Verena Schöwel has a vision. The doctor wants to help people who suffer from rare, previously incurable forms of progressive muscle atrophy. To do so, she wants to store stem cells from these patients’ muscle tissue in a medicinally compatible biobank until suitable therapies are available. Professor Simone Spuler’s entire lab, whose focus is myology (the study of muscles), is working intensively on the development of such approaches with a primary focus on gene corrections.
Schöwel and her colleagues have now been awarded the Science4Life Award for their concept for a biobank called “MyoPax.” The prize supports prospective entrepreneurs from the life sciences, chemicals, and energy sectors in translating their innovative ideas into a functioning business plan and establishing as many contacts as possible with investors and companies. “Our concept is now in place and we are currently working on financing the biobank,” says Schöwel, who is currently being supported in her project by the Helmholtz Enterprise funding program.
After a week in the refrigerator, only stem cells are left
For a long time, it was difficult to nearly impossible to treat muscle disorders with adult stem cells. “But this was due to the fact that the stem cells always began to differentiate quickly in the laboratory,” Schöwel explains. This led to the loss of their ability to transform into functioning muscle cells in the organism. “Very early clinical studies were particularly disappointing because the cells injected into the muscles were no longer stem cells,” Schöwel says.
However, the researchers in Spuler’s lab have now been successful in developing a particularly gentle method to isolate and multiply highly sought-after stem cells from muscle tissue – so that they retain their regenerative potential. “Like all other researchers around the world, we used enzymes or antibodies to separate stem cells from the surrounding connective tissue cells,” Schöwel says. However, it appears that this initiates processes in the stem cells that trigger their differentiation.
Now scientists in Spuler’s lab only process the muscle tissue obtained by biopsy mechanically. First, it is slightly dissected with tweezers and then hypothermically treated for seven days at a temperature of 4 degrees Celsius. Everything but the muscle stem cells die off, leaving a pure stem cell culture behind with highly native characteristics. “It sounds simple,” Schöwel says, “but when my colleague Dr. Andreas Marg successfully applied the method for the first time, it was a real eureka moment.”
Long-term effects can also be expected from the cells
Spuler and her team have already shown that not only new muscles can be generated in mice with the help of stem cells obtained hypothermically. “The body’s own stem cell pool can also be enriched by the cells so that we can expect long-term effects, particularly in the case of progressive muscle diseases,” Schöwel says.
Together with a team from the GMP Laboratory at the University Hospital Tübingen, Janine Kieshauer from Spuler’s team has been successful in adapting the new method so that stem cells can also be produced to meet the requirements for medicines. Currently, the safety of the cells is being tested in-depth. “The goal is to be able to use them in humans for the first time in two to three years as part of a clinical study,” Schöwel says.
The researchers plan to do this in two stages. First, they want to use the patient’s own stem cells to repair muscular defects that do not result from a genetic disorder. The second step involves developing therapies for hereditary muscle diseases. These will also be treated with stem cells from the patients themselves. However, before Spuler and her team can inject the cells into their patients’ key muscle groups, they will have to remove the genetic defect by gene editing – i.e., by specifically repairing defective genes. The group is currently working to establish and optimize these methods.
For many patients, it’s a race against time
Genetic muscular dystrophies are associated with a progressive loss of muscle mass and have previously been untreatable. “But it is already technically possible for us to store the patient’s own stem cells at a level that meets medicines standards,” Schöwel says. Schöwel therefore hopes to be able to offer patients with muscular dystrophies the option to have stem cells removed at the earliest possible stage of the disease and stored in the biobank.
“This would allow patients the prospect of using their own stem cells in future therapy,” the physician says. For many patients, waiting for new research results and therapeutic advances is a race against time. Yet Schöwel is firmly convinced that the prospects for success are not bad at all.