Treatment of Spinal Muscolar Atrophy with Intrathecal Mesenchymal Cells

Background and Objectives:

• SMA1 is a genetic disease causing progressive apoptosis of the second motoneuron, leading to complete paralysis.
• Given the reported efficacy of mesenchymal cells in treating other neurological diseases, the study aimed to treat children with SMA1 using these cells.

Methods and Results:

• Four children with SMA1 were treated with intrathecal injections of mesenchymal cells.
• All patients showed improvement in motility after three weeks, especially in distal muscles. Proximal muscles were less affected.
• The treatment’s effect lasted for about 30 days, necessitating repeated treatments once a month for 3 to 8 months.
• One patient who stopped the treatment died after 45 days. Another became completely paralyzed two months after stopping the therapy but regained skills after a new injection. The remaining two patients remained stable after initial improvement.

Conclusions:

• Intrathecal injections of mesenchymal cells enhance the motility of children with SMA1.
• Early treatment, before irreversible neurological damage, might potentially cure the disease.

Additional Information:

• Spinal muscular atrophy (SMA) is a group of diseases caused by a defect in the SMN1 gene. The most severe form, SMA1, manifests in the first few months of life, leading to severe muscle activity decline and eventual respiratory failure.
• Current drugs can slow the disease’s progression, but a definitive cure is not yet available.
• Mesenchymal cells have been used to treat neurological diseases like Parkinson’s and brain injuries. They can cross the blood-brain barrier, produce factors that interfere with apoptosis and neuronal growth, and can mature into tissues like neurons and glia.
• The study suggests that the short-lived effect of intrathecal mesenchymal cells is due to the local release of soluble factors, not regeneration.