From bench to bedside: muscle stem cell therapy reaches the clinic

In a recent article in Analysevisie, Erika describes the remarkable journey of a muscle stem cell therapy from an initial research idea to clinical application.
The work started more than a decade ago with the observation that some patients with mitochondrial DNA (mtDNA) disorders have muscle stem cells (mesoangioblasts) containing very low levels of the disease-causing mtDNA mutation. This opened the possibility of isolating a patient’s own healthy stem cells, expanding them and returning them to the muscle to improve muscle function. Developing the necessary isolation and culture protocols, establishing GMP-compliant production and completing the first safety studies required years of collaborative work. In November 2025, this journey reached an important milestone when the first patient with a rare mitochondrial muscle disease was treated with their own stem cells. Initial clinical studies have shown that the procedure is safe, while ongoing research will determine the extent to which it can improve muscle strength and fatigue. For Erika, who has worked with Bert Smeets’ research team for almost 30 years, seeing laboratory research translate into a potential treatment, and meeting the patients who may benefit from it, makes the long road from bench to bedside particularly rewarding.

Curious about the article? You can read it in the trade magazine: Analysevisie, Issue NR 3, June 2026