Parkinson’s Institute and Clinical Center and Retrotope Announce Collaboration on New Drug Treatment Therapies for Parkinson’s Disease Patients

Los Altos, California, UNITED STATES

SUNNYVALE and LOS ALTOS, Calif., Oct. 04, 2018 (GLOBE NEWSWIRE) -- Parkinson’s Institute and Clinical Center, which provides comprehensive patient care, while discovering new treatment options to improve the quality of life for all Parkinson’s disease (PD) patients, and Retrotope announced today a collaboration on a new drug therapy to treat patients with Progressive SupraNuclear Palsy (PSP), a type of treatment-resistant PD.

“The Parkinson’s Institute and Clinical Center brings novel therapies for PD to patients in need,” said Dr. Carrolee Barlow, M.D., Ph.D., CEO of the Institute. “Retrotope’s approach to reducing mitochondrial oxidative damage by fortifying lipids against peroxidation is truly novel and differentiated from other therapies both available and in development. Retrotope has responded to our physicians’ requests for Expanded Access, and we have initiated treatment in two PSP patients under the care of the Institute with Retrotope’s RT001. We look forward to working with Retrotope in evaluating the responses to this therapeutic candidate in these patients.”

The study is expected to help guide the design of future randomized placebo-controlled trials in PSP. RT001 is the first-in-class of a new drug category called deuterated polyunsaturated fatty acids (D-PUFAs), which are a novel approach to protect against lipid peroxidation damage resulting in cell death that is a hallmark of numerous neurodegenerative diseases including Alzheimer’s disease, Parkinson’s disease, PSP, Huntington’s disease, ALS and several inherited disorders including Friedreich's ataxia (FA) and infantile neuroaxonal dystrophy (INAD) in which Retrotope is planning formal clinical trials.

PSP is believed to be a tauopathy, which is a progressive aggregation of hyper-phosphorylated tau protein in the brain, with accompanying brain atrophy and neuronal cell death.  Symptoms include loss of balance, lunging forward when mobilizing, fast walking, bumping into objects or people, and falls. Later symptoms are dementia (typically including loss of inhibition and ability to organize information), slurring of speechdifficulty swallowing, and difficulty moving the eyes, particularly in the vertical direction. Approximately six per 100,000 people are believed to be affected with the disease. There are currently no approved treatments.

“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,” said Dr. Barlow. “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.  It is a win-win for both the Institute and Retrotope.”

Peter G. Milner, M.D., Chief Medical Officer of Retrotope said, “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. 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.”

Robert J. Molinari, Ph.D., Chief Executive Officer and Co-Founder of Retrotope, added, “As lipid peroxidation is believed to be involved in PSP, it makes a great deal of sense mechanistically to determine if the well-documented downregulation of lipid peroxidation by RT001 might help PSP patients. Therefore, while Retrotope initiates its pivotal studies of RT001 in INAD and Friedreich’s ataxia, the Company is willing to respond to requests of informed physicians for treatment of other diseases in which lipid peroxidation is implicated.”

About the Parkinson’s Institute and Clinical Center:
Parkinson’s Institute and Clinical Center is at the forefront of patient care and therapy development. We are the nation’s only Institute that provides patient care, basic science research, and clinical research in one integrated model. Our unique freestanding organization directly connects research to patient care – from the "bench to bedside.”  For 30 years, our team of epidemiologists, physicians, scientists, and therapists, have been at the forefront of every clinical and scientific discovery in the field of Parkinson’s research.

Since our inception, patients have come from 38 states and many foreign countries for their care. We have managed more than 135 clinical research trials, medically evaluating more than 95 different drugs and therapies for Parkinson’s. The result: our Institute has been directly involved in bringing every Parkinson’s drug currently available to the market. The Institute is at the forefront of discovering and developing the latest, most innovative treatments and therapies for people living with Parkinson's.

For more information visit or call (408) 734-2800.

About Retrotope:
Retrotope, a privately held, clinical-stage pharmaceutical company is creating a new category of drugs for the treatment of neurodegenerative diseases. Composed of proprietary compounds that are chemically stabilized forms of essential nutrients, these compounds are being considered as disease-modifying drug therapies for many intractable diseases, such as Infantile Neuroaxonal Dystrophy (INAD), ALS, Late Onset Tay Sachs (LOTS), Progressive SupraNuclear Palsy (PSP), Huntington’s disease, mitochondrial myopathies, and retinopathies.  RT001, Retrotope’s first lead candidate, is being tested in placebo-controlled clinical trials for the treatment of Friedreich's ataxia, a fatal orphan disease, and in compassionate use studies for other neurodegenerative diseases such as INAD, LOTS, PSP, ALS and a genetic form of Alzheimer’s disease.  For more information about Retrotope, please visit

Contact for Parkinson’s Institute and Clinical Center:
Chris Di Salvo
Phone: (408) 506-0455

Contact for Retrotope: Nancie Steinberg, Burns McClellan
212-213-0006, ext. 318,