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Cerebral palsy (CP) has a significant impact on the development of the muscle and skeletal systems after birth. It causes the progressive tightening, thinning and shortening of muscles, often leading to the development of so-called “muscle contractures”. Contractures prevent joints to move freely, making it difficult and often painful to walk, or to reach forward to grasp and hold objects. Over time, movement can get even more limited: as the patient grows, muscles get shorter, thinner, weaker and less flexible, which can make it harder to live an active, healthy and independent life.
Our research has shown that development of muscle contractures in CP are linked to a failure of the muscle to grow properly after birth. We have data showing that this is in part caused by a reduction of number and activity of stem cells in the muscles. The job of these stem cells, also called satellite cells, is to “repair” muscles when they get injured, and to help muscles grow bigger and stronger when the child develops into an adult. We are currently researching the causes for this deficiency in muscle stem cell activity and muscle growth in CP.
As a positive implication of our research, we have discovered that on the market there is an FDA-approved drug, called 5-Azacytidine, that could potentially help rescue the muscle growing capacity of these stem cells in CP. We showed that after being treated with this drug in a dish, the stem cells from children with CP grew new muscle tissue much better than did untreated stem cells. In effect, 5-Azacytidine seems to “reset” and unmask the growth potential of these stem cells in contractured muscle. Although further research and clinical trials will be needed, we hope that pharmacological treatment with this class of drugs in combination with physical therapy may one day slow down, prevent or even reverse, the progression of “muscle contractures” in CP.
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