A researcher at Michigan State University secured federal backing for a study to examine the potential to use existing drugs to treat so-called orphan diseases.
Dr. Bin Chen, an assistant professor at MSU’s College of Human Medicine in the departments of pediatrics and human development, and pharmacology and toxicology, will use the $2.1 million, five-year from the National Institutes of Health to search databases of some 12,000 compounds that have already been developed. His research team wants to match the genetic characteristics of rare, or orphan, diseases to see if they are treatable with existing drug compounds.
Chen hopes to identify new or better treatments for an estimated 6,000 rare diseases. He’ll lead a research team that includes experts in informatics, statistics, computer science, biology and medicine from MSU, Stanford University, the University of California at Berkeley, and Grand Rapids-based Spectrum Health.
“Although individually those diseases afflict relatively few people, combined they are suffered by about 25 million Americans, some whose illnesses are life-threatening,” Chen said.
The NIH defines an orphan disease as one that affects fewer than 200,000 patients. Because orphan diseases are so rare, pharmaceutical companies generally do not invest in researching treatments.
Chen’s research initially will focus on three diseases: multiple organ dysfunction syndrome, a type of brain tumor called diffuse intrinsic pontine glioma, and a form of liver cancer known as hepatocellular carcinoma. Chen’s previous research identified an anti-parasite drug called Niclosamide that could treat hepatocellular carcinoma. In his current study, he hopes to optimize Niclosamide for use in clinical trials.
“Although the new study initially will focus on those three diseases, the same approach could be used to find treatments for other understudied diseases,” Chen said.
His research study fits with a separate initiative that received $867,000 in funding to fund innovative uses of electronic health records from The Spectrum Health – Michigan State University Alliance.
Chen’s clinical counterpart and co-investigator at Spectrum Health, Dr. Surender Rajasekaran, plans to research how and why children suffer from organ failure in the Pediatric Intensive Care Unit. Rajasekaran is a pediatric intensivist and medical director of research at Spectrum’s Helen DeVos Children’s Hospital.
Multiple organ failure in pediatric patients could stem from an infection, the flu or injuries in a vehicle crash, Rajasekaran said. He wants to understand how that can lead to organ failure and the interaction with a patient’s genetics.
“It is frustrating that all our scientific understanding has not led to any meaningful change in the way we treat these patients,” he 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.”