Amyotrophic Lateral Sclerosis (ALS)

What is Amyotrophic Lateral Sclerosis (ALS) ?

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, Charcot disease, or motor neuron disease (MND), is usually caused by the dysfunction of the motor neurons in humans. Motor neurons are nerve cells located in the brain, brainstem, and spinal cord that act as control units and important communication links between the body's nervous system and voluntary muscles. As a consequence, ALS patients’ muscles can not get the signal to move, leading to gradual weakness and atrophy of the muscles in the bulbar, extremities, trunk, chest, and abdomen. ALS usually develops in middle age and men are about one and a half times more likely to have this disease than women. Most patients die within two to five years after the onset of ALS, and only about 10% of them live more than ten years.

Fig.1 How the muscular movements due to deformation in the nerve cells in ALS disease are restricted. (OZKAN, 2017)

ALS Symptoms

When upper motor neurons are affected, patients can experience tight limbs, lack of coordination, muscle rigidity, and even severe throttling. Meanwhile, patients' knees and ankles often tremble at the touch of their limbs due to abnormally enhanced nerve reflexes. If the lower motor neurons are affected, muscle atrophy begins in the palms of the hands or soles of the feet and spreads upward, affecting the legs, arms, shoulders, neck, tongue, and breathing muscles in most patients. Eventually, the patient will be paralyzed with respiratory failure and has to rely on a respirator to survive.

Type of ALS in Adults

Currently, ALS can be divided into familial ALS (FALS) and Sporadic ALS (SALS), depending on its different causes. FALS is mainly caused by mutations of the Cu/Zn superoxide dismutase 1 (SOD1) gene in vivo. To date, more than 180 variants have been found that are associated with FALS development. Pilot studies have illustrated that SOD1 mutations are a group of toxic gain-of-function mutations, including but not limited to, neurotoxic protein aggregation, mitochondrial dysfunction, oxidative damage, as well as axonal transport defects. SALS is the most common type of ALS and may be caused by a common mutation in many genes or malfunctioning of certain physiological pathways or mechanisms.

The Treatment Options for ALS

Recently, a wide variety of therapies, like gene therapy, stem-cell therapy, T-cell therapy, have been developed for treating ALS in humans. Gene therapy involves inserting new genes into the cells of ALS patients to replace disease-causing genes. In addition, motor neuron-related cells, such as glial cells, have become targets for gene therapy. Glial cells surround and support motor neuron cells, and there is evidence showed that genetic modifications within glial cells also help restore motor neuron function. Furthermore, stem cell therapy, as a promising treatment, has attracted much attention and high expectations. Several research groups around the world are investigating the feasibility of treating ALS with stem cells that can proliferate and differentiate into a variety of cells to maintain motor neuron activity and function. Besides, treatment strategies attempting to increase the body’s production of Tregs have been used for ALS patients. The data have indicated that T-cell therapy may improve symptoms and slow ALS progression in early-stage clinical trials.

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Reference

1. OZKAN, N.; et al. Design and Acquisition of EOG Based Interactive Communications for ALS Patients. International Journal of Applied Mathematics Electronics and Computers. 2017, 1: 1-4.