Amyotrophic Lateral Sclerosis: The Quest for a Cure

According to a story from Bioscience Today, amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease or motor neurone disease, has continued to be a mysterious disease for the world’s scientific and medical experts. So far there have been very few treatment options developed for it and their impact is wildly inconsistent. The grim reality is that the illness is still a death sentence. However, continued research developments are inching bit by bit towards more effective treatments or hopefully a cure.

About Amyotrophic Lateral Sclerosis (ALS)

Amyotrophic lateral sclerosis, otherwise known as Lou Gehrig’s disease, is a rare, degenerative disease that causes the death of nerve cells associated with the voluntary muscles. Little is known about the origins of the disease, with no definitive cause in about 95 percent of cases. The remaining five percent appear to inherit the disease from their parents. Symptoms initially include loss of coordination, muscle weakness and atrophy, muscle stiffness and cramping, and trouble speaking, breathing, or swallowing. These symptoms worsen steadily over time; most patients die because of respiratory complications. Treatment is mostly symptomatic and the medication riluzole can prolong life. Life expectancy after diagnosis ranges from two to four years, but some patients can survive for substantially longer. To learn more about amyotrophic lateral sclerosis, click here.

The State of Research

A common characteristic of the disease the development of adhesive protein clumps in the brain tissue. One such protein is called TDP-43. Normally found only in the cell nucleus, this protein become toxic outside of its normal environment. The drug arimoclomol has the potential to stop the development of these toxic protein clumps and is currently in the midst of clinical testing for amyotrophic lateral sclerosis.

Some studies of the brains of patients have also revealed that free radicals could be part of the disease mechanism, causing oxidative stress that destroys neurons. Another experimental therapy called Copper ATSM, which is supposed to selectively deliver copper to cells deprived of oxygen, is also being tested for potential efficacy in the disease.

These are just some of experimental therapies that are currently being developed and tested. Other researchers are investigating the role of genetics; about ten percent of amyotrophic lateral sclerosis cases are linked to genetic mutations. Antisense therapies, which have been successfully used to treat other genetic disorders such as spinal muscular atrophy, are being tested that act on two of the most commonly affected genes in the disease: SOD1 and C9orf72. 

Other research is investigating the possibility that the virus retrovirus-K (HERV-K) plays a role in the illness. Some studies have suggested that the virus could release toxic proteins that could play a role in neuron death.

While it may be a while before a more effective treatment or a cure for the disease is available, the field of research in amyotrophic lateral sclerosis is slowly but surely making progress.


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