50 Million euro investment for LUMC spin-off RougeTx advances therapies for inherited bleeding disorders.
The group of Dr. Heiko Locher, principal investigator at reNEW Leiden, uses stem cell-derived models to study the human inner ear. He will now receive EUR 800,000 from the Reggeborgh Foundation to further develop these inner ear models. The improved models could lead to novel, targeted and much-needed treatments for hearing loss and balance disorders.
“For some years now, we have been able to grow cells of the human inner ear from stem cells,” says Locher. “This is truly a breakthrough. Previously, there were no suitable models to properly study the inner ear and related disorders. Patients with hearing or balance disorders hence currently depend on technical aids, which unfortunately do not provide a solution for everyone.”
Locher and his group are among the few worldwide who can culture and use these unique inner ear models. With the new funding, the researchers will take the next step. By improving scalability and reproducibility, they will make the stem cell models broadly applicable for translational research and therapy testing.
An important element of the research plan involves the development of stem cell lines with fluorescent reporters. These reporters allow researchers to follow the development of different cell types in their models live. They will also use these reporters to isolate specific cell types using a machine that sorts cells from their inner ear models (FACS).
Dr. Wouter van der Valk, a senior researcher in the lab, explains why this is important: “Our models are complex and consist of dozens of cell types. That complexity is needed to study development, but it hinders the use of the models for therapy testing. If we isolate single cell types, such as hair cells or neurons, we can test therapies on them at larger scale.”
Using spatial transcriptomics – a technique that enables the creation of a genetic map of tissues – the team will further investigate the human inner ear. By translating knowledge of regulatory processes and signaling pathways during embryonic development into new culture conditions, they will create stem cell-derived models that better reflect the human inner ear.
Locher: “In recent years, we have made great progress. The results of this study may contribute to the development of long-awaited new therapies for hearing loss and balance problems. With the improved stem cell models, we want to make a direct impact on patients’ lives.”
