The new research center will be RNA drugs for better metabolism treatment

Most people are familiar with RNA in the form of an mRNA vaccine against COVID-19. But in a medical sense, RNA can be used for much more than that. For example, RNA drugs have been shown to restore muscle strength in patients with muscular dystrophy and treat other rare or common genetic diseases.

A new research center, RNA-META, led by Professor Jørgen Kjems at Aarhus University, with a grant of DKK 60 million from the Novo Nordisk Foundation, will develop new types of RNA drugs and methods for delivering them to diseased cells for targeted disease treatments. In addition to Kjems, an interdisciplinary team of top researchers will participate: Kurt Gothelf from Aarhus University, Louise Dalgaard from Roskilde University and Markus Stoffel from ETH Zurich. Their common focus in research centers is metabolic diseases such as diabetes, fatty liver, renal fibrosis and atherosclerosis.

RNA is both a key and a barrier to targeted therapy

RNA is found in all of our cells and acts as a messenger from our DNA code to the production of proteins. The beauty of using RNA in drug development, as opposed to traditional medicine, is that you can program drugs to affect the amount of any protein in the body in principle. “RNA can neutralize or even replace disease-causing proteins in affected cells. This means RNA drugs have almost unlimited potential to treat human diseases,” said Jørgen Kjems.

However, there are still many hurdles to be overcome before this potential can finally be realized. Drug RNA is large and unstable and one of the main challenges is delivering drug RNA effectively to and within the cells of organs that control metabolism, such as the pancreas, kidney, liver, muscle and brain.

Skills to go all the way

The field of RNA and the work to develop RNA treatments is not new territory for researchers. Professor Jørgen Kjems, for example, has worked with RNA for more than 40 years and has published a number of groundbreaking works in the field. Expertise within the research team is highly interdisciplinary and ranges from organic chemistry to cell biology to preclinical disease models, meaning that researchers have the professional strengths to optimize RNA treatment at all levels.

With this new center, researchers will focus on developing new and better treatments for metabolic diseases. The first step in developing this new type of drug is equipping it with the navigation to find its way to diseased cells in the body.

Researchers will design and manufacture powerful RNA drugs marked with markers that will help RNA drugs find their targets in the body. Here, they would tap into cellular machinery and regulate the genes that control the body’s metabolism, thereby alleviating the patient’s symptoms.

RNA drugs must first be tested on cells in the laboratory and then in animal models before they can be tested on humans in clinical trials. To complete the final step, researchers will need to work closely with the pharmaceutical industry to ensure that the newly developed RNA drug will be ready to treat patients.

Be aware of ambitious research

The Novo Nordisk Foundation gives amounts in the triple-digit million (DKK) range through its Challenge Program to ambitious projects that can help solve society’s great challenges. Jørgen Kjems and his team are one of seven experienced researchers to receive a Novo Nordisk Foundation Challenge Program grant this year.

“This grant is a huge recognition from the Foundation and makes a huge difference to our research. Hopefully in 5-10 years we will have drugs for a number of metabolic diseases for which there are currently only ineffective treatments or no treatment at all. In this way, we hope to make a difference to a large group of patients suffering from metabolic disorders,” says Jørgen Kjems.

Key participant in “RNA THERAPEUTIC CENTERS TOWARDS METABOLIC (RNA-META) DISEASES”

• Professor Jørgen Kjems, iNANO and Department of Molecular Biology and Genetics, University of Aarhus. Mail: (email protected), telephone: +45 28992086
• Professor Kurt Vesterager Gothelf, iNANO and Department of Chemistry, Aarhus University
• Professor Louise Torp Dalgaard, Department of Science and Environment, University of Roskilde
• Professor Markus Stoffel, Department of Biology, Swiss Federal Institute of Technology in Zurich (ETH Zurich)