RARE Daily

New Non-Profit Seeks to Systematize Repurposing of Generic Drugs

September 20, 2022

As a medical student, David Fajgenbaum became a big proponent of repurposing drugs while facing death from the rare immune condition Castleman disease after he was able to identify an already approved drug for a different indication could be used to save his own life.

Now, with the backing of the Clinton Global Initiative, Fajgenbaum has co-founded Every Cure, a nonprofit, data-driven hub that seeks to accelerate connections between drugs and diseases they may be able to treat.

Ahead of a formal announcement expected this week, USA Today reported on the Every Cure effort saying it is seeking to raise $55 million to identify generic drugs that can help people with rare diseases and advance clinical trials for the most promising candidates.

“No one is responsible for ensuring that drugs are fully utilized for all diseases they can help,” Fajgenbaum told USA Today. “We’re taking on that responsibility.”

Repurposing existing drugs, the use of a drug approved for one indication to treat another, offers a fast way to identify drugs known to be safe that could provide benefit in other conditions. Unfortunately, it is often not in the interest of a drug company to make the effort to find additional conditions a drug may benefit, particular if the indication is rare and the drug is no longer protected by patents.

“Systemic barriers impede repurposing, so patients suffer while potential treatments are not fully utilized,” Every Cure said on its website. “We overcome these barriers to systematically identify and advance promising repurposing opportunities and save lives.”

Fajgenbaum was lying in a hospital bed dying when the drug that saved his life was available at a local pharmacy and had been approved for 50 years. The problem, the organization said, is that there have been no systemic efforts to unlock the full potential of approved drugs across diseases.

Though many diseases share common mechanisms and can benefit from the same drugs, the estimated 3,000 FDA-approved treatments are only approved for about 3,000 human diseases. Another roughly 9,000 diseases affecting millions of people do not have any approved treatments.

Today it takes about $1 billion to $2 billion and up to 15 years to develop a single, new FDA-approved drug. Repurposing safe, widely available drugs for new indications is faster and less expensive, with the greatest return on investment for saving lives.

In addition to the drug that saved Fajgenbaum’s life and many other people who have Castleman disease, Every Cure says its team has identified nine other CD treatments as well as treatments for cancer and COVID-19, including guiding the selection of drugs for the groundbreaking ACTIV-6 clinical trial. The group notes that dexamethasone and tocilizumab, which were rapidly repurposed as treatments for COVID-19, have likely saved the most lives during this pandemic.

Every Cure uses an artificial intelligence engine to identify the most promising drug repurposing opportunities. It then performs what it describes as “efficient” clinical trials in new indications

“Unfortunately, insufficient incentives, siloed data, misaligned stakeholders, and other market failures have impeded the identification of all potential uses for all drugs, especially low-cost, generic drugs,” the organization said. “The incomplete utilization of existing drugs and focus on new, expensive drugs has a disproportionately negative impact on individuals in areas with reduced access to medicines.”

Author: Rare Daily Staff

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