The Parkinson’s Institute and Clinical Center and Retrotope are teaming up to study a potential new therapy for patients with progressive supranuclear palsy. For more information about this collaboration, you can view the source press release at Globe Newswire by clicking here.
Progressive Supranuclear Palsy (PSP)
PSP is a progressive neurological condition caused by the premature loss of nerve cells in certain areas of the brain. Over time, it can lead to difficulties with balance, movement, vision, speech, and swallowing, however, each case is unique. According to the NHS, PSP is caused by a build-up of the protein tau. Usually, tau is broken down in the brain, but in people with PSP, this process doesn’t occur properly. As a result, tau collects in clumps that can cause damage to the brain. The amount and location of these clumps can vary between patients, leading to a diverse range of symptoms.
Dr. Carrolee Barlow, CEO of the Parkinson’s Institute, says that after a request from their physicians, Retrotope has agreed to provide their experimental drug RT001 for the treatment of two patients with PSP under an Expanded Access scheme. The Institute and Retrotope will work together to evaluate the effects of the drug in these patients. The information they gain from this study may be useful when it comes to designing future randomised control trials of RT001 in patients with PSP.
RT001 is part of a new category of drugs known as D-PUFAs (deuterated polyunsaturated fatty acids) and is designed to protect against cell death caused by lipid peroxidation damage. Since this process has been linked to several neurodegenerative conditions, including PSP, Dr Molinari from Retrotope says that it “makes a great deal of sense” to see if downregulating it could help patients. RT001 is also being investigated for use in other conditions, and pivotal studies of the drug in INAD and Friedreich’s ataxia are currently being initiated.