Neuroprotective molecules are a group of particles that have the capacity to preserve the nerve cells’ structure or function.

There are many particles with this capacity. Some exist naturally in humans and regulate the structure and function of neurons. Others are derived from other organisms, like plants, or are pharmaceutically produced to treat diseases that cause degeneration of the cells of the central nervous system (CNS).

How do these treatments work?

Neurons are cells that have the capacity of transmitting information between one another and also to different organs in our body. The physical space where one neuron meets the next in the information route is called a synapse.

Many neuroprotective molecules act at these spots to modulate neuronal function, either by increasing the stimulation of the next cell in line or by decreasing the amount of the signal that is propagated onward.

Neurons exist in an intricate network that allows information to travel from one to the next until it reaches its end point. When one or more of these neurons do not work properly or die, important information can get lost.

Some molecules with neuroprotective capacity prevent neuron degeneration by reducing the amount of stress in a cell, thereby averting cell death.

Each neuroprotective molecule has its specific mechanism of action. These particles do not normally act directly on the cause of the disease. Instead, these treatments aim to compensate for the neuronal loss specific to each disease.

Neuroprotective treatment for SMA

SMA affects motor neurons, so-called because they are in direct contact with a muscle cell. Motor neurons are therefore the last to carry the information to the cells that have the ability to take action and create movement.

Unlike other treatments for SMA, the aim of neuroprotective therapy is not to increase the levels of the survival motor neuron (SMN) protein, the protein that is found in insufficient amounts in people with SMA. Instead they focus on problems these low levels cause on the information traveling to its final destination – the muscle cell to be impaired.

Mestinon (pyridostigmine bromide) is a neuroprotective molecule being studied for the treatment of SMA. It modulates neuronal function by increasing stimulation at the synapses and allowing the information to better reach the muscle cells.

Another neuroprotective molecule with increasing interest to treat SMA is olesoxime. It helps prevent neuronal death by preserving mitochondrial function and thus decreasing the level of stress in neurons.

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