Diagnostics for rare rheumatic diseases
Autoimmune diseases such as systemic sclerosis, systemic lupus erythematosus, or Sjögren’s syndrome trigger inflammatory reactions in connective tissue. They are therefore grouped as connective tissue diseases (CTDs) and count among rare rheumatic diseases. The optimal therapy depends on which arm of the immune system drives the inflammatory response. Based on this, patients could be grouped into two categories, but a definitive diagnosis has not yet been possible. Researchers at TWINCORE, the HZI, and MHH aim to develop reliable diagnostic tools to distinguish the groups precisely. The PREDICT-CTD consortium (PREcision Diagnostics to CombaT Connective Tissue Diseases) brings together basic researchers, clinicians, and clinician-scientists who, using modern multi-omics technologies such as genetic diagnostics and immunological phenotyping, will refine the causes of CTDs. “The insights gained will help us develop more precise diagnostic procedures,” says Prof. Yannic Bartsch, head of the Helmholtz Young Investigator Group “Antiviral Antibody-Omics” at TWINCORE. Bartsch, together with Dr. Dr. Theresa Graalmann, head of the Clinical Junior Research Group “Translational Immunology” at TWINCORE, is responsible for leading the project, which is funded for five years with about €2.7 million.
Pathoblockers for the liver: A new approach against primary sclerosing cholangitis
Primary sclerosing cholangitis (PSC) is a chronically progressive inflammation of the bile ducts that can progress to liver failure. To date, the disease can only be treated surgically or with antibiotics in the event of infection. Researchers from MHH and HZI are pursuing a new therapeutic approach in the project “Sialidase targeting pathoblocker therapy for primary sclerosing cholangitis” (StopPSC). The putative cause of PSC is bacteria that attack sugar structures on the surface of bile duct cells using the enzyme sialidase. In StopPSC, the researchers aim to develop an agent that inhibits this enzyme, thereby specifically turning off or weakening the disease-causing properties of the bacteria. Such agents are referred to as pathoblockers or virulence inhibitors. HZI’s “Chemical Biology” division, led by Prof. Mark Brönstrup, as well as the research group “Pharmacokinetics and Pharmacodynamics”, lead by Dr. Katharina Rox, are already developing pathoblockers against various clinically relevant pathogens as antibiotic substitutes. “We are excited to bring HZI’s expertise in bacterial virulence inhibitors to the field of rare chronic inflammatory liver diseases in close partnership with MHH,” says Brönstrup. “Our goal is to develop tailor-made therapies for the orphan disease primary sclerosing cholangitis using medicinal-chemistry and pharmacologically optimized agents to improve patients’ prognosis,” adds Rox. The project coordinated at MHH by Prof. Benjamin Heidrich is funded with €2 million, about 40 percent of which goes to the HZI.
