Study: Verdiperstat as a Treatment for Amyotrophic Lateral Sclerosis (ALS)

The HEALY ALS Platform Trial, being conducted by Biohaven Pharma, has begun enrolling patients. This study is intended to test the efficacy of three treatments on amyotrophic lateral sclerosis (ALS), including verdiperstat. Medical professionals hope that this study will not only improve the knowledge of ALS medications, but that it will better the lives of those with this condition as well.

About Amyotrophic Lateral Sclerosis (ALS)

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. 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 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.

The HEALY ALS Platform Trial

This trial is being conducted across 50 sites throughout the United States with the goal of identifying new ALS treatments in a faster and more innovate way. Top experts in this field, along with the Northeast ALS Consortium clinical trial network, are able to evaluate multiple drugs at the same time.

About 160 people are expected to enroll in this trial, and they will be randomized 3:1. Three groups will receive verdiperstat while the other will receive a placebo, which they will take for 24 weeks. Using the ALS Functional Rating Scale-Revised, researchers will look for changes in disease severity as the primary endpoint. They will also look for survival, changes in respiratory function, and muscle strength.

As ALS patients face an unmet medical need, this trial is very important. Its ability to discover new drugs at a faster rate could greatly improve the lives of patients. To find out more about the HEALY ALS Platform Trial, click here.

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