Parkinson’s Institute and Clinical Center and Retrotope have paired up to explore the therapeutic potential of the investigational agent RT001 in patients with progressive supranuclear palsy (PSP), a type of treatment-resistant Parkinson’s disease.
Under Retrotope’s expanded access program for RT001, the institute’s physicians started dosing two PSP patients with the investigational therapy. This pilot study is expected to provide information to help guide future randomized placebo-controlled trials in PSP.
“The Parkinson’s Institute and Clinical Center brings novel therapies for [Parkinson’s disease] to patients in need,” Carrolee Barlow, MD, PhD and CEO of the institute, said in a press release. “We look forward to working with Retrotope in evaluating the responses to this therapeutic candidate in these patients.”
RT001 belongs to a new therapy class called deuterated polyunsaturated fatty acids, or D-PUFAs, that protect mitochondria and cells from damage caused by oxidation of fat molecules, which often results in cell death that is a hallmark of numerous neurodegenerative diseases including Parkinson’s. Mitochondria provide energy and are known as the “powerhouses” of the cells.
The investigational agent is an engineered, more stable form of linoleic acid that mimics the protective effects of the fatty molecule Omega-6.
RT001 is integrated in cell and mitochondria membranes and, as it is a stabilized fatty molecule, does not react with damaging free reactive oxygen elements — toxic compounds produced as a consequence of cellular oxidative stress — restoring membrane function and cellular health.
“The patients we treat with these fatal, degenerative diseases are willing to try therapies which appear safe and for which there is a mechanistic rationale that they may benefit from the treatment,” Barlow said. “Retrotope is a willing partner with a highly unique and well-studied approach to addressing neurodegenerative diseases.”
“The institute’s goal is to find a cure for Parkinson’s and Expanded Access programs like Retrotope’s are really at the forefront of generating hope for patients from novel therapies, and data for pharmaceutical companies to plan their drug trials,” she added.
The therapeutic potential of this investigational agent is being tested in patients with inherited Friedreich’s ataxia (FA) and infantile neuroaxonal dystrophy (INAD).
In addition, this new agent has been shown in preclinical studies to hold therapeutic potential in other illnesses where mitochondria function and induced cell death play a role, including Alzheimer’s, Parkinson’s, Huntington’s, and amyotrophic lateral sclerosis (ALS).
Preclinical data has shown that RT001 orally administered to mice with induced Parkinson’s disease could effectively improve the levels of dopamine by 2.5 times in the brain area most affected by the disease. In addition, the treatment effectively prevented brain cells’ degeneration in these animals.
“Early results in clinical trials of RT001 and Expanded Access programs for a variety of neurodegenerative diseases have generated encouraging results along with a favorable safety profile,” said Peter G. Milner, MD and chief medical officer of Retrotope. “We look forward to providing updates on this and other studies in the coming months as we support patients and investigators exploring the utility of RT001 to block lipid peroxidation in a range of primary neurodegenerative diseases.”