Enrollment Complete in RT001 Trial for PSP


In a recent news release, biopharmaceutical company Retrotope shared that enrollment was complete in its Phase 2 clinical trial. During the trial, researchers will evaluate RT001, a first-in-class oral treatment, for patients with progressive supranuclear palsy (PSP). Altogether, researchers fully enrolled patients within this clinical trial in under 6 weeks.


So what exactly is RT001? In a February 2020 article from NeurologyLive, author Marco Meglio describes the treatment as:

a chemically stabilized fatty acid that incorporates into mitochondrial and cellular membranes and, in turn, stabilizes them. The patented, first-in-class, orally available isotopic-reinforced polyunsaturated fatty acids (D-PUFA) has been shown to decrease levels of lipid peroxidation in PSP patient mesenchymal stem cells, as well as restore mitochondrial function and structure to damaged cells.

The treatment, a synthetic linoleum acid, was first discovered using Retrotope’s own novel technology designed to reduce oxidative stress associated with lipid peroxidation. When lipid peroxidation is unbalanced, that is when a variety of neurodegenerative conditions may develop. RT001 received Orphan Drug designation, a special status granted to drugs or biologics intended to treat those with rare conditions. In America, rare conditions are defined as those affecting under 200,000 people. Beyond PSP, Retrotope is evaluating RT001 for patients with amyotrophic lateral sclerosis (ALS), Friedreich’s ataxia (FA), and infantile neuroaxonal dystrophy.

RT001 for PSP

Currently, there are limited therapeutic options available for patients with PSP. Most treatments focus on symptom management as opposed to treating the underlying condition. Thus, new treatment options are urgently needed. This is highlighted in how quickly enrollment filled up; the PSP community is looking forward to advancing research within this realm.

Cell culture research first spurred researchers to consider RT001 as a potential treatment for patients with PSP. Within this study, researchers will compare the safety, efficacy, and tolerability of RT001 in comparison with a placebo. Patients will receive either a placebo or RT001 each day for a 48-week period. Researchers hope to build off of already established clinical data from 4 patients with PSP who have already received treatment through an expanded access program. During the trial, researchers hope to evaluate:

  • How effective RT001 is in treating patients with PSP according to the PSP Rating Scale and a secondary scale from the FDA
  • Whether RT001 improves gait, behavior, or ability to perform daily activities
  • How safe RT001 is for long-term treatment

Progressive Supranuclear Palsy (PSP)

Progressive supranuclear palsy (PSP), a neurodegenerative brain disorder, occurs as nerve cells in the brain stem become gradually more damaged, though what causes that is still unknown. In a small amount of cases, inherited MAPT gene mutations cause PSP. Regardless of the cause, PSP is characterized by abnormal tau protein deposits in the nerve cells. As these proteins accumulate, and nerve cells die, it results in hindered movement, impaired speech, and lessened ability to control eye movement. Typically, PSP affects those between ages 45-75, though it more commonly occurs in older individuals. Additionally, more patients are males than females. Because of similar symptoms, PSP may be misdiagnosed as Parkinson’s disease. Symptoms associated with progressive supranuclear palsy include:

  • Depression and apathy
  • Slowed movement (bradykinesia)
  • Impaired gait and balance
  • More frequent falling (backwards)
  • Blurred or double vision
  • Difficulty looking downward
  • Slurred speech
  • Stiff or awkward movements
  • Mild tremors in the hands
  • Light sensitivity
  • Sleep disturbances
  • Difficulty eating or swallowing
  • Altered mood, behavior, personality, or judgment
  • Impulsivity
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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