Publications by authors named "Frederic Heerinckx"
Objectives: Friedreich ataxia is (FRDA) an autosomal recessive neurodegenerative disorder associated with intrinsic oxidative damage, suggesting that decreasing lipid peroxidation (LPO) might ameliorate disease progression. The present study tested the ability of RT001, a deuterated form of linoleic acid (D2-LA), to alter disease severity in patients with FRDA in a double-blind placebo-controlled trial.
Methods: Sixty-five subjects were recruited across six sites and received either placebo or active drug for an 11-month study.
Background: Progressive supranuclear palsy (PSP) is a progressive movement disorder associated with lipid peroxidation and intracerebral accumulation of tau. RT001 is a deuterium reinforced isotopologue of linoleic acid that prevents lipid peroxidation (LPO) through the kinetic isotope effect.
Methods: The effects of RT001 pre-treatment on various oxidative and bioenergetic parameters were evaluated in mesenchymal stem cells (MSC) derived from patients with PSP compared to controls.
Article Synopsis
- - RT001, the di-deutero isotopologue of linoleic acid ethyl ester (D2-LA), shows promise in resisting oxidative damage and is being studied for its pharmacokinetics in patients with neurodegenerative diseases.
- - In patients with Friedreich's ataxia, D2-LA is absorbed similarly to regular linoleic acid, reaching peak levels about 6 hours after taking it, with steady plasma concentrations achieved after 28 days.
- - Chronic dosing has been safe and well-tolerated in 59 participants over a span of clinical trials, with most subjects reaching therapeutic targets, supporting its potential for long-term therapeutic use in various conditions.
Background: INAD is an autosomal recessive neurogenetic disorder caused by biallelic pathogenic variants in PLA2G6. The downstream enzyme, iPLA, plays a critical role in cell membrane homeostasis by helping to regulate levels of phospholipids. The clinical presentation occurs between 6 months and 3 years with global developmental regression, hypotonia, and progressive spastic tetraparesis.
View Article and Find Full Text PDFBackground: Infantile neuroaxonal dystrophy (INAD) is a rare, autosomal recessive disease due to defects in and is associated with lipid peroxidation. RT001 is a di-deuterated form of linoleic acid that protects lipids from oxidative damage.
Methods: We evaluated the pharmacokinetics (PK), safety, and effectiveness of RT001 in two subjects with INAD (subject 1: 34 months; subject 2: 10 months).
Background: RT001 is a deuterated ethyl linoleate that inhibits lipid peroxidation and is hypothesized to reduce cellular damage and recover mitochondrial function in degenerative diseases such as Friedreich's ataxia.
Objective: To evaluate the safety, pharmacokinetics, and preliminary efficacy of RT001 in Friedreich's ataxia patients.
Design/methods: We conducted a phase I/II double-blind, comparator-controlled trial with 2 doses of RT001 in Friedreich's ataxia patients (9 subjects each cohort).