An issue with current treatments for Parkinson’s disease is that they only provide temporary relief from symptoms. There are no therapies that halt or even slow the progression of the disease. Neuraly aims to change this with their new investigational compound NLY01. It is intended to stop the toxicity of immune cells. A Phase 2 study is currently being conducted to evaluate the safety and efficacy. Researchers are very hopeful that this therapy will be successful, as it would be the first viable treatment that stops the progression of the disease.
About Parkinson’s Disease
Parkinson’s disease is a progressive disorder that affects the central nervous system (CNS). It is characterized by its effect on movement through five different stages. As the disease progresses, severity increases. Stage one is characterized by subtle tremors on one side of the body. In stage two symptoms are more noticeable, with tremors and rigidity on both sides of the body. Stage three brings loss of balance and slow movement, while stage four makes it impossible for one to live independently. Stage five is the most severe, as patients cannot stand or walk. Hallucinations and delusions are common symptoms of this stage.
Parkinson’s disease occurs due to the death of motor neurons, some of which produce dopamine. Dopamine is important in the transmittance of messages to the muscles from the brain, so the loss of dopamine results in the loss of motor functions. Abnormal brain activity occurs when these neurons are lost. Doctors do not know why these motor neurons die, but they do suspect a few factors that play a role, such as genetics, environmental factors like toxins, and Lewy bodies.
There are no FDA approved therapies for Parkinson’s, and treatment is symptomatic. Treatment options include dopamine substitutes, carbidopa-levodopa, MAO-B inhibitors, catechol-O-methyltransferase (COMT) inhibitors, anticholinergics, and amantadine.
About NLY01
NLY01 was created by researchers at John Hopkins and it is intended to slow the progression of Parkinson’s disease. It activates the glucagon-like peptide 1 receptor in a brain-penetrating, long-term form. It is a form of exenatide, a small protein. NLY01 is neuroprotective, working to stop the degeneration of the brain, and has been indicated for the treatment of Parkinson’s and Alzheimer’s.
Study of NLY01
This trial is in its second phase, and the first patient has been dosed. Researchers are hoping to enroll 240 participants from the ages of 30-80 throughout the United States and Canada. It will continue for 36 weeks and test the safety and tolerability of NLY01. It is randomized, with patients receiving either a placebo or NLY01.
This second phase is based off of preclinical data from mouse models of Alzheimer’s and Parkinson’s. It slowed neuron death and progression while helping motor and cognitive functions. Mice who received the therapy were proven to live longer lives as well.
Phase 1 of this study was given to people without Parkinson’s disease via an under-the-skin injection. This phase was meant to test the safety and tolerability of NLY01. It was injected once a week, which was shown to provide a high concentration within the blood.
Medical professionals are hopeful that this treatment proves to be effective, as it would be the only therapy that slows the progression of neurodegenerative diseases. Results are expected to be released within two years.
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