ALS is a progressive disease that causes the loss of muscle function, and it often develops very quickly after a diagnosis. Rami Epstein, the CEO of the biopharmaceutical company called Kadimastem, stated that many people only live for five years after an ALS diagnosis. His company aims to change this with their new therapy, AstroRx. They are currently speaking with the FDA to advance this treatment so that it may be widely available one day.
About Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic lateral sclerosis (ALS) is a progressive, neurological disease that is characterized by the destruction of nerve cells in the brain stem, brain, and spinal cord. Due to this destruction, muscles weaken and people lose control and voluntary movement. As the disease progresses, the muscles necessary for breathing weaken, leading to death. There are two types of this disease: sporadic and familial. Sporadic is the most common, as 90-95% of cases fall into this category. The latter means that it is inherited. ALS is a very rare disease, with an incidence of 3.9 of every 100,000 people in the United States. While anyone can have ALS, white males aged 60-69 are at the highest risk.
The cause of ALS is unknown. In the familial form of the disease it is known that a mutated gene is inherited from parents, but it is still not fully understood and only accounts for 5-10% of cases. It is believed that a connection between frontotemporal dementia and ALS exists. Other theories are that exposure to certain substances or toxins leads to the development of ALS.
Symptoms of ALS vary from patient to patient. They also worsen as the disease progresses. Symptoms begin as difficulty with small movements and everyday things like walking. At the beginning of the disease, people may trip and feel weakness in their arms, hands, and legs. As it progresses, people experience difficulties with speaking and swallowing, slowed and slurred speech, twitches and cramps in the muscles, and difficulty holding good posture. At the end of the progression people will be unable to move their muscles gradually, which affects the entire body. This inability affects movements like blinking. While people with ALS experience loss of muscle function, they do not lose any of their cognitive abilities. Their senses are generally not affected either.
A diagnosis is reached after a physician notices the symptoms and performs tests to rule out other conditions like lyme disease, HIV, or multiple sclerosis. Blood tests may be used to accomplish this. After other conditions are ruled out, doctors will use electromyographies (EMGs), nerve conduction studies (NCSs), and magnetic resonance imaging (MRIs). After a diagnosis is obtained, treatment is often symptomatic, as there is no cure for ALS. Treatment includes physical and speech therapy, nutritional and ventilation support, medication for depression or anxiety, medication to relieve tenseness and pain in muscles, hospice care, and riluzole, which reduces damage to the motor neurons.
About AstroRx
AstroRx cells are meant to replace the functions of destroyed cells through the use of off-the-shelf stem cells. These stem cells are differentiated into nervous system support cells such like astrocytes. Astrocytes remove toxic material from damaged neurons, and they can slow the deterioration of these cells through neuroprotection. These cells are injected through the spinal canal. The company is considering using this therapy to treat other neurodegenerative diseases as well.
About Kadimastem’s Study
Five patients participated in cohort A of Phase 1/2 of this study, and they were dosed with 100×10⁶ AstroRx cells. Post-treatment follow up consisted of the following six months. After these six months the ALS Functional Rating Scale-Revised (ALSFRS-R) was used to evaluate disease progression. This test showed that AstroRx did support the malfunctioning neurons and slowed the progression of the disease.
This test was performed in the three months before treatment, and the average rate of muscle function saw a decrease of -.87 per month. After treatment the same test was conducted, and muscle function increased at a rate of +.26 per month. Along with an increase in muscle function, there were no serious adverse effects or toxicities that would limit dosages.
The company will conduct cohorts B and C in order to test higher dosages and repeated treatments. Researchers want to see how effective the AstroRx cells can be. These positive results and the safety profile will hopefully allow Kadimastem to work with the FDA and make this treatment widely available.
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