A local medical researcher has won a $2.1 million federal grant to explore treatments for rare diseases.
The National Institutes of Health awarded the grant to Michigan State University College of Human Medicine researcher Bin Chen to search vast databases of existing drugs that could be effective in treating “orphan diseases” — an estimated 6,000 diseases considered too rare to attract much research, according to MSU this month.
An orphan disease is generally defined as one that affects fewer than 200,000 patients.
“Although individually those diseases afflict relatively few people, combined they are suffered by about 25 million Americans, some whose illnesses are life-threatening,” said Chen, an assistant professor in the College of Human Medicine’s departments of pediatrics and human development, as well as pharmacology and toxicology.
Chen’s study will look at some 12,000 compounds that have already been developed and could be repurposed for treating under-studied diseases. Using a high-tech approach called next-generation sequencing, a team of researchers led by Chen plans to match the molecular and genomic characteristics of rare diseases against databases showing which compounds act on those characteristics.
Chen will lead a team of experts in informatics, statistics, computer science, biology and medicine from MSU, Stanford University, the University of California at Berkeley and Spectrum Health.
Initially, the research will focus on three under-studied diseases: multiple organ dysfunction syndrome; a type of brain tumor called diffuse intrinsic pontine glioma; and a form of liver cancer called hepatocellular carcinoma.
An extension of the project’s clinical component also received $867,000 to fund innovative uses of electronic health records from the Spectrum Health-Michigan State University Alliance.
Chen’s earlier research identified an anti-parasite drug called Niclosamide that could be repurposed to treat the liver cancer hepatocellular carcinoma. Under the current study, he hopes to optimize Niclosamide so that it can be used in clinical trials.
His clinical counterpart and co-investigator at Spectrum Health is Dr. Surender Rajasekaran, a pediatric intensivist working in critical care medicine, who also serves as medical director of research at Spectrum Health Helen DeVos Children’s Hospital.
Chen's study dovetails with one of Rajasekaran’ s primary research objectives: to better understand how and why children suffer from organ failure in the pediatric intensive care unit. Studies carried out over the past three decades show that any disease or trauma can potentially lead to organ failure, Rajasekaran said.
“It is frustrating that all our scientific understanding has not led to any meaningful change in the way we treat these patients,” Rajasekaran said. “That is why collaborating with Dr. Chen is of the utmost importance, as we can leverage his expertise into fundamentally changing the way we care for children in the ICU.”