AI Unlocks Ancient Cures for Forgotten Diseases

• AI-powered drug repurposing offers a faster, cheaper alternative to traditional drug development for rare diseases.
• Every Cure, a non-profit, is pioneering a disease-agnostic approach to drug repurposing, focusing on existing drugs with potential for new uses.
• The organization has already identified promising treatments for ultra-rare conditions, demonstrating the potential of AI in medical research.
• Challenges remain in securing funding, navigating regulatory hurdles, and ensuring the continued manufacture of repurposed drugs.

A New Battleground for Medicine

I have seen gods fall and titans crumble. Yet, a battle against disease? This is a different kind of war. The realms are filled with over 10,000 rare diseases, most without a cure. To forge a new treatment, they say it costs billions and takes longer than a Spartan's training. But what if the weapon already exists? What if the gods have already gifted us the cure, hidden in plain sight? This "drug repurposing," as they call it, seeks new uses for old remedies. And now, they wield AI – a weapon of the mind – to make this process more efficient. Hmph, efficiency. The gods could have learned a thing or two about that.

Choosing the Battlefield

This Dr. David Fajgenbaum and his league of heroes at Every Cure had a choice, a fork in the road like the one I faced in the Labyrinth. Do they answer every cry for help, every disease group begging for a miracle? Or do they use this AI to find the 'lowest-hanging fruit,' the easiest victories across all drugs and diseases? They chose the latter. A wise choice. One must be strategic, even in compassion. Every Cure seeks drug-disease matches, connecting patients with potential cures. This is a departure from the old ways, where coin dictated the path of research. Now, knowledge and efficiency lead the charge. If you seek to explore this realm further, consider reading Nvidia's AI Gamble Investors Question the Sustainability of the Boom to understand the tools being brought to bear here.

The Price of Independence

Fajgenbaum, despite his success in finding treatments, faced a familiar foe: funding. Philanthropy, they say, is often personal. Donors want to fight the battles they know, the diseases that have touched their own families. Every Cure's approach is 'disease agnostic,' meaning they do not discriminate. They seek any cure, for any disease. This noble path meant turning away coin offered with conditions. Many desired research for specific ailments, such as that of the pancreas, but Every Cure had to refuse. "We turned down a lot of money. But we felt that it was the right thing to do," Fajgenbaum said. A Spartan makes hard choices. So must those who seek to heal.

Allies in the Fight

Fortunately, some saw the wisdom in Every Cure's path. The Chan Zuckerberg Initiative, the Lydia Hill Foundation, and others joined the cause. Eventually, they secured a significant commitment from TED's Audacious Project and funding from ARPA-H. The results, they say, are promising. Since its founding, Every Cure has identified 10 active programs in its drug repurposing portfolio. They aim to treat 15 to 25 diseases with repurposed drugs by 2030. Before this, their leadership had already repurposed 14 drugs for five diseases. A respectable start. Perhaps these mortals are not as foolish as I thought.

The Power of the Machine

Fajgenbaum himself found a drug to treat his own rare condition, Castleman disease, through relentless study and self-experimentation. But Every Cure now wields AI to accelerate the process. Their technology scores thousands of drugs against thousands of diseases – a task that once took months, now completed in hours. A medical team then reviews the leads, seeking the most promising treatments. They pursue treatments that are both effective and financially feasible. Their goal is not mere discovery, but delivery – lab work, trials, regulatory discussions, and ultimately, getting the treatments to those who need them. An end-to-end solution, they claim. Unlike the gods, they seek not just to create, but to nurture.

A Second Path Forward

Fajgenbaum does not see drug repurposing as a replacement for new drug development. Both, he says, are necessary. But repurposing offers a second chance, a path forward when time is short and commercial incentives are lacking. This presents its own challenges. Sometimes, the drug companies no longer wish to manufacture older drugs, even if they could save lives. And the regulatory system, built around a traditional sponsor model, struggles to accommodate an independent non-profit like Every Cure. Still, they persist. They educate doctors, navigate the FDA, and fight for every patient. "You want to repurpose drugs to save lives, which is all we care about," Fajgenbaum said. A simple goal, but one worth fighting for. For in the face of death, even a god can find purpose.