Question

Should researchers or the family “play God” and try to correct the neuronal cells that are affected in Spinal Muscular Atrophy? Discuss your views on this potential stem cell therapy and discuss the ethical questions that you think need to be considered when developing new DNA based medical treatments.

Answer 1

Stem Cell Therapy is considered to be an alternative method for treating Spinal Muscular Atrophy (SMA) and other neurodegenerative disorders. Stem cell transplantation is a potential therapeutic strategy not only via cell replacement but also by modification of the extracellular motor neuronal environment, through a trophic and neuroprotective effect. A variety of cell sources have been considered for cell therapy.

Many of the treated patients showed major improvements in motor function, muscle tone and strength, speech and swallowing, balance and coordination, and fine and gross motor skills. Moreover, the disease progression has been reported to be significantly slower after stem cell transplantation.

Mechanisms

Mesenchymal Stem Cells (MSCs) are very attractive multipotent stem cells for Spinal Muscular Atrophy (SMA) cell therapy because of their great plasticity and their ability to provide the host tissue with growth factors and to modulate the host immune system.

The MSCs transplantation increases neuron survival and prevents gliosis, as microglia and astrocytes are both the target and cause of neuroinflammation, MSCs can rescue neurons and oligodendrocytes from apoptosis through the release of trophic and anti-apoptotic molecules, resulting in the induction of a neuroprotective microenvironment.

In addition, Mesenchymal Stem Cells can promote the proliferation and maturation of local neural precursor cells, leading to their differentiation into mature neurons and oligodendrocytes.