STATHMIN-2 (STMN2) is a protein that has been proven to play a role in axonal development, repair, and stability. This protein is highly expressed in human motor neurons; research showed that it is regulated by TDP-43. When researchers deleted STMN2 from animal models, they found that the animals experienced axonal degeneration and the loss of innervation (stimulation) in muscles, which can lead to muscle weakness and eventual paralysis. More so, research showed that poor TDP-43 function contributes to lowered STMN2 expression. Almost all people who have been diagnosed with amyotrophic lateral sclerosis (ALS) are found to have lowered STMN2 expression. Unfortunately, people with ALS also lack any effective treatment options to address their health needs and improve outcomes.
That is why biotechnology company QurAlis Corporation (“QurAlis”) began developing QRL-201, a first-in-class molecule designed to restore STMN2 function. QurAlis recently reported that the first patient was dosed in the Phase 1 ANQUR clinical study. During the course of the study, researchers will seek to understand the therapy’s safety, pharmacokinetics, and tolerability. Altogether, there will be eight cohorts of eight patients each, trying eight different intrathecally administered QRL-201 doses.
What is Amyotrophic Lateral Sclerosis (ALS)?
Also known as Lou Gehrig’s disease, ALS is a progressive neurodegenerative disease that causes nerve cells (neurons) in the brain, brain stem, and spinal cord to die. Normally, these neurons help the muscles communicate with the nervous system. When these neurons degenerate and die, that communication is interrupted, leading to muscle weakness and wasting. 90-95% of ALS cases are sporadic (occurring without a known cause), while 5-10% are familial (inherited). It is more common in males and older individuals. Symptoms of ALS may, but do not always, include:
- Difficulty performing small movements or everyday tasks
- Frequent tripping and falling
- Muscle weakness
- Difficulty speaking and swallowing
- Muscle stiffness
- Impaired reflexes
- Aspiration pneumonia
- Slowed or slurred speech
- Inappropriate laughing or crying
- Gradual inability to move muscles
Many available treatments for ALS are designed to relieve symptoms; unfortunately, there are few therapies that address the disease’s underlying causes. Riluzole, pain relievers, depression and anxiety medication, ventilatory support, and physical, speech, and nutritional therapy may all be part of the ALS care plan.
