New hope for 16-month-old battling rare, fatal genetic disorder with gene therapy
Alissa Feldborg is 16 months old and undergoing rare therapy for Sandhoff disease, a very rare, fatal genetic disorder.
Robert Deutsch, USA Today
Every drug on the market has multiple effects on the body, not just the one or two that won it approval.
A new nonprofit aims to exploit those other effects to help patients with rare diseases.
“No one is responsible for ensuring that drugs are fully utilized for all diseases they can help,” said Every Cure co-founder Dr. David Fajgenbaum. “We’re taking on that responsibility.”
Every Cure aims to raise $55 million to coordinate drug data, identify generics that might offer hope for patients with a rare disease and bring the most promising drugs through clinical trials.
The organization’s launch 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 to deadly diseases,” former president Bill Clinton said in a statement about Every Cure. “We just have connect the dots between the research and the drugs available.”
Drug repurposing has happened many times before.
The drug thalidomide caused terrible birth defects when it was used to relieve nausea during pregnancy in the 1960s. But more recently it has been shown to be a safe, effective treatment for the inflammation of leprosy as well as the blood cancer multiple myeloma.
Tocilizumab , first approved in 2010 to treat rheumatoid arthritis, was found to be useful against severe COVID-19. And the erectile dysfunction drug sildenafil citrate (Viagra), originally developed to reduce high blood pressure, also can benefit children with a rare form of lung disease.
Every Cure isn’t the first to focus on identifying other purposes for existing drugs. But Fajgenbaum and Dr. Grant Mitchell, Every Cure co-founders, want to make the effort less accidental and more systematic.
Right now, there are several major barriers to repurposing the 3,000 already-approved drugs, 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 the body’s vital organs potentially leading to life-threatening organ failure. He tried the drug after connecting insights from his own samples with information from medical literature, though it hadn’t been studied in his disease.
Drug companies don’t often conduct such studies, Fajgenbaum said, because they don’t have much financial incentive to take on the risk that comes with exploring a new condition. Data on a drug’s activity, even if it’s publicly available, isn’t in a central, readily accessible database.
And once a drug has reached generic status, which roughly 90% of approved drugs have, no one is responsible for uncovering additional potential uses or stands to make substantial money by adding to its uses, Fajgenbaum said.
Every Cure already has begun developing an algorithm to help identify possible other uses for generic drugs. In an initial pilot, it identified 106 drugs that might treat 147 of the 9,000 rare diseases.
“It’s not going to be a problem to find promising links” between drugs and rare diseases, Fajgenbaum said. “The problem is, which ones do you take into clinical trials? And how do you cover the costs to ensure that patients can benefit from all of the drugs sitting on the pharmacy shelf?”
Clinical trials can be extremely expensive even at the small scale needed to show effectiveness. So Every Cure is looking for partners such as generic manufacturers to help reduce the cost and speed up the process.
The Clinton Global Initiative plans to help bring companies, patient groups and philanthropists together to pursue drug repurposing.
“This is exactly the kind of effort that CGI was created to support,” Clinton said in his statement. “The Every Cure team has already lined up an incredible list of partners, and my hope is that through CGI we can connect them with the right groups and resources to have the biggest possible impact.”
Every Cure is working to raise $10 million to develop its open-access algorithm to identify and rank repurposing opportunities and $45 million for clinical trials.
Fajgenbaum said he expects insurance will pay for patients to get the generic drugs once Every Cure can show they work. “As long as there is some data to support it, they will cover it,” he said.
Other drug repurposing efforts exist but tend to focus on specific parts of the process. Every Cure starts without a specific disease in mind, he and Mitchell said, and aims to link all available databases of drug information, identify promising repurposing opportunities, perform clinical trials that definitively demonstrate effectiveness, and work with disease organizations to ensure they’re used in the appropriate patients.
Barbara Goodman, president and CEO of Cures Within Reach, has helped fundraise for clinical trials of repurposed drugs for 15 years. But her organization doesn’t use computer learning to identify promising prospects. She hopes to use Every Cure’s algorithm to test the drugs Cures Within Reach considers promising.
She sees the Fajgenbaum’s work, which Cures Within Reach just recognized with an award, as complementary to her efforts, not competition.
“We’re thrilled that more people are interested in this conversation. That is going to draw attention to our work,” she said, noting the two groups plan to collaborate. “We’re ready and excited to help Ever Cure’s initiative, because we expect to be part of that.”
Fajgenbaum and Mitchell, who were medical school roommates at the University of Pennsylvania, where Fajgenbaum still works as an assistant professor, described Every Cure as the convergence of everything they’ve both worked on since those early days.
“I’ve been dreaming about being able to expand the approach that saved my life to more people,” said Fajgenbaum, who has since married and had two children.
Mitchell, president of Every Cure, 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 the technology could do,” he said. “Once it finds one drug-disease linkage, that bolsters its ability to find another. If you don’t scrap it after one drug it gains in power.”
The two, who also attended Penn’s Wharton School of business together, won an award for developing a business plan for drug repurposing. But they could never figure out how to turn their dream into a company.
More recently they realized their concept would work better as a non-profit, 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 someone else losing,” Fajgenbaum added. “It’s about changing the way the system works to incentivize this sort of innovation.”
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