The head of the team of researchers Demetrios Vassilatis, of the Biomedical Research Foundation of the Academy of Athens (BRFAA), noted that the discovery was still at a preclinical stage but might lead to a new treatment route in the future for the specific neurodegenerative disease, which affects millions of people worldwide.
“My research team in the United States, in collaboration with the neurology team at Baylor College of Medicine, was the first to discover the mutations that suppress the Nuclear receptor-related 1 (Nurr1) gene in patients with Parkinson’s Disease. The research in my laboratory in Athens in recent years has focused on answering the question whether the activation of the Nurr1 can be therapeutic in animal models of Parkinson’s Disease. Because in the dopaminergic neurons, Nurr1 creates a heterodimer (a molecule composed of paired proteins with some amino-acid sequence variations) with Retinoid X receptor A (RXRa), we decided to target Nurr1:RXRα chemically.”
Vassilatis also added that “In collaboration with Dr. Demosthenes Fokas of Ioannina University, we discovered various chemical compounds, among them BRF110, that selectively activate Nurr1:RXRα. In my laboratory at the BRFAA with my colleague Dr. Athanasios Spathis, we showed that BRF110 not only has the capacity to protect dopaminergic neurons from degeneration but also to improve the symptoms in animal models of Parkinson’s Disease.”
When asked the next steps that will be taken in the research, and if/when would clinical trials on people are likely Vassilatis said that “The questions arising from our experiments are many and the research required has many levels, including the basic and translational research. The discovery of pharmaceuticals is a lengthy and multi-stage process and we are in a pre-clinical stage. We would like to be the first to assess Nurr1 activators/antagonists in clinical trials. In this direction, we worked with Dr. Jens Schwamborn from the Luxembourg Centre for Systems Biomedicine (LCSB) and assessed BRF110 on dopaminergic neurons derived from the stem cells of a Parkinson’s Disease patient with very positive results. The next stages include improving the existing chemical compounds in potential clinical molecules, which could be developed in the next two to three years.”