Results of the Cantabio Pharmaceuticals’ therapeutic program targeting the protein DJ-1 as a new therapeutic avenue for Parkinson’s disease will be presented at the 13th International Conference on Alzheimer’s and Parkinson’s Disease, being held in Vienna on March 29-April 2.
The presentation, to be delivered by Cantabio’s CEO Gergely Toth, PhD, will include data on the effect of the DJ-1 protein targeting small-molecule drugs. Cantabio’s studies in cellular and animal models of Parkinson’s disease have shown that treatment with these drugs significantly extended life span in fruit flies with oxidative stress, reduced cell toxicity and loss of dopaminergic neurons triggered by increased expression of alpha-synuclein (a protein linked to Parkinson’s pathology).
Previous studies have shown that the DJ-1 protein participates in molecular pathways that prevent oxidative stress, a damaging process that occurs in cells when free radicals are produced faster than the body can remove them. Mutations leading to defective levels of this protein can increase the risk of developing Parkinson’s and Alzheimer’s diseases, ALS, Type 2 diabetes and stroke.
DJ-1 is viewed as one of the principal targets for the treatment of Parkinson’s disease because it has been genetically connected to the familial form of the disease.
“We are thrilled to share further results on our DJ-1 protein targeting pharmacological chaperone program, as well as exciting findings in regard to DJ-1 loss of function relevant to Parkinson’s disease onset and progression,” Toth said in a press release. “These results from our in-house and collaborators’ work further advance the development of our DJ-1 targeting therapeutic candidates as a disease-modifying therapeutic for [Parkinson’s disease], and further validate our approach of tackling the disease at its source by preventing the formation of the toxic aggregates and oxidative stress that have been shown to be among the root causes of [Parkinson’s] and other neurodegenerative diseases.”
Parkinson’s disease is caused by a loss of dopamine-producing cells in the substantia nigra, a brain region that controls the starting and stopping of movement. Clinical manifestations associated with loss of dopaminergic neurons include freezing, gait problems, tremors, and rigidity. By damaging molecules that are crucial for the proper function of neurons, oxidative stress promotes neuronal death and loss of dopamine-producing neurons, thereby contributing to Parkinson’s disease.