Abstract Details

Title RT001 to treat neurodegeneration—Case Study: Improvement in a single patient with Late Onset Tay-Sachs Disease (LOTS)

Topic Movement Disorders

Presentation(s) P3 - Poster Session 3 (5:30 PM-6:30 PM)

Poster/Presentation
Number 10-018

Objective We report on the use of RT001 in a single patient with LOTS.

Background Lipid peroxidation (LPO) is a self-propagating, free-radical chain reaction that amplifies toxic triggering effects in a variety of neurodegenerative conditions. RT001 is a di-deuterated isotope of linoleic acid (D-LA) that is readily incorporated into membranes and prevents oxidative damage.

Design/Methods

Baseline measurement of PK, Activities of Daily Living (ADL), 25 foot walk time (25FWT), and 6 minute walk distance (6MWD) were made. RT001 (2.7 g BID) was administered, and periodic repeat assessments were made. ADLs were measured individually on a scale of 0-5 across a 12 element panel representing speech, strength, coordination, etc.

Results

Plasma and RBC D-LA achieved significant percentages of plasma and RBC lipids after chronic administration within 30 d. Improvements in ADL, 25FWT, and 6MWD have also been seen. No major toxicities have been seen.

Time	D-LA (%) D-LA + H-LA		Composite ADL Score	# of ADLs improved	6MWD (M)	25FWT (Sec)
Time	Plasma	RBC	Composite ADL Score	# of ADLs improved	6MWD (M)	25FWT (Sec)
Baseline	0	0	0	n/a	112	12.7
30 d	36	28	+6	6	n/a	12.1
120 d	n/a	n/a	+9	8	129	11.2

Conclusions 1. D-LA is elongated to D-AA; both achieve significant plasma levels and RBC membrane incorporation within 1 month of administration.
2. Early efficacy signs of inhibition of disease progression and some regression were seen.
3. RT001 was well tolerated with no toxicities reported.

Authors/Disclosures
PRESENTER	No disclosure on file
Robert J. Molinari, PhD (Medstars, Inc.)	No disclosure on file
	No disclosure on file
Mikhail S. Shchepinov, PhD (Retrotope, Inc.)	No disclosure on file

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