Dr. A team led by Yongchao K. Ma has identified a new culprit in the progression of SMA: increased activity of an enzyme called cyclin-dependent kinase 5 (Cdk5). This enzyme disrupts the cells’ power plant, known as mitochondria, leading to motor neuron degeneration before visible symptoms appear.
Surprisingly, the researchers showed that a Cdk5 inhibitor reversed mitochondrial dysfunction and motor neuron degeneration in mouse and human induced SMA pluripotent stem cell models. Use of the inhibitor led to significant improvement in SMA symptoms.
Dr. Ma expressed his excitement by saying, “We promise a completely new treatment for children with SMA.” The proposed treatment may benefit all SMA patients, including those who cannot benefit from existing gene therapies. Future developments aim to improve the inhibitor for safer and more effective treatment.
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Source: Ferra
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