NeuroSense Therapeutics, a biotechnology company, is in the process of developing PrimeC. PrimeC was created for the treatment of amyotrophic lateral sclerosis (ALS), and it is meant to slow or even halt the progression of the disease. There are currently two clinical studies being conducted, one in Tel-Aviv, Israel and the other in the U.S., specifically New York and Arizona. Not only are these studies being conducted to evaluate the efficacy of the therapy, but the FDA has also granted PrimeC Orphan Drug designation.
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.
About PrimeC
PrimeC is a combination drug that is meant to slow or stop the progression of ALS. It targets two of the underlying causes of ALS. In its pre-clinical studies it was shown to be neuroprotective in zebra fish with ALS-causing mutations. Not only did the analysis of motor neurons provide positive results, but fish that were treated with PrimeC better maintained their swimming abilities when compared to fish that were left untreated.
About the Clinical Trials
Two Phase IIa studies will be conducted in multiple locations in order to fully understand and evaluate the efficacy, safety, and tolerability of PrimeC. The ability to slow or stop disease progression will also be studied.
These studies will be taking place in Tel-Aviv, Phoenix, Arizona, and Colombia University in New York City. Each site will enroll 15 patients. Researchers are excited by the results that were shown in the pre-clinical studies, and they are hopeful that results will be just as positive in the participants of this study.
Along with these clinical trials, PrimeC has been given the Orphan Drug designation, which has also excited those working on the treatment. They hope that this designation in combination with their clinical studies will help to better the lives of those with ALS.
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