CytRx Corporation has recently received the Fast Track designation for their ALS treatment, arimoclomol. This status was also given for the treatment of Niemann-Pick disease and sporadic Inclusion Body Myositis (sIBM). This news is very exciting for CytRx, who works to create therapies for various cancers and neurodegenerative conditions.
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.
Arimoclomol is a medication meant to amplify the production of heat-shock proteins. These proteins are responsible for improving lysosomal function, clearing protein aggregates, and fixing misfolded proteins. Patients take this drug orally, allowing it to cross the blood brain barrier. It has most recently received a designation from the FDA for the treatment of ALS, but it has been indicated for Gaucher disease, Niemann-Pick disease, and sIBM.
The Fast Track designation was given to arimoclomol after finding success in seven Phase 1 and 2 trials. It is now being evaluated in a Phase 3 study, whose results are expected to be announced in the first half of 2021.
CytRx is excited to see the progress of arimoclomol, as they hope that it will be able to better the lives of those with multiple neurodegenerative conditions.
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