Leveraging AI to repurpose generic drugs and save patients’ lives

Problem

Of the world’s approximately 18,500 recognized diseases, only about 4,000 have FDA-approved treatments. More than 300 million people globally suffer from diseases without treatment, while many, many more struggle with undertreated diseases where existing cures are expensive, burdensome or inaccessible. Developing new drugs is an extremely costly process, requiring between $1-2 billion and 10-15 years to bring to fruition, and facing a 90% failure rate. To maximize profit, drug companies focus on developing new drugs for the largest and wealthiest markets, while underinvesting in diseases that disproportionately affect marginalized groups. Over 80% of FDA-approved drugs are generic or off-patent and could hold the key to new treatments — but drug companies lack the financial incentives to study them further. These drugs sit on pharmacy shelves, vastly underutilized.

Big Idea

Drug repurposing can advance life-saving treatments at a fraction of the cost and time of novel drug discovery. Many diseases share common mechanisms in the body, and the average drug binds to more than 30 proteins, each of which may play a role in treating different diseases. In the past, drug repurposing has relied on serendipity and been focused on specific diseases, but Every Cure works to flip this process and look for shared connections between all 18,500 existing diseases and all 4,000 approved drugs simultaneously. Shared connections aren’t always obvious, but advanced AI allows Every Cure to maximize the chance of finding effective drugs, searching the forest for low-hanging fruit instead of one tree at a time. With this approach, Every Cure will unlock generic repurposed treatments for 15-25 diseases by 2030, alleviating suffering for millions of people worldwide and ensuring that every drug it advances helps the most people possible.

Plan

Every Cure’s AI approach enables the systematic analysis of the world’s biomedical knowledge. Their platform will look at all 74 million potential drug-disease matches and quantify the strength of each. With this as a starting point, Every Cure’s team will work with medical experts and patient advocacy groups to advance the matches that have the greatest chances for success and the potential to benefit overlooked populations. From there, Every Cure will generate the evidence through clinical trials, lab studies and more to change clinical practice guidelines, which physicians consult for treatment recommendations. In some cases, they’ll pursue official FDA label-change, and in others they’ll use off-label prescribing — an effective strategy, as physicians can prescribe any drug for any disease (today, 32% of prescriptions are off-label). When Every Cure succeeds in adding a repurposed drug to clinical guidelines, they will work with affected populations and physicians to reach patients everywhere. 

Why will it Succeed?

Every Cure was founded to ensure that patients don’t suffer while treatments hide in plain sight. Its founder, Dr. David Fajgenbaum, saved his own life with a drug he repurposed — and the organization has built an impressive track record ever since. Over 15 years, their team and partners have helped to advance 28 repurposed drugs — 14 directly led by Dr. Fajgenbaum. Their AI platform has already confirmed two repurposing opportunities, including the use of the autoimmune disorder treatment adalimumab as a medication for Castleman disease. Every Cure has partnerships across AI, medicine, the pharmaceutical industry and the regulatory field, and has successfully spurred others to pursue drug repurposing too. For example, Every Cure supported the DADA2 Foundation in pursuing repurposed treatment for DADA2, a rare pediatric disease. Today, this class of treatments — TNF inhibitors — are used to treat patients with DADA2 around the world and are covered by insurance in nearly all cases.