According to a story from the Houston Chronicle, researchers are hoping that an immunotherapy treatment that was originally funded by an Ice Bucket Challenge grant could be a viable option for treating amyotrophic lateral sclerosis. Clinical trials testing the therapy against the disease are expected to be underway soon. There are currently few available treatments for this illness, and it is ultimately fatal.
About Amyotrophic Lateral Sclerosis
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 amyotrophic lateral sclerosis, 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 Immune System and Amyotrophic Lateral Sclerosis
The immunotherapy in question involves the extraction of immune cells from a patient, expanding and restoring their functionality, and then reintroducing these cells into the patient’s body. Hopefully, these restored immune cells will be able to prevent the neuron death that happens in the brain in amyotrophic lateral sclerosis.
Dr. Stanley Appel has been researching this disease for decades and has helped uncover the role that the immune system plays in disease activity. The immune system ultimately finishes off the neurons once they have become damaged. Dr. Appel’s research also helped reveal that many patients have a family history of immune diseases such as thyroid disease and multiple sclerosis.
It may seem contradictory to use an immunotherapy to treat a disease that involves the immune system, but when Dr. Appel’s team bred an ALS mouse with a mouse that had no immune system, it died much more rapidly than usual, indicating that the immune system was still fighting against ALS to some extent. Preliminary tests with some human patients indicate that this treatment could be a real breakthrough.
