UC Berkeley is embarking on a new approach to advance medicine and global health. The Center for Unmet, Rare, and Emerging Diseases (CURED) will unite researchers across campus to find cures that other organizations are not pursuing.
Pharmaceutical companies seek a return on their investments. Medical schools and research centers require nearby patients. But what happens if a disease is new, uncommon, or concentrated in developing countries?
Immunology and molecular medicine professor Andrew Dillin encountered several of these challenges when his lab identified a better way to treat Leigh’s disease. The rare mitochondrial disease causes seizures, developmental delays, heart problems, and breathing difficulties. The condition is fatal, and children who develop symptoms rarely survive into their teenage years.
After a group at Harvard found that a mitochondria-inhibiting antibiotic significantly improved a mouse model, Dillin’s lab discovered the mechanism behind the treatment and synthesized new molecules to fight the disease. Unfortunately, pharmaceutical companies have yet to express interest in utilizing the breakthrough.
“Because there are not many patients, the profit margins don't justify them spending millions of dollars to get it to the clinic,” said Dillin. “It's unfortunate and short-sighted, so we have to find a different route.”
Dillin formed CURED to bridge the gap between the foundational knowledge generated by UC Berkeley and the clinical applications that directly save lives. CURED will rely on novel technologies being developed at research centers across campus, such as the Innovative Genomics Institute, Molecular Therapeutics Initiative, and Stem Cell Center. Then, when a discovery is made, the CURED initiative will connect researchers with the Bay Area’s robust philanthropic, venture capital, and biotech communities.
“We are decentralizing the normal path to clinical breakthrough,” said Dillin. “The idea behind CURED is that we can take the discovery science that Berkeley is known for, translate that into medical discoveries, and be socially responsible while doing it. We are taking a new approach to health science.”
When researchers were developing the concept for CURED, they looked for areas where Berkeley could make the greatest impact. They decided to use immunology to target four areas of concern: cancer, rare diseases, autoimmune and inflammatory diseases, and infectious and emerging diseases of global concern.
The goal is not to develop singular technologies that can only aid patients who have one specific disease, but to reveal fundamental mechanisms that govern multiple diseases and disorders. For example, Dillin expects that treatments for some rare muscular diseases will have broader applicability to more common diseases like Alzheimer’s, ulcerative colitis, and Crohn’s Disease.
“We want to do something that's never been done before; that's why we’re so invigorated,” said Dillin. “Berkeley has already proven that we can make massively important medical discoveries. We already have two of the biggest biomedical discoveries of the last hundred years with CRISPR and immune checkpoint inhibition. With this track record, we think we’re going to be just as successful with CURED.”
Andrew Dillin