Every Cure: Pioneering Drug Repurposing for Rare Diseases
Dr. David Fajgenbaum co-founded Every Cure, a nonprofit dedicated to drug repurposing, which seeks new applications for existing drugs.
There are more than 10,000 rare diseases, with 95% lacking cures. Developing new treatments can run into billions and take over a decade, mainly because many rare diseases affect only a handful of patients. This situation discourages pharmaceutical companies from making substantial investments.
Drug repurposing changes that dynamic by exploring innovative uses for existing drugs. Some organizations are even employing AI to enhance this process.
When Fajgenbaum and his team considered launching Every Cure, they faced a significant choice. “We could either wait for patients to approach us for help or leverage AI to identify potential drug-disease matches,” he recalled. They opted for the latter.
Every Cure doesn’t focus on finding specific treatments for particular diseases; instead, it broadly seeks existing drugs that may address various diseases. This marks a shift from traditional methods of developing rare disease treatments.
“Traditionally, funding would come from those seeking specific disease research,” Fajgenbaum explained. “But that’s not our approach.” Communicating this unique stance was challenging at times.
Despite being known for successfully identifying existing drugs that help rare conditions—he estimates saving over 1,000 lives in 12 years—initial fundraising proved difficult. “In our first year, we couldn’t secure any funds,” he admitted.
Philanthropy in rare disease research is often deeply personal; donors tend to support causes that resonate with their own experiences. Early offers to fund specific cancers, for instance, came in, but Fajgenbaum’s disease-agnostic approach meant turning away those funding debates. “We turned down plenty of money because we believed it was the right thing to do,” he noted. “I didn’t want to risk spending years and significant funding without finding results.”
Fortunately, Fajgenbaum found partners willing to support this broader mission, including the Chan Zuckerberg Initiative and other philanthropic organizations. Eventually, Every Cure secured a substantial $60 million commitment from TED’s Audacious Project, along with over $130 million from the Advanced Research Projects Agency for Health (ARPA-H).
Initial results have been promising. Since its launch in late 2022, Every Cure has identified 10 active drug repurposing programs.
Fajgenbaum stated, “In our first year, we focused on fundraising. The second year was about building our team. Now we’re concentrating on developing our pipeline.” He anticipates that most of these programs will benefit patients.
The Role of AI in Research
Fajgenbaum’s personal journey with Castleman disease—a serious rare condition—drove his interest in repurposing drugs. His previous work involved extensive research, analyzing blood samples, and even self-testing.
Every Cure, however, is leveraging AI to enhance efficiency. The team evaluates around 4,000 existing drugs against over 18,000 known diseases each month—a staggering 75 million potential matches. What once took 100 days now takes only about 17 hours.
From there, a medical team reviews the best options, conducting deeper analyses to pinpoint treatments worth pursuing. Every Cure commits to exploring treatments that are both effective against serious diseases and feasible financially for further development, as clinical trials can cost between $3 to $7 million per drug.
The nonprofit aims not just to find matches, but to navigate them through the entire process—from lab work to trials, and ultimately to patients.
To exemplify their work, Fajgenbaum cited progress on Bachmann-Bupp syndrome, a rare neurodevelopmental disorder. Collaborating with the original researchers, they discovered that a decades-old drug for African sleeping sickness could potentially inhibit a protein driving the condition. So far, six patients—including five children—have shown notable improvements.
Balancing Drug Repurposing and New Development
Fajgenbaum doesn’t view drug repurposing as a replacement for developing new treatments. He recognizes some diseases need entirely new medicines and desires collaboration with those pursuing novel therapies, believing both approaches should proceed together.
“Both pathways are essential,” he maintained. “New drugs are necessary, but so are evaluations of existing ones. Not every disease can rely solely on older medicines, but if there’s a chance for a cure with them, we must pursue it.”
Addressing criticisms that drug repurposing diverts funds from new research, he countered with facts. “Creating a new drug costs between $1 to $2 billion and takes 10 to 15 years,” he stated. “Repurposing is typically far less expensive.”
However, challenges arise when dealing with older drugs that might still be patented but lack financial incentives for production. Fajgenbaum explained, “Some companies hesitate to manufacture if it’s not profitable. Currently, we’re addressing this issue with one of our programs.”
He argued that while the medical system thrives on newer drugs, it tends to overlook the potential of older medications. “Once a drug becomes generic, it’s often unprofitable to explore its new uses,” he remarked.
Yet, Fajgenbaum believes Every Cure can bridge this gap. In the realm of rare diseases, where urgent action is crucial, having a parallel route for repurposing can prevent unnecessary loss of life.
Navigating the regulatory landscape isn’t straightforward, though. The FDA’s approval process remains anchored to traditional models. “We’re seen as atypical by regulators because we don’t fit into familiar sponsorship roles,” Fajgenbaum said, underscoring the challenges involved.
This situation has made educating healthcare providers a critical mission for Every Cure. While FDA approval isn’t always mandatory for a repurposed drug, it still offers advantages in terms of insurance and confidence for doctors and patients alike.
Despite various obstacles, Every Cure forges ahead, and Fajgenbaum believes in the importance of their mission. “Our focus is on repurposing drugs to save lives. That’s our sole purpose—to enhance and save lives,” he remarked. For families of those with rare diseases, who often feel sidelined, Every Cure symbolizes hope and determination. They are building a system that seeks solutions no matter how challenging the path may be.
