The FDA has recently granted their approval to a Phase 2 trial of FHL-301, a treatment for those in the earlier stages of Parkinson’s disease. Forest Hills Lab will conduct the trial with the goal of evaluating the efficacy and tolerability of the potential therapy.
This repurposed peroxisome proliferator-activated receptor alpha (PPARa) agonist is able to bind to the receptor that it was named for. This treatment targets the GDNF gene, attaching to the DNA sequences and activating the gene. Once activated, this gene plays a role in the production of the protein glial cell line-derived neurotrophic factor. This protein goes on to aid in the survival, growth, and differentiation of neurons.
Because the loss of dopaminergic nerve cells is a major characteristic of Parkinson’s disease, preventing it could provide better outcomes for patients. In fact, preclinical demonstrated that FHL-301 could slow and even reverse disease progression.
Forest Hills Lab is very excited to launch this Phase 2 trial, which will be double-blind and run for 52 weeks. Researchers hope that FHL-301 continues to demonstrate positive results, as it could fill an unmet need for this patient population.
In addition, the lab is also in the process of developing therapies for other disorders of the central nervous system. Their future plans include a Phase 2 trial for treatments for diseases like Alzheimer’s.
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 slowed movement. 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.
Find the source article here.