Prevail Therapeutics has released an update on their Parkinson’s disease treatment, PR001. They are currently dosing participants, and enrollment remains open. As of now, the study remains on track. Results are expected to be released in the second half of 2020. Researchers are excited for this study, as PR001 addresses an unmet need in the medical world. It is the first disease-modifying therapy for Parkinson’s, making it a viable option for those with GBA1 mutations.
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 and leads to Parkinson’s. 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 PR001
PR001 is a disease-modifying AAV-based gene therapy. It corrects the error in the GBA1 gene, as it fixes the deficiency of lysosomal enzymes.
It has been indicated for the treatment of Parkinson’s disease with a GBA1 mutation and Gaucher’s disease. Prevail Therapeutics specializes in treatments for neurodegenerative conditions, working specifically with gene therapies.
PR001 Update
PR001 is currently being studied in the PROPEL clinical trial, which is in Phase 1/2. Enrollment remains open as dosing continues, and results should be presented in the second half of this year. This trial is randomized and double-blind. It is intended to evaluate the tolerability and safety of increasing doses of PR001. It will also monitor biomarkers of disease activity.
This treatment has the potential to be an effective treatment for both Parkinson’s disease and Gaucher disease. Researchers are hopeful that it will be a viable treatment, as it would better the lives of many people who have experienced failed treatment in the past.
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