Every Cure seeks new uses for generic drugs to treat rare diseases

Every drug on the market has multiple effects on the body, not just the one or two that got it approved.

A new nonprofit aims to harness these other effects to help patients with rare diseases.

“No one is responsible for ensuring that drugs are fully utilized for all the illnesses they can help,” said Every remedy co-founder Dr. David Fajgenbaum. “We take that responsibility.”

Every Cure aims to raise $55 million to coordinate drug data, identify generics that could offer hope to patients with a rare disease, and move the most promising drugs through clinical trials.

The launch of the organization will be officially announced this week by the Clinton Global Initiative, which brings together leaders from around the world to address pressing challenges.

“We already have the tools we need to find new cures for deadly diseases,” former President Bill Clinton said in a statement on Every Cure. “We just have to make the connection between the research and the drugs available.”

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Drug redirection has happened many times before.

The drug thalidomide caused terrible birth defects when used to relieve nausea during pregnancy in the 1960s. But more recently it has been shown to be a safe and effective treatment for the inflammation of leprosy as well than for blood cancer multiple myeloma.

Tocilizumab, first approved in 2010 to treat rheumatoid arthritis, has been shown to be useful against severe COVID-19. And sildenafil citrate (Viagra), an erectile dysfunction drug originally developed to reduce high blood pressure, may also benefit children with a rare form of lung disease.

Every Cure isn’t the first to focus on identifying other targets for existing drugs. But Fajgenbaum and Dr. Grant Mitchell, Every remedy co-founders, want to make the effort less accidental and more systematic.

Right now, there are several major hurdles to reusing the 3,000 drugs already approved, said Fajgenbaum, who, with Mitchell’s help, repurposed the kidney transplant drug sirolimus eight years ago to save his own life.

Fajgenbaum has a form of Castleman’s disease, in which the immune system attacks vital body organs, potentially leading to life-threatening organ failure. He tried the drug after relating information from his own samples to information from the medical literature, although it was not studied in his disease.

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Pharmaceutical companies don’t often conduct such studies, Fajgenbaum said, because they don’t have much financial incentive to take on the risk of exploring a new condition. Drug potency data, while publicly available, is not located in a central, easily accessible database.

And once a drug has achieved generic status, which about 90% of approved drugs have, no one is responsible for discovering additional potential uses or making substantial money by adding to its uses. said Fajgenbaum.

Every Cure has already started developing an algorithm to help identify other possible uses for generic drugs. In a first pilot project, he identified 106 drugs capable of treating 147 of the 9,000 rare diseases.

“It won’t be a problem to find promising links” between drugs and rare diseases, Fajgenbaum said. “The problem is which ones are you taking in clinical trials? And how do you cover the costs to make sure that patients can benefit from all the drugs that are on the pharmacy shelves?”

Clinical trials can be extremely expensive, even on a small scale, to demonstrate their effectiveness. So Every Cure is looking for partners such as generic manufacturers to help reduce costs and speed up the process.

The Clinton Global Initiative plans to help bring together corporations, patient groups and philanthropists to pursue drug redirection.

Former President Bill Clinton speaks at a Clinton Global Initiative America annual rally in Denver.

“This is exactly the kind of effort that CGI was created to support,” Clinton said in her statement. “The Every Cure team has already lined up an incredible list of partners, and I hope that through CGI we can connect them with the right groups and resources to have the biggest impact possible.

Every Cure is working to raise $10 million to develop its open-source algorithm to identify and rank referral opportunities and $45 million for clinical trials.

Fajgenbaum said he expects insurance to pay patients to get the generic drugs once Every Cure can show they work. “As long as there’s data to back it up, they’ll cover it,” he said.

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Other drug redirection efforts exist but tend to focus on specific parts of the process. Each treatment begins without a specific disease in mind, he and Mitchell said, and aims to link all available databases of drug information, identify promising opportunities for reuse, perform clinical trials that demonstrate definitely efficiency and to work with disease organizations to make sure they are used. in appropriate patients.

Barbara Goodman, president and CEO of Cures Within Reach, has helped raise funds for clinical trials of repurposed drugs for 15 years. But his organization does not use computer learning to identify promising prospects. She hopes to use Every Cure’s algorithm to test drugs that Cures Within Reach sees promise.

She sees the work of Fajgenbaum, which Cures Within Reach just awarded with a prizeas a complement to its efforts, not competition.

“We’re excited that more people are interested in this conversation. It’s going to bring attention to our work,” she said, noting that the two groups plan to collaborate. “We are ready and excited to help the Ever Cure initiative, as we hope to be part of it.”

Fajgenbaum and Mitchell, who were roommates at the University of Pennsylvania Medical School where Fajgenbaum still works as an assistant professor, described Every Cure as the convergence of everything they’ve worked on since those early days.

Drs.  David Fajgenbaum and Grant Mitchell (middle and right), co-founders of Every Cure, with colleague Tracey Sikora.

“I dreamed of being able to extend the approach that saved my life to more people,” said Fajgenbaum, who has since married and had two children.

Mitchell, president of Every Cure, has worked as a consultant, using machine learning to identify new uses for existing drugs. But he analyzed the data for one specific drug at a time and for the benefit of a company, not society. “I saw what technology could do,” he said. “Once it finds a drug-disease link, it boosts its ability to find another one. If you don’t remove it after a drug, it gains strength.”

The two, who also attended Penn’s Wharton School of Business together, won an award for developing a drug reuse business plan. But they never knew how to turn their dream into a business.

More recently, they realized their concept would work better as a nonprofit, targeting drugs and diseases that companies aren’t pursuing, filling a major gap in the system. “We’re expanding the use of something that already exists,” Mitchell said. “It should be cheap and accessible.”

“It’s not about losing someone else,” Fajgenbaum added. “It’s about changing the way the system works to encourage this kind of innovation.”

Contact Weintraub at [email protected]

Coverage of patient health and safety at USA TODAY is made possible in part by a grant from the Masimo Foundation for Ethics, Innovation and Competition in Healthcare. The Masimo Foundation does not provide editorial input.

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