A New Therapeutic Approach for an Incurable Disease
PCD (Primary Ciliary Dyskinesia) is a rare disease that disrupts the function of cilia in the airways. As a result, the mucociliary clearance system fails, preventing the effective removal of mucus and pathogens. This leads to chronic respiratory infections and progressive lung damage. Currently, there are only symptomatic treatments, and no approved causal therapy exists.
In their project, "Exploring the Therapeutic Potential of hiPSC-Derived Basal Cells in the Context of Primary Ciliary Dyskinesia (PCD) Utilizing an In Vitro Cell Therapy Model," which is funded with nearly €150,000, the research tandem explores an innovative solution: The transplantation of hiPSC-derived basal cells (iBCs)—stem cells derived from a patient’s own cells, genetically corrected, and differentiated into basal cells. These cells are expected to integrate into the damaged airway epithelium and form functional cilia. “Our research could mark a turning point in PCD treatment,” explains Dr. Laura von Schledorn. “If we succeed in regenerating the diseased epithelium with genetically corrected stem cells, we could develop a causal therapy for PCD in the long term.”

Targeted Cell Therapy Using Innovative Methods
To facilitate the integration of stem cells into the airway epithelium, the team is investigating various Airway Surface Preparation Techniques (ASPT)—targeted methods to prepare the airways for cell transplantation. These include chemical and physical procedures that selectively remove diseased tissue to create space for transplanted cells.
“Even a small proportion of healthy, cilia-bearing cells could be enough to restore the lung’s self-cleaning function,” says Dr. Ben Ole Staar. “We are examining the minimum number of functional cells needed to achieve a significant improvement in mucociliary clearance.”

From Laboratory Research to Clinical Application
Initially planned for 24 months, the project will evaluate the therapeutic potential of stem cell therapy in an in vitro model. In the long term, this approach could be further developed in clinical trials and potentially applied to other lung diseases such as cystic fibrosis (CF) or COPD.
Funding for the project is provided by the nTTP-GCT program, which supports innovative research approaches in gene and cell therapy. The tandem’s work could be a key milestone on the path to a sustainable therapy for PCD patients.

We wish Dr. von Schledorn and Dr. Staar great success with this promising project and in their future research endeavors!
 

Text: BREATH/AB

Photo: private