Michigan State University (MSU) College of Medicine investigator Bin Chen just was awarded $2.1 million by the National Institutes of Health (NIH) to scour vast databases of existing drugs in a quest to treat orphan diseases. The investigative team received an additional $867K to leverage EHRs from Spectrum Health-Michigan State University Alliance.
An estimated 6,000 diseases are considered too rare to attract much research according to MSU. Generally an orphan disease is defined as one that affects less than 20,000 patients. However, the amount of human suffering is severe as there are many millions suffering rare diseases around the globe. For example, in America approximately 25 million suffer due to some rare disease elements. In the case of this upcoming study the key indications include multiple organ dysfunction syndrome, diffuse intrinsic pontine glioma (brain tumor) and a rare form of liver cancer called hepatocellular carcinoma.
The NIH funding will help Chen probe and analyze 12,000 preexisting compounds with an eye toward repurposing for the treatment of under-studied diseases. Chen and team will use next-generation sequencing, matching molecular and genomic characteristics of rare disease against databases revealing which compounds may act on which characteristics. Chen’s team will include experts in informatics, statistics, computer science, biology and medicine from not only MSU but also Stanford University, UC Berkeley and Spectrum Health.
The team will initially focus on three-understudied diseases including 1) multiple organ dysfunction syndrome 2) diffuse intrinsic pontine glioma (brain tumor) and 3) hepatocellular carcinoma (a form of liver cancer). We offer a brief overview of these targeted rare disease. Multiple organ dysfunction syndrome was the focus of a study in 2017 that concluded it was commonly observed in pediatric intensive care units (PCIU) but that “a full understanding of the epidemiology and outcome of multiple organ dysfunction syndrome in children is limited by inconsistent definitions and populations studied.” Scholars believe sepsis is a leading cause. Diffuse intrinsic pontine glioma occurs in at least 300 children per year. The rare form of liver cancer called hepatocellular carcinoma causes 662,000 deaths per year.
Previous Work to Present
Chen isn’t new to this type of research project. He identified an anti-parasite drug named Niclosamide that he found could be repurposed to treat hepatocellular carcinoma (liver cancer). In the current study he hopes to leverage his finding (e.g. Niclosamide) so it can go into clinical trials.
Additionally co-investigator Dr. Surender Rajasekaran has focused on children who suffer from organ failure has dovetailed with Chen’s initiatives hence why the two join forces.
Bin Chen, PhD, assistant professor
Call to Action: If you or a loved one have a diagnosis concerning one of the targeted disease types in this study TrialSite News algorithms automatically track their research. Sign up for the Daily Digest. Biopharma companies interested in these disease segments should also consider tracking this research and even partnering with MSU and professor Chen.