leading to five near-death experiences. He was diagnosed with idiopathic multicentric Castleman disease, a rare and deadly disorder where the immune system attacks vital organs. Traditional chemotherapy provided temporary relief, but he continued to relapse, eventually being told there were no more treatment options available.

Discovery of His Cure

• 00:35:05 Facing his fifth deadly relapse, David decided to relentlessly search for a cure himself, realizing the existing chemotherapies used to save his life were repurposed from lymphoma treatments. He analyzed his own blood samples and identified an overactive immune communication pathway (mTOR). This led him to a hypothesis: the existing drug sirolimus, an immunosuppressant used in organ transplantation, could turn off this pathway. After a doctor agreed to prescribe it, he achieved full remission, which has lasted over 11 and a half years.

Repurposing Drugs for Patients

• 00:39:26 Inspired by his own survival, Dr. Fajgenbaum became obsessed with the idea of 'hidden cures' in existing drugs. He established a center at the University of Pennsylvania to study immune systems and drug repurposing, where they began treating other Castleman disease patients with sirolimus. This drug has been effective for about 25% of patients, enabling individuals like Joey, a young boy with Castleman disease, to recover and lead full lives, highlighting the profound impact of repurposed medicines.

Every Cure and AI Innovation

• 00:51:52 Recognizing the systemic challenge that generic drugs lack profit incentives for new research, Dr. Fajgenbaum co-founded Every Cure, a nonprofit dedicated to saving and improving lives by repurposing existing medications. The organization uses a biomedical knowledge graph and artificial intelligence to analyze 4,000 FDA-approved drugs against 18,000 diseases, including 14,000 without approved treatments, to identify the most promising matches for further research and clinical trials.

Challenges and Future of Repurposing

• 00:53:51 The biggest roadblock to drug repurposing is the lack of profitability for generic drugs, as pharmaceutical companies focus on patent-protected medicines. Every Cure aims to overcome this by securing federal contracts and philanthropic donations to fund research into generic drugs. They advocate for a system where effective repurposing discoveries are disseminated globally, potentially through comprehensive databases accessible to all physicians, to ensure all drugs are utilized for every condition they can treat.