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World first in stem cell therapy: new treatment for hereditary diseases

“We started a first clinical study this week, in which we added healthy stem cells from the patient with a hereditary disease to her blood. body are rejected,” says Smeets.

“The difference is that we now grow the stem cells in large numbers outside the body and administer them again via the blood. In this way we give muscle production a boost. Those stem cells fuse with the existing muscle fibers and can repair the muscles in this way.”

Stem cells get ‘a color’

“We are now treating the lower leg of one of the patients. I spoke to her yesterday and she is doing well. Because they are the body’s own stem cells, we cannot distinguish them from the cells she already has. So we give the stem cells a color Smith explains.

“Today we will take a muscle biopsy from her. Then we will see how many new stem cells have arrived in that muscle and compare it with the other leg. This gives us an idea of ​​the effectiveness of the therapy.”

Muscle loss due to cancer

“This can mainly be used for diseases where you know that you are adding healthy stem cells. Also for non-hereditary diseases where people have muscle loss, such as cancer or aging. It becomes more difficult in diseases where it is unclear where the defect comes from and how you that has to be repaired, for example in ALS, because you have to know that the cells you return are healthy.”

Smeets: “There is still some work to be done, but the fact that we are working on the treatment of patients shows that we are already well on our way. This is further than much other research in this field.”

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