ProMIS Neurosciences is a biotechnology company that works specifically with antibody treatments, and they have recently developed intrabodies for the treatment of amyotrophic lateral sclerosis (ALS). This same therapy has been indicated for other neurodegenerative diseases, such as frontotemporal dementia.
About Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic lateral sclerosis (ALS) is a progressive, neurological disease in which nerve cells in the brain stem, brain, and spinal cord deteriorate. Due to this deterioration, muscles weaken and people lose control of them and their voluntary movement. In the late stages of ALS, the muscles necessary for breathing weaken, resulting in death. There are two forms of this disease: sporadic and familial. The former is the most common, with 90-95% of cases falling into this category. Familial means that it is inherited. ALS is a very rare disease, as its incidence is 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.
Medical professionals do not know the cause of ALS. 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. Researchers believe that there is a connection between frontotemporal dementia and ALS. Another theory is that exposure to certain substances or toxins leads to the development of ALS.
Symptoms of ALS vary between individuals. They also worsen as the disease progresses. Symptoms begin with difficulty with small movements and everyday things like walking. At the onset 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. In the later stages 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 obtained 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). Once 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.
Intrabodies are an antibody therapy that target the toxic proteins that play a major role in neurodegenerative diseases. They block these proteins from spreading to other cells and increase the speed at which they die. The specific protein that is targeted by ProMIS’ therapy is TDP-43.
Using a gene therapy vector, medical professionals are able to inject the intrabodies directly into the cell. Preclinical data has provided positive results, giving hope to researchers that this treatment may be able to help those with ALS and other neurodegenerative diseases. They are also evaluating other disorders that may be aided by intrabodies, as this therapy has the ability to target any misfolded or toxic protein.
Hopefully the results of this therapy will continue to be positive, as those with ALS are in need of a viable therapy.
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