Abstract Details

Title Case Report: Expanded Access Treatment of a mid stage Huntington’s Disease (HD) Patient with a Novel Stabilized Polyunsaturated Fatty Acid Drug (RT001)

Topic Movement Disorders

Presentation(s) P5 - Poster Session 5 (8:00 AM-9:00 AM)

Poster/Presentation
Number 3-015

Objective To determine if RT001 could improve symptoms of HD in a single patient, observational study.

Background

HD is a neurodegenerative disease with motor, psychiatric, and cognitive deficits, which has no approved disease-modifying treatment. Stabilized polyunsaturated fatty acid drugs have been demonstrated in neurodegenerative disease preclinical models, including HD^1, to mitigate both cell death and disease symptoms. RT001 has been shown to be safe in a Phase 1/2 study in Friedreich’s ataxia and in multiple expanded access trials in serious neurological conditions. As HD displays lipid peroxidation damage, the risk/benefit to test RT001 in an HD patient was favorable.

1. Hatami et. al. “Deuterium-reinforced linoleic acid lowers lipid peroxidation and mitigates cognitive impairment in the Q140 knock in mouse model of Huntington’s disease”, The FEBS Journal, 6/18/2018

Design/Methods The patient is a 59 year old male diagnosed with HD in 2006, on stable doses of Amantadine, Bupropion, Sertraline, Tetrabenazine, and over-the-counter supplements, with a care team including caregivers, physical therapists (PT), and speech therapists. Weight was stable on a modified diet. Single subject IND and informed consent were obtained. Monthly physician visits and caregiver observations related to patient comfort, capabilities, and activities of daily living prior to and on the drug protocol were obtained. The patient started RT001 therapy at a dose of 2.9 g BID in March, 2019.

Results

During the first 6 months on drug, caregivers noted the following improvements: reduced nightly urination needs, better sleep, ability to independently transfer from chair to commode, improved restlessness, increased stamina during PT, marked improvement in speech and writing.

In quantitative measures, the patient improved on the Stroop Interference Test. The Unified Huntington’s Disease Rating Scale (UHDRS) improved from 39 to 28 points.

Conclusions

Summarized by the patient, “This medicine has helped me a lot”. A controlled trial of RT001 in HD patients should be considered.

Authors/Disclosures
Susan L. Perlman, MD (UCLA) PRESENTER	Dr. Perlman has nothing to disclose.
	No disclosure on file
	No disclosure on file
Frederic Heerinckx, PharmD (Retrotope, Inc.)	No disclosure on file

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