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

Key Takeaways

Many existing FDA-approved drugs have potential to treat other diseases.
Drug repurposing faces systemic and commercial challenges within the pharmaceutical industry.
Patient self-advocacy and expert networks are crucial for finding novel treatment options.
AI and integrated medical databases are critical tools for accelerating drug repurposing efforts.
Dr. Fajgenbaum's personal battle with Castleman disease led to a life-saving repurposed drug.
The nonprofit Every Cure systematically identifies new uses for existing drugs.
Resilience, social support, and a 'hope, action, impact' mindset are vital in overcoming severe illness.
Certain compounds, like lithium and GLP-1 agonists, show promise for preventive health.

Patients are increasingly recognizing the need for self-advocacy in their healthcare journeys since 2020.
Many effective treatments and potential cures exist among the 4,000 FDA-approved drugs, a fact not widely known by medical professionals.
The current medical system is not optimized to discover new applications for existing medications.
Aspirin is cited as an example, used for pain, heart attack prevention, and reducing colon cancer recurrence.

A person's knowledge network significantly influences their health outcomes in navigating treatment options.
There is a recognized need for integrated medical databases and AI platforms to map drug-disease connections.
The nonprofit Every Cure utilizes biomedical knowledge graphs and AI to quantify drug-disease likelihoods.
Every Cure currently has 9 active programs aimed at bringing potential repurposed treatments to patients.

Every Cure identifies promising new uses for existing medications by analyzing vast datasets.
Thalidomide, initially causing birth defects, later received FDA approval for leprosy and multiple myeloma due to anti-angiogenic properties.
Colchicine, an anti-inflammatory medication for gout, received FDA approval for reducing heart attack risk in a specific subpopulation.
Pembrolizumab, originally for melanoma, is now used for dozens of cancers, leading to nine-year remission for one patient.

The guest's life direction shifted at 18 when his mother was diagnosed with glioblastoma.
His mother's fatal battle with brain cancer inspired him to dedicate his life to finding cancer treatments.
She demonstrated resilience during a complex surgery, using humor despite the severe procedure.

Upon receiving his Castleman disease diagnosis, no approved treatments were available at the time.
Chemotherapy proved life-saving, but a drug discovered in Japan for Castleman disease was not known to his doctors.
Physicians often lack the time to stay current with all medical literature, leading to fragmented information access.
The 'Santa Claus theory of civilization' highlights a gap between the ideal and actual pace of collaborative scientific progress.

After a near-fatal relapse of Castleman disease in medical school, the guest faced organ failure.
He conducted self-research, identifying an overactive mTOR pathway in his immune system despite lacking laboratory skills.
He proposed using Rapamycin (Cerolimus), an mTOR inhibitor approved for organ transplant rejection, to treat his condition.
This repurposed treatment has kept him in remission for over 11 years after nearly dying five times.

The guest's athletic background, specifically playing football at Georgetown, taught him resilience through losses and physical challenges.
During a six-month ICU stay, his recovery was aided by a clear vision for the future, strength from family, and athletic fortitude.
His sister's encouragement to 'just breathe' helped him endure severe pain during a near-death experience with Castleman disease.
He learned to focus on short intervals of time to survive, a mindset partly attributed to his football experience.

The guest's lab initially focused on Castleman disease, successfully treating patients like Joey with rapamycin and Kyla with Ruxolitinib.
Over 11 years, 14 drugs have been found for diseases they were not originally intended for.
Every Cure was established three years ago to utilize artificial intelligence to scan all drugs and diseases for optimal treatment matches.
A TNF inhibitor (adalimumab), identified by machine learning, successfully treated a patient named Al with severe Castleman disease for two years.

A Nobel Prize-winning neuroscientist reportedly uses nicotine (via Nicorette) for potential protective effects against Alzheimer's and Parkinson's.
Intermittent lithium use and GLP-1 agonists are discussed, with GLP-1s potentially reducing risks of Parkinson's, Alzheimer's, and breast cancer.
Rapamycin (Cerolimus), originally found in Rapa Nui soil, was developed into a drug approved for organ transplant rejection.
Bioprospecting involves finding natural compounds for drug development, but unregulated over-the-counter products carry risks.

The guest outlines a 'hope, action, and impact circuit' in healthcare, where hope drives subsequent action.
This circuit, also linked to neuroscience research on the anterior mid-cingulate cortex, appears connected to tenacity and a strong will to live.
AI's role in drug discovery involves connecting existing research to identify patterns and prioritize potential treatments.
The guest describes himself as a rigorous parent who asks many questions and stays informed about his children's health.